Perspectives
Quarterly Drug Pipeline: July 2024
July 25, 2024Clinical insights and competitive intelligence on anticipated drugs in development
Drug pipeline
Clinical insights and competitive intelligence on anticipated drugs in development.
Table of contents
Editor-in-chief’s message
Welcome to the Prime + Magellan Rx Quarterly Drug Pipeline! Dive into clinical insights and competitive intelligence on anticipated drugs in development, so you are well-sourced on the drug pipeline.
METHODOLOGY
The drug pipeline is complex and fluid. Our talented and committed team of clinical and analytics experts are excited to bring you this robust publication after thoughtful research. Specialty and traditional drugs that are covered under the pharmacy and medical benefits are featured. New molecular entities, pertinent new and expanded indications for existing medications, biosimilars, and regenerative medicines, such as gene and cellular therapies, are also profiled.
Quarterly Drug Pipeline details both agents submitted for U.S. Food and Drug Administration (FDA) review and those in phase 3 study with a likelihood to apply to the FDA. Our Deep Dives consider the evidence, the products’ potential to fill an unmet need or become the new standard of care, and the ability to replace existing therapies.
A market agnostic financial forecast primarily from Evaluate™ is included for select agents to assist payers with assessing the potential budgetary impact of the pipeline. Five-year projected annual U.S. sales are forecasted.
REFLECTION
Thus far in 2024, the agency has approved 22 novel drugs, roughly 21% fewer approvals compared to about the same time last year. Of note, most of the approvals so far in 2024 use at least one of the FDA’s expedited approval programs. Some of the noteworthy approvals include a new agent for Alzheimer’s disease, new option for chronic obstructive pulmonary disease (COPD), a new injectable for paroxysmal nocturnal hemoglobinuria (PNH), and a new therapy for primary biliary cholangitis (PBC). Moreover, first-time biosimilars to Eylea®, the first interchangeable biosimilar to eculizumab (Soliris®), and a respiratory syncytial virus (RSV) vaccine built on an mRNA platform were approved. While numbers do not tell the entire story, they do represent significant innovation in patient care and advance public health for the American public.
ON THE HORIZON
The FDA decisions for specialty medications (73%) and for Orphan Drugs (35%) continue to grow for agents with applications submitted to the FDA. Three agents are seeking FDA’s Accelerated Approval.
Notable FDA decisions expected for third quarter 2024 include several firsts:
- First psychedelic-assisted therapy for post-traumatic stress disorder (PTSD)
- First SC version of Ocrevus®
- First new options for Niemann-Pick disease type C
- First new mechanism for gastroparesis
- First new mechanism to treat schizophrenia in decades
We hope you enjoy the report!
Maryam Tabatabai
Vice President, Clinical Information
Editorial team
Maryam Tabatabai, PharmD
Editor-In-Chief
Vice President, Clinical Information
Carole Kerzic, RPh
Executive Editor
Clinical Pharmacist, Drug Information
Nicole Kjesbo, PharmD, BCPS
Executive Editor
Clinical Program Director, Pipeline
Andrea Henry, PharmD, MBA, BCPS
Specialty Drug Information Pharmacist
Katie Lockhart
Senior Manager, Analytics
Olivia Pane, PharmD, CDCES
Clinical Pharmacist, Drug Information
Michelle E. Pannone-Booth, PharmD
Senior Director, Clinical Account Services
The drug pipeline is fluid; the dates and information within this publication are subject to change. Nothing herein is or shall be construed as a promise or representation regarding past or future events and Prime Therapeutics/Magellan Rx Management expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Prime Therapeutics/Magellan Rx Management.
Pipeline
Deep dive
The Deep Dive section features an analysis for select pipeline drugs that are expected to be FDA-approved in the upcoming quarters. Selected agents are expected to have a high clinical and/or financial impact on healthcare. Agent selection considers the evidence — the products’ potential to fill an unmet need, become the new standard of care, or the ability to replace existing therapies. Typically, Deep Dives focus on new moieties, however, existing drugs that may offer a novel mechanism for existing conditions may be featured. Agents granted designations from the FDA to expedite review and/or support development and evaluation of pipeline drugs are also considered.
Specialty drug names appear in magenta throughout the publication.
CARDIOLOGY – acoramidis
acoramidis oral
Manufacturer: Bridgebio / AstraZeneca
PROPOSED INDICATIONS
Transthyretin amyloid cardiomyopathy (ATTR-CM)
CLINICAL OVERVIEW
Mechanism of action
Acoramidis is a tetrameric transthyretin (TTR) stabilizer.
Clinical trials
The randomized, double-blind, placebo-controlled, phase 3 ATTRibute-CM trial (NCT03860935) evaluated acoramidis in 632 patients with ATTR-CM and clinical heart failure. There were two co-primary endpoints. The first co-primary endpoint reported no statistically significant difference in the change in 6MWD with acoramidis compared to placebo (LS mean change, -26.51 meters versus -24.54 meters, respectively) at 12 months. The second co-primary endpoint was a four-step hierarchical analysis including (in order) death from any cause, CV-related hospitalization, the change from baseline in the N-terminal pro–B-type natriuretic peptide (NT-proBNP) level, and the change from baseline in the 6MWD after 30 months of treatment, which produced a win ratio of 1.8 (p<0.001), favoring acoramidis over placebo. In addition, the study showed that acoramidis significantly increased serum TTR levels at day 28 (p<0.001), which remained stable through month 30; an increase in serum TTR is associated with improved clinical outcomes. Acoramidis also demonstrated about a 25% relative risk reduction (RRR) in death from any cause and a 50% RRR in CV-related hospitalizations compared to placebo at 30 months. Similar safety profiles were observed with acoramidis and placebo.
Dosage and administration
In the ATTRibute-CM trial, acoramidis 800 mg was administered orally twice daily.
PLACE IN THERAPY
ATTR-CM results from the misfolding and accumulation of the tetrameric protein TTR into the extracellular space of the myocardium, which leads to progressive thickening of the ventricular wall and stiffening of the heart. Life expectancy after ATTR-CM diagnosis is about 2 to 6 years. There are two types of ATTR-CM. Wild-type ATTR-CM is due to misfolded wild-type (normal) TTR, the mechanism for which is unclear. Hereditary ATTR-CM is due to mutations in the TTR gene, which predisposes TTR to misfold. Based on limited data, an estimated 5,000 to 7,000 new cases of ATTR-CM are diagnosed each year in the U.S.; however, estimates are increasing due to advancements in diagnostic methods. ATTR-CM typically presents in individuals ≥ 60 years of age. Patients with ATTR-CM often have symptoms of chronic heart failure.
TTR is synthesized by the liver, and liver transplantation is the gold standard for treating transthyretin-related amyloidosis (ATTR). Multi-organ transplantation (heart, liver, and kidney) has been successful in slowing the natural course of the disease. Tafamidis/tafamidis meglumine (Vyndaqel®, Vyndamax®) is a TTR stabilizer approved by the FDA to treat ATTR-CM. The oral NSAID diflunisal may also stabilize TTR but is rarely used off-label for ATTR-CM due to poor tolerance and uncertain efficacy. Notably, standard treatments for CV disease such as beta-blockers, ACEIs, ARBs, diuretics or digoxin can worsen ATTR-CM symptoms.
Acoramidis leads to near-complete TTR stabilization with improved potency and stabilization compared to tafamidis. The ATTRibute-CM trial reported mixed data for acoramidis compared to placebo. While acoramidis did not significantly improve 6MWD at 12 months, significant benefit was demonstrated at 30 months in a hierarchical analysis that included all-cause mortality, CV-related hospitalization, NT-proBNP, and 6MWD. Notably, in its pivotal trial, tafamidis led to a 30% RRR in death from any cause and a 32% RRR in CV-related hospitalization compared to placebo at 30 months. If approved, acoramidis will be the second medication in the U.S. indicated to treat ATTR-CM and will directly compete with tafamidis agents.
FDA APPROVAL TIMELINE
November 29, 2024
FDA Designations: Orphan Drug
FINANCIAL FORECAST (reported in millions)
Year | 2024 | 2025 | 2026 | 2027 | 2028 |
Projected Yearly U.S. Sales | $16 | $110 | $293 | $591 | $866 |
NEUROLOGY – arimoclomol & N-acetyl-L-leucine
arimoclomol oral
Manufacturer: Zevra
PROPOSED INDICATIONS
Niemann-Pick disease type C (NPC)
CLINICAL OVERVIEW
Mechanism of action
Arimoclomol preserves cellular function and prevents cell death in cells experiencing lysosomal stress. It does this by amplifying the natural response to cellular stress through production of heat shock proteins (HSPs) that prevent protein misfolding.
Clinical trials
A 12-month, double-blind, phase 2/3 trial (NPC-002) evaluated arimoclomol for the treatment of NPC in addition to routine clinical care; stable miglustat therapy was allowed. Enrolled patients (n=50) were 2 to 18 years of age and were able to walk independently or with assistance. The primary endpoint was change from baseline to 12 months in NPC severity as measured by the 5-domain (5D)-NPC Clinical Severity Scale (NPCCSS) score, which includes parameters for ambulation, cognition, fine motor skills, speech, and swallowing (range, 0 to 25) with higher scores indicating greater impairment. The study demonstrated a 65% relative reduction in annual disease progression with arimoclomol based on the 5D-NPCCSS score (difference versus placebo, -1.4 point; p=0.046). Among patients also receiving miglustat, the difference in mean change in 5D-NPCCSS from baseline to 12 months with arimoclomol compared to placebo was -2.06 points (p=0.006). At 12 months, 50% and 37.5% of patients in the arimoclomol and placebo groups, respectively, had stable or improved disease (responders) (p=0.546). In addition, there was no significant difference between arimoclomol and placebo in the secondary endpoint of change from baseline to 12 months in the Scale for Assessment and Rating of Ataxia (SARA) score (LS mean difference, 0.28 points; p=0.79). Arimoclomol was well tolerated. Upper respiratory tract infection, decreased weight, and urticaria were reported more frequently with arimoclomol than placebo.
Interim data from an open-label extension (OLE) trial, in which all patients received arimoclomol, reported sustained reduction in NPC progression through 36 months. In addition, early treatment initiation with arimoclomol resulted in greater benefit compared to delayed start (in patients who switched from placebo to arimoclomol at the start of the OLE trial).
Dosage and administration
In the clinical trials, a weight-based dose (93 to 372 mg/day) of arimoclomol was administered by mouth or by feeding tube three times per day.
FDA APPROVAL TIMELINE
September 21, 2024
The FDA’s Genetic Metabolic Diseases Advisory Committee will review arimoclomol for the treatment of Niemann-Pick disease type C on August 2, 2024.
FDA designations: Breakthrough Therapy, Fast Track, Orphan Drug, RPD
FINANCIAL FORECAST (reported in millions)
Year | 2024 | 2025 | 2026 | 2027 | 2028 |
Projected Yearly U.S. Sales | $2 | $38 | $62 | $80 | $95 |
N-acetyl-L-leucine oral
Manufacturer: Intrabio
PROPOSED INDICATIONS
Niemann-Pick disease type C (NPC)
CLINICAL OVERVIEW
N-acetyl-L-leucine (NALL) is a modified, acetylated derivative of the natural essential amino acid leucine that normalizes energy production, improves lysosomal function and cellular signaling, and mitigates neuronal inflammation.
A double-blind, placebo-controlled, crossover trial (NCT05163288) evaluated NALL in 60 patients ≥ 4 years of age with NPC. Enrolled patients had mild to severe NPC symptoms correlating to a Scale for the Assessment and Rating of Ataxia (SARA) score between 7 to 34 at baseline; lower SARA scores indicate better neurologic status. Patients were randomized to NALL or placebo for 12 weeks, followed by alternate treatment for 12 weeks. The primary endpoint was mean change from baseline to week 12 in the SARA total score; however, the FDA requested use of a modified SARA (mSARA) score, which did not include stance and sitting as part of the assessment. After 12 weeks, the mean change from baseline in the SARA total score was -1.97 points with NALL and -0.6 points with (LS mean difference, -1.28 points; p<0.001). Based on the mSARA, the LS mean difference after NALL and after placebo therapies was -0.96 point (p-value not reported). Exploratory data for NPCCSS (scale, 0 to 54) showed a difference in change from baseline between NALL and placebo of -0.5 point. The difference in change in the Clinical Global Impression of Improvement (CGI-I) after NALL and after placebo therapies was -0.6. No serious TEAEs occurred with NALL. One case of anal incontinence, restless legs, and rosacea were reported as transient TEAEs associated with NALL.
Dosage and administration
In the clinical trial, NALL was administered orally two to three times per day. Patients 4 to 12 years of age received 2 g to 4 g per day and those ≥ 13 years of age received 4 g per day.
PLACE IN THERAPY
NPC is a rare, inherited, lysosomal storage disorder with an estimated incidence of 1 per 100,000 live births. It is caused by mutations in the NPC1 gene (95% of cases) or NPC2 gene (5% of cases) that result in dysfunctional NPC proteins giving rise to abnormal accumulation of lipids in tissues and organs, including the brain. Signs and symptoms of NPC include hepatosplenomegaly as well as neurologic manifestations, such as impaired motor function, cognitive function, swallowing and speech. Clinical presentation and disease progression depend on the patient’s age when neurological symptoms first appear, with younger age associated with more aggressive disease.
There is no cure for NPC. Miglustat has been used off-label in the U.S. It inhibits synthesis of glycolipids and has demonstrated slowing of disease progression in clinical trials. The use of miglustat may be considered in patients with mild to moderate neurologic, psychiatric, or cognitive manifestations, ideally starting use at the time of onset of neurologic manifestations.
If approved, arimoclomol and NALL would be the first agents FDA-approved for the treatment of NPC, a disorder with high unmet medical need. In clinical trials, both oral agents demonstrated improvement in neurologic status but used different tools to evaluate symptoms of cerebellar ataxia. Phase 3 trials for arimoclomol displayed long-term data with reduction in disease progression for up to 3 years based on the validated 5D-NPCCSS score, whereas improvement in neurologic status by NALL was revealed in results from a 12-week study based on the SARA score, which is validated for evaluating effects on spinocerebellar ataxia, but not for NPC. Moreover, data revealed neurologic symptoms improved during treatment with NALL; although, neurologic status declined when the agent was stopped. Notably, there is no validated biomarker for NPC or surrogate endpoint that indicates clinical improvement.
Intrathecal and IV formulations of cyclodextrin are also in phase 3 trials for NPC.
FDA APPROVAL TIMELINE
September 24, 2024
FDA designations: Fast Track, Orphan Drug, Priority Review, RPD
FINANCIAL FORECAST (reported in millions)
The financial forecast for NALL is not currently available.
ONCOLOGY – axatilimab
axatilimab IV
Manufacturer: Syndax
PROPOSED INDICATIONS
Chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy
CLINICAL OVERVIEW
Mechanism of action
Axatilimab is a monoclonal antibody targeting the colony stimulating factor 1 receptor (CSF-1R), which is expressed on monocytes and macrophages and is activated through its ligands, IL-34 and CSF-1.
Clinical trials
The randomized, open-label, phase 2 AGAVE-201 (NCT04710576) trial evaluated axatilimab in 241 allogeneic HCT recipients with recurrent or refractory cGVHD. Patients were randomized to one of three doses of axatilimab. The primary endpoint of ORR at 24 weeks (6 cycles) was 74% with 0.3 mg/kg every 2 weeks, 67% with 1 mg/kg every 2 weeks and 50% with 3 mg/kg every 4 weeks; complete response rates were 1%, 0% and 1%, respectively. In addition, 60%, 60% and 53% of patients in the respective cohorts maintaining response at 12 months. The most frequent grade ≥ 3 TEAEs reported were infection or infestations, elevated blood creatine phosphokinase and pneumonia.
Dosage and administration
In the clinical trial, axatilimab was administered IV every 2 weeks at doses of 0.3 mg/kg or 1 mg/kg, or 3 mg/kg every 4 weeks.
PLACE IN THERAPY
Chronic GVHD is a complication of allogenic HCT in which the transplanted cells attack host cells resulting in inflammation and fibrosis. Chronic GVHD can be life-threatening, affecting multiple organ systems. It is characterized by fibrosis and various clinical features like autoimmune disorders. It is estimated to occur in approximately 30% to 50% of allogenic HCT recipients. Typically, cGVHD develops within the first year after HCT, but can be present many years later.
High-dose corticosteroids are the standard first-line treatment for cGVHD, with response observed in about 50% of patients; however, more than half of patients will require second-line therapy within 2 years. The kinase inhibitors, ruxolitinib (Jakafi®; preferred), belumosudil (Rezurock®), and ibrutinib (Imbruvica®) are approved to treat cGVHD as second or later line therapy. Many other agents have been used off-label depending on organ involvement and include imatinib (Gleevec®), abatacept (Orencia®), alemtuzumab (Campath®, Lemtrada®), calcineurin inhibitors (cyclosporine, tacrolimus), etanercept (Enbrel®), hydroxychloroquine, interleukin-2, low-dose methotrexate, mTOR inhibitors, mycophenolate mofetil, pentostatin and rituximab (Rituxan® or biosimilars).
CSF-1R–dependent monocytes play a key role in profibrotic (M2) macrophage differentiation, polarization, and function and promote sustained inflammation and tissue injury during cGVHD. In addition, these cells accelerate maladaptive tissue repair and fibrosis. If approved, axatilimab will provide a new mechanism in combating cGVHD, a disorder that continues to have unmet medical need.
FDA APPROVAL TIMELINE
August 28, 2024
FDA designations: Fast Track, Orphan Drug, Priority Review
FINANCIAL FORECAST (reported in millions)
Year | 2024 | 2025 | 2026 | 2027 | 2028 |
Projected Yearly U.S. Sales | $1 | $32 | $72 | $91 | $116 |
NEUROLOGY / GENE THERAPY – eladocagene exuparvovec (Upstaza™)
eladocagene exuparvovec (Upstaza™) intraputaminal
Manufacturer: PTC Therapeutics
PROPOSED INDICATIONS
Aromatic L-amino acid decarboxylase (AADC) deficiency
CLINICAL OVERVIEW
Mechanism of action
Eladocagene exuparvovec is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy. It delivers a functional dopa decarboxylase (DDC) gene to increase AADC enzyme levels and restore dopamine production.
Clinical trials
In a study conducted in Taiwan, 26 patients were enrolled in three consecutive trials (including compassionate use, phase 1/2, and phase 2b) with similar treatment protocols and completed follow-up evaluations at 1-year. The mean age at the time of treatment was 4.1 years (range, 1.7 to 8.5 years). The mean duration of follow up was 5.4 years of age (range, 2 to 10.2 years of age). Motor ability was evaluated using the Peabody Developmental Motor Scales–Second Edition (PDMS-2) that assesses fine motor function and the Alberta Infant Motor Scale (AIMS) that assesses gross motor skills. Eladocagene exuparvovec led to a significant increase in the PDMS-2 score from baseline (10.4 ± 5.4) to 1 year (80.5 ± 43.4; n=25), 2 years (114.5 ± 55.2; n=22), and 5 years (116.1 ± 59.8; n=11) after the dose (p<0.01 for each). Likewise, the AIMS score significantly increased from baseline (1.8 ± 1.8) at 1 year (18.8 ± 11), 2 years (26.9 ± 15.5), and 5 years (24.5 ± 15) after the dose (p<0.001 for each). Secondary endpoints included change in the levels of the dopamine and serotonin metabolites, homovanillic acid (HVA) and 5-hydroxyindoleacetic acid (5-HIAA), in the cerebrospinal fluid after 12 months; significant increases in HVA were reported (p<0.001) but not in 5-HIAA. While improvements were observed in all patients who received eladocagene exuparvovec, greater improvement was associated with treatment at a younger age. Adverse events reported include post-surgery complications such as cerebrospinal fluid leakage, which were managed with SOC. No treatment-related brain injuries occurred. Mild to moderate dyskinesia was reported in most patients and resolved within a few months.
Dosage and administration
Eladocagene exuparvovec is administered as a one-time dose via intraputaminal (base of forebrain) infusion.
PLACE IN THERAPY
AADC deficiency is an ultra-rare, autosomal recessive, neurometabolic disorder. It is caused by a mutation in the DDC gene that encodes the production of AADC, an enzyme that plays a key role in the production of serotonin, dopamine, epinephrine, and norepinephrine. Impairment of these neurotransmitters leads to developmental delays, intellectual disabilities, and life-long movement disorders and autonomic nervous system dysfunction. Symptom onset usually occurs within the first six months of life. Approximately 150 to 350 cases have been identified in the literature, with it reported more often in certain Asian populations (Taiwanese, Japanese, Chinese). Symptom severity can range from relatively mild to very severe due to a broad phenotypic spectrum for the disease. Individuals with AADC deficiency are at an increased risk of early death due to complications such as infection or an acute cardiorespiratory event. Many who are affected do not live through childhood, but those with milder disease may reach adulthood.
There is no cure for AADC deficiency. Medications used to treat the condition include selective dopamine agonists, monoamine oxidase inhibitors (MAOIs) and pyridoxine. However, treatment response varies greatly among affected individuals. Agents for symptom management, such as anticholinergics, benzodiazepines, alpha-adrenoreceptor blockers and melatonin may also be used.
If approved, the one-time gene therapy, eladocagene exuparvovec, will be the first treatment to target the underlying cause of AADC deficiency. In clinical trials, significant and durable improvement in motor skills was reported.
FDA APPROVAL TIMELINE
November 13, 2024
FDA Designations: Orphan Drug, Priority Review, RPD, seeking Accelerated Approval
FINANCIAL FORECAST (reported in millions)
Year | 2024 | 2025 | 2026 | 2027 | 2028 |
Projected Yearly U.S. Sales | $3 | $19 | $33 | $46 | $57 |
IMMUNOLOGY – garadacimab
garadacimab SC
Manufacturer: CSL
PROPOSED INDICATIONS
Prophylactic treatment for hereditary angioedema (HAE)
CLINICAL OVERVIEW
Mechanism of action
Garadacimab is a recombinant monoclonal antibody that targets activated factor XIIa (FXIIa). It inhibits the kallikrein-kinin cascade early in the cascade.
Clinical trials
The 6-month, double-blind, placebo-controlled VANGUARD trial (NCT04656418) evaluated garadacimab as a prophylactic treatment in patients ages ≥ 12 years with type I or type II HAE (n=65). During the treatment period, garadacimab reduced the number of HAE attacks per month (primary endpoint) by 87% compared with placebo (0.27 versus 2.01, respectively; p<0.0001). The median number of HAE attacks per month was zero for garadacimab and 1.35 for placebo. The most common TEAEs reported were upper-respiratory tract infections, nasopharyngitis and headaches. No increased risk of bleeding or thromboembolic events was observed. The extension phase of the VANGUARD trial is ongoing.
Dosage and administration
In the clinical trial, garadacimab was administered SC as a 400 mg (two 200 mg injections) loading dose, followed by five additional 200 mg SC self-administered (or caregiver-administered) doses once monthly.
PLACE IN THERAPY
HAE is a rare, dominant autosomal genetic disorder that affects approximately 6,000 individuals in the U.S. Patients with HAE have low levels of endogenous or functional C1 esterase inhibitor (C1-INH). There are two types of C1-INH deficient HAE. Type I, in which the body does not produce enough C1-INH, accounts for about 85% of cases. Type II HAE is caused by the presence of normal or high levels of a dysfunctional C1-INH. HAE is characterized by recurrent episodes of nonpruritic, nonpitting, SC or submucosal edema involving the skin or mucosal tissues of the upper respiratory and GI tracts. Although swelling can resolve spontaneously in several days, without treatment, laryngeal edema can be fatal, and the pain of GI attacks can be incapacitating. Symptoms may begin as early as 2 years of age and persist throughout life with unpredictable severity and frequency of attacks. It is thought that minor trauma and stress can lead to an attack; although, many attacks occur without any apparent trigger.
The IV-administered C1-INH (Cinryze®), SC-administered C1-INH (Haegarda®), SC-administered plasma kallikrein inhibitor lanadelumab-flyo (Takhzyro®), and orally administered plasma kallikrein inhibitor berotralstat (Orladeyo®) are available for long-term HAE prophylaxis in children and adults. If approved, once monthly SC garadacimab, which has the potential to be administered by the patient or caregiver, would become the first treatment for HAE in the U.S. that targets activated FXIIa. It will compete with Haegarda® and Takhzyro® that are self- or caregiver-administered every 2 weeks; Takhzyro’s® dosing interval can be extended to every 4 weeks in patients who are well-controlled for 6 months.
FDA APPROVAL TIMELINE
October to December 2024
FDA Designations: Fast Track, Orphan Drug
FINANCIAL FORECAST (reported in millions)
Year | 2024 | 2025 | 2026 | 2027 | 2028 |
Projected Yearly U.S. Sales | $28 | $64 | $101 | $138 | $175 |
METABOLIC – govorestat
govorestat oral
Manufacturer: Applied Therapeutics
PROPOSED INDICATIONS
Classic galactosemia
CLINICAL OVERVIEW
Mechanism of action
Govorestat is an aldose reductase inhibitor (ARI) that penetrates the CNS.
Clinical trials
The placebo-controlled, phase 2/3, ACTION-Galactosemia Kids study (NCT04902781) evaluated govorestat in 47 patients ages 2 to 17 years with galactosemia. The primary endpoint was the Global Statistical Test, a composite sum of change of the following measures: Oral and Written Language Scales-Second Edition (OWLS-2) Oral Expression (OE), OWLS-2 Listening Comprehension (LC), Behavior Assessment System for Children-Third Edition (BASC-3) Activities of Daily Living (ADL), and BASC-3 Behavior Symptoms Index (BSI). While the primary endpoint was not met (p=0.103), there was systematic improvement over time for the overall primary endpoint. Govorestat demonstrated improvement in galactitol biomarker levels and clinically meaningful improvements in activities of daily living, behavior, cognition, adaptive skills, and tremor. Statistically significant improvements were seen in behavior and activities of daily living (p=0.0205) and tremor (p=0.0428, at 18 months). Govorestat was well tolerated. The study includes an open-label extension as an active treatment extension for patients who received placebo treatment.
A long-term open-label study in adults with galactosemia is ongoing.
Dosage and administration
In the clinical trials, govorestat was given orally. In Part A, govorestat was administered at 5 mg/kg for all ages. Part B administered the drug at an optimum dose determined in Part A.
PLACE IN THERAPY
Classic galactosemia is a rare, genetic disorder caused by mutations in the GALT gene that leads to a deficiency of the galactose-1-phosphate uridylyl transferase (GALT) enzyme. Patients lack the ability to convert galactose to glucose, which leads to the accumulation of the toxic metabolite galactitol in tissues and organs. Newborns are screened for classic galactosemia as early diagnosis and a lactose-and galactose-free diet is imperative to avoid profound intellectual disability, liver failure and death within days to weeks from birth. However, since galactose is produced by the body, long-term complications will still occur even with a restricted diet. Classic galactosemia is estimated to affect 1 in 16,000 to 1 in 48,000 births.
There is currently no pharmacologic treatment for classic galactosemia. If approved, govorestat will be the only medication available to treat the disease. In clinical trials, while the composite primary endpoint was not met, clinically meaningful improvements, including in activities of daily living, behavior, cognition, adaptive skills, and tremor were experienced with govorestat.
Govorestat is also in phase 3 trials for sorbitol dehydrogenase (SORD) deficiency.
FDA APPROVAL TIMELINE
November 28, 2024
An FDA advisory committee meeting to discuss the application is planned.
FDA designations: Fast Track, Orphan Drug, Priority Review, RPD
FINANCIAL FORECAST (reported in millions)
The financial forecast for govorestat is not currently available.
ONCOLOGY / CELLULAR THERAPY – obecabtagene autoleucel
obecabtagene autoleucel IV
Manufacturer: Autolus
PROPOSED INDICATIONS
Relapsed/refractory (R/R) adult B-cell acute lymphoblastic leukemia (ALL)
CLINICAL OVERVIEW
Mechanism of action
Obecabtagene autoleucel (obe-cel) is an autologous CD19-directed CAR T therapy.
Clinical trials
The ongoing, open-label, single-arm, phase 1b/2 FELIX trial (NCT04404660) evaluated obe-cel in adults with R/R B-cell ALL. The study included 126 patients with morphological disease ≥ 5% bone marrow (BM) blasts (cohort A), or in second or later complete remission (CR)/CR with incomplete hematologic recovery (CRi) with measurable residual disease (MRD) (cohort B), or with isolated extramedullary disease (EMD) (cohort C). Philadelphia chromosome-positive (Ph+) B-cell ALL was reported in 27% of patients. Enrollees had a median of 2 (range, 1 to 6) prior lines of therapy. Topline results of pooled data from all three cohorts revealed, at a median follow-up of 11 months (range, 0.9 to 30.6 months), CR/CRi (primary endpoint) occurred in 77% of patients who received obe-cel, with CR in 57% of patients. Among evaluable responders, 96% were considered to have MRD-negative status. The median DOR had not been reached. Grade ≥ 3 CRS occurred in 2.4% of patients, and grade ≥ 3 immune effector cell associated neurotoxicity syndrome (ICANS) occurred in 7.1% of patients.
Dosage and administration
In the FELIX trial, after pre-conditioning with chemotherapy (cyclophosphamide and fludarabine), obe-cel was administered IV at a target dose of 410 × 106 cells as a split dose on day 1 and day 10 (± 2 days).
PLACE IN THERAPY
In ALL, the bone marrow produces an excessive number of lymphocytes. Nearly 30% of patients with ALL are diagnosed at ≥ 45 years of age and nearly 14% are diagnosed at ≥ 65 years of age. In addition, about 75% of adult cases develop from B cell lineage, and 25% emerge from T cell precursors. Risk factors for developing ALL in adulthood include age > 70 years, prior treatment with chemotherapy or radiation therapy, environmental radiation exposure, and certain genetic conditions (e.g., Down syndrome). In general, prognosis of ALL decreases with age, with a 5-year survival rate of 20% to 40% in adults.
Pharmacologic agents that target B-cell surface antigens have demonstrated improved response in R/R B-cell ALL. This includes select tyrosine kinase inhibitors (in Ph+ B-cell ALL only; CR/CRi rates, 42%-47%), a bispecific CD19-directed CD3 T-cell engager (blinatumomab [Blincyto®]; CR rate, 43%), an anti-CD22 monoclonal antibody (inotuzumab ozogamicin [Besponsa®]; CR/CRi rate, 78.4%), and CD19-directed CAR T cell therapies (brexucabtagene autoleucel [Tecartus®] and tisagenlecleucel [Kymriah®]; CR rate, 69%-81%). Allogeneic HCT may also be considered in eligible patients. Choice of treatment should be individualized based on the presence or absence of target antigen (CD19 and CD22), prior exposure to one of these agents, patient immune status, and disease burden.
HCT is currently the only treatment considered a cure for R/R ALL, but many patients are not eligible for HCT. Notably, the CAR T cell therapies have served as a bridge, allowing patients with initial poor remission status to become eligible for HCT; however, high rates of toxicities, including CRS and neurologic toxicities, have complicated their use.
Obe-cel is a CAR T cell therapy that was created with a fast target binding off-rate that minimizes excessive activation of the programmed T cells. Clinical trials have demonstrated that its fast off-rate profile reduces toxicity and T cell exhaustion, leading to improved persistence and high levels of durable remissions compared to existing CD19-directed CAR T cell therapies in adults with R/R ALL. If approved, obe-cel will compete with the other CD19 CAR T cell therapies, Kymriah® and Tecartus®, in the adult R/R B-cell ALL setting. While all three CAR T therapies demonstrate high response rates, obe-cel’s improved safety profile may differentiate it from the other agents. In non-comparative clinical trials in patients with ALL, the rates of grade ≥ 3 CRS were 2.4% with obe-cel, 26% with Tecartus®, and 48% with Kymriah®. Likewise, serious neurologic toxicities were reported at rates of 7.1% with obe-cel, 35% with Tecartus®, and 22% with Kymriah®.
FDA APPROVAL TIMELINE
November 16, 2024
FDA designations: Orphan Drug, RMAT
FINANCIAL FORECAST (reported in millions)
Year | 2024 | 2025 | 2026 | 2027 | 2028 |
Projected Yearly U.S. Sales | $3 | $56 | $83 | $121 | $129 |
GASTROINTESTINAL – tradipitant
tradipitant oral
Manufacturer: Vanda / Eli Lilly
PROPOSED INDICATIONS
Gastroparesis (GP)
CLINICAL OVERVIEW
Mechanism of action
Tradipitant is a neurokinin receptor 1 (NK-1R) antagonist and may treat gastroparesis by acting centrally in the nausea-vomiting centers of the brain and peripherally in the smooth muscle of the intestines.
Clinical trials
A double-blind, placebo-controlled, phase 3 trial (NCT04028492) assessed the efficacy of tradipitant in relieving GP symptoms in patients with idiopathic and diabetic GP. The primary endpoint was the change from baseline to week 12 (days 78 to 84) in average nausea severity as measured by the Gastroparesis Core Symptom Daily Diary (GCSDD) in the ITT population (n=201). The GCSDD records the patient’s rating of the worst occurrence of each cardinal symptom of gastroparesis in the past 24 hours on a scale of 0 (no symptoms) to 5 (very severe). The study demonstrated that the difference between tradipitant and placebo in the primary endpoint was not significant (-1.55 versus -1.49, respectively; p=0.741). However, exploratory analysis revealed that an unbalanced use of rescue medications (e.g., ondansetron, prochlorperazine, promethazine) may have confounded the results. Among patients who did not use rescue medication, the average nausea severity was significantly improved for patients on tradipitant compared with placebo, overcoming the large placebo effect, at week 12 (-2.01 versus -1.32; p=0.0372). In addition, average nausea severity was significantly improved at week 12 for patients in the severity adjustment population, which excluded patients with zero or the highest severity of vomiting (p=0.0276) and significant effects were seen with adequate tradipitant exposure based on a pharmacokinetic analysis. The incidence and type of adverse events reported were similar between the treatment groups and included diarrhea, urinary tract infection, sinusitis, dizziness, and headache. Discontinuation due to adverse effects occurred in 6.9% of patients in the tradipitant group and 3% of patients in the placebo group.
In a double-blind, placebo-controlled, phase 2 trial (NCT02970968), tradipitant led to a significant decrease in GCSDD score at week 4 compared with placebo (-1.25 versus -0.73, respectively; p=0.0099) and a significant increase in the percentage of nausea-free days a week 4 (28.8% versus 15%, respectively; p=0.016) among 152 adults with idiopathic or diabetic GP.
Dosage and administration
In the clinical trials, tradipitant 85 mg was administered orally twice daily.
Place in therapy
GP is a chronic disorder of delayed gastric emptying without mechanical obstruction. Its characteristic symptoms of early satiety, nausea, vomiting, bloating, and abdominal pain have a significant effect on QOL, morbidity, and mortality. Definite diagnosis of GP (based on gastric emptying scintigraphy testing) was estimated at 21.5 per 100,000 persons in the U.S. in 2018; however, actual prevalence may be much higher since many patients remain undiagnosed. GP occurs mostly in women, with causes including diabetes, medications, surgery, or unknown.
Management of GP includes a diet that promotes gastric emptying (e.g., 4 to 5 small meals per day, decreased fat and fiber, increased liquid nutrition). Use of prokinetic medications, such as metoclopramide, cisapride (investigational limited access program) and erythromycin (4 weeks, off-label), to increase gastric emptying rate, and antiemetics for symptom control are suggested based on low quality of evidence. Notably, metoclopramide is FDA-approved for acute and recurrent diabetic GP, but its use is limited to a 3-month duration due to the risk of developing tardive dyskinesia.
NK-1R antagonists are proven to be effective in reducing nausea and vomiting in chemotherapy-induced emesis. If approved, tradipitant will be the first agent of this class to treat GP symptoms and the first new mechanism for GP to be approved in over 40 years; however, in clinical trials, it demonstrated mixed results in improving nausea in patients with GP. Tradipitant is also in phase 3 trials for motion sickness.
FDA APPROVAL TIMELINE
September 18, 2024
FINANCIAL FORECAST (reported in millions)
The financial forecast for tradipitant is not currently available.
ONCOLOGY – zanidatamab
zanidatamab IV
Manufacturer: Jazz / Beigene
PROPOSED INDICATIONS
Previously treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer (BTC)
CLINICAL OVERVIEW
Mechanism of action
Zanidatamab is a HER2-targeted bispecific antibody that provides dual HER2 signal blockade, removal of HER2 protein from the cell surface, and immune-mediated cytotoxicity.
Clinical trials
Zanidatamab monotherapy was evaluated in the open-label, single-arm, phase 2b HERIZON-BTC-01 trial (NCT05152147) in patients with unresectable, locally advanced, or metastatic HER2+ BTC. Enrolled patients were previously treated with gemcitabine-based therapy but were naïve to HER2-targeted therapy. Cohort 1 (n=80) included patients with in situ hybridization [ISH] positive and immunohistochemistry [IHC] 2+ or 3+ disease. Long-term follow-up data confirmed an ORR (primary endpoint) of 41.3%, with a complete response rate of 2.5%. Among patients with IHC 3+ (HER2+) tumors, the ORR was 51.6%, median DOR was 14.9 months, median PFS was 7.2 months, and median OS was 18.1 months. Among patients with IHC 2+ (borderline HER2 status) tumors, the ORR was 5.6%, median OS was 5.2 months, and median PFS was 1.7 months. In the single patient with IHC 2+ disease who responded to therapy, the DOR was 7.5 months. Zanidatamab was generally well tolerated, with a manageable safety profile. A serious increase in AST and ALT was experienced by one patient, which did not lead to treatment discontinuation.
Dosage and administration
In the HERIZON-BTC-01 trial, zanidatamab was administered IV as 20 mg/kg every 2 weeks.
PLACE IN THERAPY
BTCs include gallbladder cancer and extrahepatic cholangiocarcinoma (CCA). In 2023, it was estimated that 12,220 new cases of BTC would be diagnosed in the U.S. and 4,510 deaths would occur due to the condition. BTCs are associated with a poor prognosis, as patients often have advanced disease upon diagnosis. Radiation therapy may be used in patients with unresectable disease. Systemic treatment for unresectable or metastatic BTC includes gemcitabine/cisplatin ± durvalumab (Imfinzi®; preferred) or pembrolizumab (Keytruda®). Subsequent therapy includes FOLFOX (folinic acid, fluorouracil, and oxaliplatin). HER2 overexpression or pathway activation is exhibited in about 5% to 20% of CCAs, and 15% to 30% of gallbladder cancer, for which off-label fam-trastuzumab deruxtecan-nxki (Enhertu®, for IHC 3+), trastuzumab/pertuzumab (Phesgo®) and trastuzumab + tucatinib (Herceptin® + Tukysa®) are recommended for HER2+ tumors after disease progression.
If approved, zanidatamab will be the first HER2-targeted treatment specifically indicated for patients with HER2+ locally advanced or metastatic biliary tract cancer. It demonstrated efficacy particularly in patients with IHC 3+ tumors. Zanidatamab is being evaluated in combination with first-line cisplatin and gemcitabine for the treatment of HER2+ BTC in a phase 2 trial (NCT03929666). Zanidatamab is also in phase 2 and phase 3 trials for HER2+ breast cancer and HER2+ gastric cancer, respectively.
FDA APPROVAL TIMELINE
November 29, 2024
FDA Designations: Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, seeking Accelerated Approval
FINANCIAL FORECAST (reported in millions)
Year | 2024 | 2025 | 2026 | 2027 | 2028 |
Projected Yearly U.S. Sales | $0 | $4 | $11 | $15 | $22 |
Pipeline
Keep on your radar
Notable agents that are further from approval have been identified in this unique watch list. These are products with the potential for significant clinical and financial impact. Their development status is being tracked on the Quarterly Drug Pipeline radar. These pipeline products, their respective class or proposed indication, as well as an estimated financial forecast for the year 2028, are displayed. The financials are projected total annual U.S. sales, reported in millions.
Table view
Drug Generic Name | Therapeutic category | July2024 Pipeline – Total US sales for 2028 (Dollars in millions) |
botaretigene sparoparvovec | Ophthalmology / Gene therapy | $158 |
crinecerfont | Endocrine | $325 |
datopotamab deruxtecan | Oncology | $1,429 |
etripamil | Cardiovascular | $134 |
inavolisib | Oncology | $397 |
lebrikizumab | Immunology | $997 |
linvoseltamab | Oncology | $193 |
marstacimab | Hematology | $197 |
olezarsen | Cardiovascular | $254 |
palopegteriparatide | Endocrine | $694 |
RGX-121 | Metabolic / Gene therapy | $71 |
sebetralstat | Immunology | $192 |
vanzacaftor/tezacaftor/deutivacaftor | Respiratory | $4,191 |
zenocutuzumab | Oncology | $82 |
Pipeline
Drug list
The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2026. It is not intended to be a comprehensive inventory of all drugs in the pipeline; emphasis is placed on drugs in high-impact categories. Investigational drugs with a recent Complete Response Letter (CRL) are also reported.
Gene & cellular therapies
Submitted new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
afamitresgene autoleucel | Adaptimmune | Synovial sarcoma | IV | BLA; Orphan Drug, Priority Review, RMAT | 08/04/2024 |
remestemcel-L (Ryoncil®) | Mesoblast | Acute GVHD (steroid-refractory) | IV | BLA; Fast Track, Orphan Drug | Sep 2024-Jan 2025 |
eladocagene exuparvovec (Upstaza™) | PTC Therapeutics | Aromatic L-amino acid decarboxylase (AADC) deficiency | Intraputaminal | BLA; seeking Accelerated Approval, Orphan Drug, Priority Review, RPD | 11/13/2024 |
obecabtagene autoleucel | Autolus | ALL (R/R, B-cell, adults) | IV | BLA; Orphan Drug, RMAT | 11/16/2024 |
tabelecleucel | Pierre Fabre | Epstein-Barr virus-associated post-transplant lymphoproliferative disease | IV | BLA; Breakthrough Therapy, Orphan Drug, Priority Review | 01/15/2025 |
Submitted supplemental drugs
None
Phase 3 new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
AAV8-ranibizumab (RGX-314) | Abbvie | DME; Wet AMD | Subretinal | BLA; Orphan Drug | TBD |
aglatimagene besadenovec | Candel | Prostate cancer | Intratumoral | BLA; Fast Track | TBD |
botaretigene sparoparvovec | Janssen | Retinitis pigmentosa | Subretinal | BLA; Fast Track, Orphan Drug | TBD |
deramiocel | Nippon Shinyaku | DMD | IV | BLA; Orphan Drug, RMAT, RPD | TBD |
dirloctocogene samoparvovec | Genentech | Hemophilia A | IV | BLA; Breakthrough Therapy, Orphan Drug | TBD |
giroctocogene fitelparvovec | Pfizer | Hemophilia A | IV | BLA; Fast Track, Orphan Drug, RMAT | TBD |
OCU400 | Ocugen | Retinitis pigmentosa | Subretinal | BLA; Orphan Drug, RMAT | TBD |
RGX-121 | Regenxbio | Mucopolysaccharidosis II (Hunter syndrome) | Injection | BLA; seeking Accelerated Approval, Fast Track, Orphan Drug, RMAT, RPD | TBD |
rilparencel | Prokidney | CKD | Injection | BLA; RMAT | TBD |
Phase 3 supplemental drugs
None
Submitted new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
afamitresgene autoleucel | Adaptimmune | Synovial sarcoma | IV | BLA; Orphan Drug, Priority Review, RMAT | 08/04/2024 |
remestemcel-L (Ryoncil®) | Mesoblast | Acute GVHD (steroid-refractory) | IV | BLA; Fast Track, Orphan Drug | Sep 2024-Jan 2025 |
eladocagene exuparvovec (Upstaza™) | PTC Therapeutics | Aromatic L-amino acid decarboxylase (AADC) deficiency | Intraputaminal | BLA; seeking Accelerated Approval, Orphan Drug, Priority Review, RPD | 11/13/2024 |
obecabtagene autoleucel | Autolus | ALL (R/R, B-cell, adults) | IV | BLA; Orphan Drug, RMAT | 11/16/2024 |
tabelecleucel | Pierre Fabre | Epstein-Barr virus-associated post-transplant lymphoproliferative disease | IV | BLA; Breakthrough Therapy, Orphan Drug, Priority Review | 01/15/2025 |
Submitted supplemental drugs
None
Phase 3 new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
AAV8-ranibizumab (RGX-314) | Abbvie | DME; Wet AMD | Subretinal | BLA; Orphan Drug | TBD |
aglatimagene besadenovec | Candel | Prostate cancer | Intratumoral | BLA; Fast Track | TBD |
botaretigene sparoparvovec | Janssen | Retinitis pigmentosa | Subretinal | BLA; Fast Track, Orphan Drug | TBD |
deramiocel | Nippon Shinyaku | DMD | IV | BLA; Orphan Drug, RMAT, RPD | TBD |
dirloctocogene samoparvovec | Genentech | Hemophilia A | IV | BLA; Breakthrough Therapy, Orphan Drug | TBD |
giroctocogene fitelparvovec | Pfizer | Hemophilia A | IV | BLA; Fast Track, Orphan Drug, RMAT | TBD |
OCU400 | Ocugen | Retinitis pigmentosa | Subretinal | BLA; Orphan Drug, RMAT | TBD |
RGX-121 | Regenxbio | Mucopolysaccharidosis II (Hunter syndrome) | Injection | BLA; seeking Accelerated Approval, Fast Track, Orphan Drug, RMAT, RPD | TBD |
rilparencel | Prokidney | CKD | Injection | BLA; RMAT | TBD |
Phase 3 supplemental drugs
None
Biosimilars
Submitted new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
aflibercept (biosimilar to Regeneron’s Eylea®) | Amgen | DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD | Intravitreal | BLA | Jul-Sep 2024 |
ustekinumab (biosimilar to Janssen’s Stelara®) | Formycon | PSO; PsA; CD; UC | IV, SC | BLA | September 2024 |
ustekinumab (biosimilar to Janssen’s Stelara®) | Intas | PSO; PsA; CD; UC | IV, SC | BLA | Oct-Nov 2024 |
denosumab (biosimilar to Amgen’s Prolia®/Xgeva®) | Celltrion | Osteoporosis/osteopenia | SC | BLA | 12/01/2024 |
trastuzumab (biosimilar to Genentech’s Herceptin®) | Tanvex | Breast cancer (HER2+); Gastric cancer (HER2+) | IV | BLA | 01/06/2025 |
insulin aspart (biosimilar to Novo Nordisk’s Novolog®) | Amphastar | T1DM; T2DM | SC | BLA | 01/10/2025 |
tocilizumab (biosimilar to Genentech’s Actemra®) | Celltrion | RA; Giant cell arteritis; Systemic sclerosis-associated interstitial lung disease; JIA (polyarticular, systemic); COVID-19; CRS | IV, SC | BLA | 01/28/2025 |
aflibercept (biosimilar to Regeneron’s Eylea®) | Celltrion | DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD | Intravitreal | BLA | pending |
insulin glargine (biosimilar to Sanofi’s Lantus®) | Gan & Lee/Sandoz | T1DM; T2DM | SC | BLA | pending |
insulin lispro (biosimilar to Eli Lilly’s Humalog®) | Gan & Lee/Sandoz | T1DM; T2DM | SC | BLA | pending |
Submitted supplemental drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
adalimumab-bwwd 40 mg/0.4 mL (Hadlima™) (biosimilar to Abbvie’s Humira®) | Organon | RA; AS; PSO; PsA; JIA; CD; UC; HS; Uveitis for interchangeability | SC | sBLA | 09/06/2024 |
omalizumab (biosimilar to Genentech’s Xolair®) | Celltrion | Asthma; Chronic rhinosinusitis; Food allergies; Nasal polyposis; Urticaria for interchangeability | SC | sBLA | 03/10/2025 |
Phase 3 new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
aflibercept (biosimilar to Regeneron’s Eylea®) | Novartis | DME; Wet AMD | Intravitreal | BLA | TBD |
bevacizumab (biosimilar to Genentech’s Avastin®) | Essex | DME; Wet AMD | Intravitreal | BLA | TBD |
Phase 3 supplemental drugs
None
Submitted new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
aflibercept (biosimilar to Regeneron’s Eylea®) | Amgen | DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD | Intravitreal | BLA | Jul-Sep 2024 |
ustekinumab (biosimilar to Janssen’s Stelara®) | Formycon | PSO; PsA; CD; UC | IV, SC | BLA | September 2024 |
ustekinumab (biosimilar to Janssen’s Stelara®) | Intas | PSO; PsA; CD; UC | IV, SC | BLA | Oct-Nov 2024 |
denosumab (biosimilar to Amgen’s Prolia®/Xgeva®) | Celltrion | Osteoporosis/osteopenia | SC | BLA | 12/01/2024 |
trastuzumab (biosimilar to Genentech’s Herceptin®) | Tanvex | Breast cancer (HER2+); Gastric cancer (HER2+) | IV | BLA | 01/06/2025 |
insulin aspart (biosimilar to Novo Nordisk’s Novolog®) | Amphastar | T1DM; T2DM | SC | BLA | 01/10/2025 |
tocilizumab (biosimilar to Genentech’s Actemra®) | Celltrion | RA; Giant cell arteritis; Systemic sclerosis-associated interstitial lung disease; JIA (polyarticular, systemic); COVID-19; CRS | IV, SC | BLA | 01/28/2025 |
aflibercept (biosimilar to Regeneron’s Eylea®) | Celltrion | DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD | Intravitreal | BLA | pending |
insulin glargine (biosimilar to Sanofi’s Lantus®) | Gan & Lee/Sandoz | T1DM; T2DM | SC | BLA | pending |
insulin lispro (biosimilar to Eli Lilly’s Humalog®) | Gan & Lee/Sandoz | T1DM; T2DM | SC | BLA | pending |
Submitted supplemental drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
adalimumab-bwwd 40 mg/0.4 mL (Hadlima™) (biosimilar to Abbvie’s Humira®) | Organon | RA; AS; PSO; PsA; JIA; CD; UC; HS; Uveitis for interchangeability | SC | sBLA | 09/06/2024 |
omalizumab (biosimilar to Genentech’s Xolair®) | Celltrion | Asthma; Chronic rhinosinusitis; Food allergies; Nasal polyposis; Urticaria for interchangeability | SC | sBLA | 03/10/2025 |
Phase 3 new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
aflibercept (biosimilar to Regeneron’s Eylea®) | Novartis | DME; Wet AMD | Intravitreal | BLA | TBD |
bevacizumab (biosimilar to Genentech’s Avastin®) | Essex | DME; Wet AMD | Intravitreal | BLA | TBD |
Phase 3 supplemental drugs
None
Specialty
Submitted new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
narsoplimab | Omeros | Transplant-associated thrombotic microangiopathy | IV, SC | BLA; Breakthrough Therapy, Orphan Drug | Jul-Sep 2024 |
dasatinib (Dasynoc®) | Xspray | CML | Oral | 505(b)(2) NDA; Orphan Drug | 07/31/2024 |
deuruxolitinib | Sun | Alopecia areata (moderate to severe) | Oral | NDA; Breakthrough Therapy, Fast Track | 08/06/2024 |
midomafetamine | Lykos | PTSD | Oral | NDA; Breakthrough Therapy, Priority Review | 08/11/2024 |
denileukin diftitox (Lymphir™ ) | Citius | Cutaneous T cell lymphoma (R/R, ≥ 2nd-line) | IV | BLA; Orphan Drug | 08/13/2024 |
nemolizumab | Galderma | Prurigo nodularis | SC | BLA; Breakthrough Therapy, Priority Review | 08/14/2024 |
palopegteriparatide | Ascendis | Hypoparathyroidism | SC | NDA; Orphan Drug | 08/14/2024 |
seladelpar | Gilead | Primary biliary cholangitis (including pruritus, ursodeoxycholic acid inadequate response/intolerance) | Oral | NDA; Breakthrough Therapy, Orphan Drug, Priority Review | 08/14/2024 |
vorasidenib | Les Laboratoires Servier | Glioma (diffuse, IDH-mutant) | Oral | NDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review | 08/20/2024 |
linvoseltamab | Regeneron | Multiple myeloma (4th-line) | IV | BLA; Fast Track, Priority Review | 08/22/2024 |
axatilimab | Syndax | Chronic GVHD (≥ 2 prior lines of systemic therapy, ages ≥ 6 years) | IV | BLA; Fast Track, Orphan Drug, Priority Review | 08/28/2024 |
ocrelizumab/hyaluronidase (Ocrevus® SC) | Genentech | MS | SC | BLA | 09/13/2024 |
atezolizumab/hyaluronidase (Tecentriq® SC) | Genentech | Alveolar soft part sarcoma; HCC; Melanoma; NSCLC; SCLC; Urothelial cancer | SC | BLA | 09/15/2024 |
arimoclomol | Zevra | Niemann-Pick disease type C | Oral | NDA; Breakthrough Therapy, Fast Track, Orphan Drug, RPD | 09/21/2024 |
N-acetyl-L-leucine | Intrabio | Niemann-Pick disease type C | Oral | NDA; Fast Track, Orphan Drug, Priority Review, RPD | 09/24/2024 |
revumenib | Syndax/Abbvie | AML (R/R KMT2A-rearranged) | Oral | NDA; Breakthrough Therapy, Orphan Drug, Priority Review, RTOR | 09/26/2024 |
garadacimab | CSL | HAE (prophylactic treatment) | SC | BLA; Fast Track, Orphan Drug | Oct-Dec 2024 |
marstacimab | Pfizer | Hemophilia A and B (without factor VIII or IX inhibitors) | SC | BLA; Fast Track, Orphan Drug | Oct-Dec 2024 |
paliperidone palmitate ER | Luye | Schizophrenia | IM | 505(b)(2) NDA | 10/09/2024 |
octreotide (Oclaiz™) | Camurus | Acromegaly | SC | NDA | 10/21/2024 |
treosulfan | Medexus | Allogeneic HSCT (preparation, in combination with fludarabine) | IV | NDA; Orphan Drug | 10/30/2024 |
docetaxel | Zhuhai Beihai | Breast cancer; Gastric cancer; NSCLC; Prostate cancer | IV | 505(b)(2) NDA | 11/01/2024 |
zolbetuximab | Astellas | Gastric cancer (unresectable or metastatic, HER2-, CLDN18.2+, 1st-line) | IV | BLA; Orphan Drug | 11/09/2024 |
inavolisib | Genentech | Breast cancer (HR+/HER2-, 1st-line, in combination with palbociclib & fulvestrant) | Oral | NDA; Breakthrough Therapy, Priority Review | 11/27/2024 |
govorestat | Applied Therapeutics | Classic galactosemia | Oral | NDA; Fast Track, Orphan Drug, Priority Review, RPD | 11/28/2024 |
acoramidis | Bridgebio/AstraZeneca | Transthyretin amyloid cardiomyopathy (ATTR-CM) | Oral | NDA; Orphan Drug | 11/29/2024 |
zanidatamab | Jazz/Beigene | Biliary tract cancer (unresectable, locally advanced or metastatic, HER2+, previously-treated) | IV | BLA; seeking Accelerated Approval, Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review | 11/29/2024 |
nemolizumab | Galderma | Atopic dermatitis (adults and adolescents, moderate to severe) | SC | BLA | 12/14/2024 |
revakinagene taroretcel | Neurotech | Macular telangiectasia type 2 | Implant | BLA; Fast Track, Orphan Drug, Priority Review | 12/17/2024 |
irinotecan liposome | CSPC | Pancreatic cancer | IV | 505(b)(2) NDA | 12/18/2024 |
olezarsen | Akcea | Familial chylomicronemia syndrome | SC | NDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review | 12/19/2024 |
datopotamab deruxtecan | Daiichi Sankyo/AstraZeneca | NSCLC (locally advanced or metastatic, nonsquamous, ≥ 2-line) | IV | BLA | 12/20/2024 |
glepaglutide | Zealand | Short bowel syndrome (dependent on parenteral support) | SC | NDA; Orphan Drug | 12/20/2024 |
nivolumab/hyaluronidase (Opdivo® SC) | Bristol-Myers Squibb | Bladder cancer; Melanoma; RCC; Solid tumors | SC | BLA | 12/27/2024 |
ustekinumab (biosimilar to Janssen’s Stelara®) | Biocon/Janssen | PSO; PsA; CD; UC | IV, SC | BLA | 12/27/2024 |
ensartinib | Xcovery | NSCLC (metastatic, ALK+) | Oral | NDA | 12/28/2024 |
crinecerfont | Neurocrine | Congenital adrenal hyperplasia | Oral | NDA; Breakthrough Therapy, Orphan Drug, Priority Review | 12/29/2024 |
cosibelimab | Checkpoint | Cutaneous squamous cell carcinoma (metastatic or locally advanced, ineligible for curative surgery or radiation) | IV | BLA | 01/02/2025 |
vanzacaftor/tezacaftor/deutivacaftor | Vertex | CF (ages ≥ 6 years) | Oral | NDA; Orphan Drug, Priority Review | 01/02/2025 |
zenocutuzumab | Merus | NSCLC (NRG1+); Pancreatic cancer (NRG1+) | IV | BLA; Breakthrough Therapy, Orphan Drug, Priority Review | 01/06/2025 |
datopotamab deruxtecan | Daiichi Sankyo/AstraZeneca | Breast cancer (HR+/HER2-, IHC 0, IHC 1+ or IHC 2+/ISH-, unresectable or metastatic) | IV | BLA | 01/29/2025 |
elamipretide | Stealth | Barth syndrome | SC | NDA; Fast Track, Orphan Drug, Priority Review, RPD | 01/29/2025 |
fitusiran | Sanofi | Hemophilia A and B (with or without inhibitors) | SC | NDA; Fast Track, Orphan Drug | 03/25/2025 |
condoliase | Ferring | Radicular leg pain associated with lumbar disc herniation | Intrathecal | BLA | 05/14/2025 |
sebetralstat | Kalvista | HAE | Oral | NDA; Fast Track, Orphan Drug | 06/18/2025 |
mirdametinib | Springworks | Neurofibromatosis type 1- associated plexiform neurofibromas (NF1-PN) | Oral | NDA; Fast Track, Orphan Drug, RPD | 07/01/2025 |
Submitted supplemental drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
iptacopan (Fabhalta®) | Novartis | IgA nephropathy (Berger’s disease) | Oral | sNDA; seeking Accelerated Approval, Priority Review | Jul-Nov 2024 |
benralizumab (Fasenra®) | AstraZeneca | Eosinophilic granulomatosis with polyangiitis | SC | sBLA; Orphan Drug | Jul-Dec 2024 |
fam-trastuzumab deruxtecan-nxki (Enhertu®) | Daiichi Sankyo/AstraZeneca | Breast cancer (HER2+, 3rd-line) | IV | sBLA; Breakthrough Therapy, Fast Track | Jul-Dec 2024 |
olaparib (Lynparza®) | AstraZeneca | Endometrial cancer (1st-line, in combination with durvalumab) | Oral | sNDA | Jul-Dec 2024 |
dostarlimab-gxly (Jemperli) | GlaxoSmithKline | Endometrial cancer (advanced or recurrent, in combination with carboplatin & paclitaxel) | IV | sBLA; Breakthrough Therapy, Priority Review | 08/23/2024 |
sodium oxybate (Lumryz™) | Avadel | Narcolepsy (pediatrics) | Oral | sNDA; Orphan Drug | 09/06/2024 |
dupilumab (Dupixent®) | Genentech | Nasal polyposis (add-on maintenance treatment for adolescents aged 12 to 17 years with inadequately controlled chronic rhinosinusitis with nasal polyposis (CRSwNP)) | SC | sBLA | 09/15/2024 |
amivantamab-vmjw (Rybrevant®) | Janssen | NSCLC (locally advanced or metastatic, EGFR exon 19 deletion or L858R substitution, disease progression on/after osimertinib) | IV | sBLA; Breakthrough Therapy | 09/20/2024 |
pembrolizumab (Keytruda®) | Merck | Mesothelioma (pleural, unresectable or metastatic malignant, 1st-line, in combination with chemotherapy) | IV | sBLA; Priority Review | 09/25/2024 |
dupilumab (Dupixent®) | Genentech | COPD | SC | sBLA; Breakthrough Therapy | 09/27/2024 |
isatuximab-irfc (Sarclisa®) | Sanofi/Abbvie | Multiple myeloma (transplant ineligible, newly diagnosed, in combination with bortezomib, lenalidomide and dexamethasone [VRd]) | IV | sBLA; Orphan Drug, Priority Review | 09/27/2024 |
bimekizumab (Bimzelx®) | UCB | AS; nr-axSpA; PsA; HS | SC | sBLA | Oct-Dec 2024 |
osimertinib (Tagrisso®) | AstraZeneca | NSCLC (unresectable, stage III EGFR-mutated, after chemoradiotherapy) | Oral | sBLA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review | Oct-Dec 2024 |
nivolumab (Opdivo®) | Bristol-Myers Squibb | NSCLC (resectable stage IIA to IIIB, neoadjuvant with chemotherapy, and adjuvant) | IV | sBLA; Breakthrough Therapy, Fast Track | 10/08/2024 |
avacincaptad pegol (Izervay™) | Astellas | Dry AMD-related geographic atrophy | Intravitreal | sNDA; Breakthrough Therapy, Fast Track | 11/19/2024 |
daratumumab/hyaluronidase-fihj (Darzalex Faspro® SC) | Janssen | Multiple myeloma (newly diagnosed, in combination with bortezomib, lenalidomide and dexamethasone for induction and consolidation, and with lenalidomide [VRd] for maintenance) | SC | sBLA | 11/28/2024 |
ribociclib (Kisqali®) | Novartis | Breast cancer (adjuvant) | Oral | sNDA; Breakthrough Therapy | December 2024 |
tislelizumab-jsgr (Tevimbra®) | Beigene | Gastric or gastroesophageal junction adenocarcinoma (locally advanced unresectable or metastatic, in combination with fluoropyrimidine- and platinum-containing chemotherapy) | IV | sBLA | December 2024 |
guselkumab (Tremfya®) | Janssen/Novartis | UC (moderate to severe) | SC | sBLA | 01/10/2025 |
lecanemab-irmb (Leqembi®) | Eisai | Alzheimer’s disease (mild, monthly maintenance dosing) | IV | sBLA; Breakthrough Therapy, Fast Track | 01/25/2025 |
mirikizumab-mrkz (Omvoh™) | Eli Lilly | CD | IV, SC | sBLA | 01/31/2025 |
cobicistat/darunavir (Prezcobix®) | Janssen | HIV-1 infection (ages ≥ 6 years of age, weighing ≥ 25 kg) | Oral | sNDA | 04/04/2025 |
guselkumab (Tremfya®) | Janssen/Novartis | CD (moderate to severe) | IV, SC | sBLA | 04/18/2025 |
ranibizumab port delivery system (Susvimo®) | Genentech | DME; Diabetic retinopathy | Intravitreal implant | sBLA | May-Jun 2025 |
upadacitinib (Rinvoq®) | Abbvie | Giant cell arteritis | Oral | sNDA | 7/11/2025 |
Phase 3 new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
abiraterone | Tavanta | Prostate cancer | Oral | NDA | TBD |
aficamten | Cytokinetics | Hypertrophic cardiomyopathy | Oral | NDA; Breakthrough Therapy, Orphan Drug | TBD |
anti-betv1 antibody (REGN-5713-5714-5715) | Regeneron | Birch allergy | SC | BLA | TBD |
anti-BK polyomavirus | Memo | BK polyomavirus infection (renal transplant recipients) | IV | BLA; Fast Track | TBD |
apraglutide | Ironwood | Short bowel syndrome | SC | NDA; Orphan Drug | TBD |
astegolimab | Genentech | COPD | IV | BLA | TBD |
ataluren | PTC | DMD | Oral | NDA; Fast Track, Orphan Drug | TBD |
atrasentan | Novartis | IgA nephropathy (Berger’s disease) | Oral | NDA | TBD |
avutometinib | Verastem | Ovarian cancer (recurrent KRAS mutant, low-grade, serous, in combination with defactinib, ≥ 1 prior systemic therapy) | Oral | NDA; seeking Accelerated Approval, Breakthrough Therapy, Orphan Drug | TBD |
bentracimab | Serb | Ticagrelor (Brilinta®) reversal | IV | BLA; Breakthrough Therapy | TBD |
cannabidiol gel | Harmony | Fragile X syndrome | Topical | NDA; Fast Track, Orphan Drug | TBD |
cetuximab sarotalocan | Rakuten | SCCHN | IV | BLA; Fast Track | TBD |
clesrovimab | Merck | RSV prevention | IM | BLA | TBD |
cobolimab | GlaxoSmithKline | NSCLC | IV | BLA | TBD |
crovalimab | Genentech | Hemolytic uremic syndrome | IV, SC | BLA | TBD |
CTX-009 | Compass | Biliary tract cancer | IV | BLA; Fast Track | TBD |
defactinib | Verastem | Ovarian cancer (KRAS mutant, low-grade, serous, ≥ 1 prior systemic therapy, in combination with avutometinib) | Oral | NDA; seeking Accelerated Approval, Breakthrough Therapy, Orphan Drug | TBD |
deoxythymidine/deoxycytidine | UCB | Thymidine kinase 2 (TK2) deficiency | Oral | BLA; Breakthrough Therapy, Orphan Drug | TBD |
depemokimab | GlaxoSmithKline | Asthma; Chronic rhinosinusitis | SC | BLA | TBD |
dersimelagon | Mitsubishi Tanabe | Porphyria | Oral | NDA; Fast Track, Orphan Drug | TBD |
dinutuximab beta | EUSA | Neuroendocrine tumors | IV | BLA; Orphan Drug | TBD |
donidalorsen | Ionis | HAE | SC | NDA; Orphan Drug | TBD |
efruxifermin | Akero | NASH | SC | BLA; Breakthrough Therapy; Fast Track | TBD |
fazirsiran | Arrowhead | Alpha-1 antitrypsin deficiency | SC | NDA; Breakthrough Therapy, Fast Track, Orphan Drug | TBD |
fenebrutinib | Genentech | MS | Oral | NDA | TBD |
fianlimab | Regeneron | Melanoma | IV | BLA; Fast Track | TBD |
garetosmab | Regeneron | Fibrodysplasia ossificans progressiva | IV | BLA; Fast Track, Orphan Drug | TBD |
giredestrant | Genentech | Breast cancer (HR+/HER2-) | Oral | NDA; Fast Track | TBD |
gold nanocrystal | Clene | ALS | Oral | NDA; Orphan Drug | TBD |
hydroxypropyl beta cyclodextrin | Cyclo | Niemann-Pick disease type C | IV | NDA; Fast Track, Orphan Drug, RPD | TBD |
ianalumab | Novartis | Autoimmne hepatitis; Sjogren’s syndrome; ITP | SC | BLA; Fast Track | TBD |
imlifidase | Sarepta | Kidney transplant rejection | IV | BLA; Fast Track, Orphan Drug | TBD |
imsidolimab | Anaptysbio | Generalized pustular psoriasis | IV, SC | BLA; Orphan Drug | TBD |
itepekimab | Regeneron | COPD | SC | BLA; Fast Track | TBD |
JDQ-443 | Novartis | NSCLC | Oral | NDA | TBD |
ketamine | Hope | Bipolar disorder (suicidal); MDD (suicidal) | IV | 505(b)(2) NDA; Fast Track | TBD |
latozinemab | Alector | Frontotemporal dementia | IV | BLA; Breakthrough Therapy, Fast Track, Orphan Drug | TBD |
lerodalcibep | LIB | Dyslipidemia/hypercholesterolemia; HeFH; HoFH | SC | BLA | TBD |
leukocyte interleukin | CEL-SCI | SCCHN | SC | BLA; Orphan Drug | TBD |
ligelizumab | Novartis | Food allergies | SC | BLA | TBD |
linerixibat | GlaxoSmithKline | Primary biliary cholangitis-related pruritus | Oral | NDA; Orphan Drug | TBD |
mepolizumab (Nucala®) | GlaxoSmithKline | COPD | SC | sBLA | TBD |
molgramostim | Savara | Pulmonary alveolar proteinosis | Inhaled | BLA; Breakthrough Therapy, Fast Track, Orphan Drug | TBD |
navepegritide | Ascendis | Achondroplasia | SC | NDA; Orphan Drug | TBD |
nipocalimab | Janssen | Myasthenia gravis | IV | BLA; Fast Track, Orphan Drug | TBD |
obicetrapib | New Amsterdam | Dyslipidemia/hypercholesterolemia | Oral | NDA | TBD |
pabinafusp alfa | JCR | Mucopolysaccharidosis II (Hunter syndrome) | IV | BLA; Orphan Drug | TBD |
paltusotine | Crinetics | Acromegaly | Oral | NDA; Orphan Drug | TBD |
pegadricase | Swedish Orphan Biovitrum | Gout | IV | BLA | TBD |
pegargiminase | Polaris | Mesothelioma | IM | BLA; Fast Track, Orphan Drug | TBD |
pegzilarginase | Immedica | Arginase 1 deficiency | IV | BLA; Breakthrough Therapy, Fast Track, Orphan Drug, RPD | TBD |
pelabresib | Novartis | Myelofibrosis | Oral | NDA; Fast Track, Orphan Drug | TBD |
pelacarsen | Novartis | CVD (elevated lipoprotein[a]) | SC | NDA; Fast Track | TBD |
plozasiran | Arrowhead | Familial chylomicronemia syndrome | SC | NDA; Fast Track, Orphan Drug | TBD |
QRX003 | Quoin | Netherton syndrome | Topical | NDA | TBD |
remibrutinib | Novartis | MS; Urticaria | Oral | NDA | TBD |
resiniferatoxin | Grunenthal | Osteoarthritis pain (knee) | Intra-articular | NDA; Breakthrough Therapy | TBD |
rilzabrutinib | Sanofi | ITP | Oral | NDA; Fast Track, Orphan Drug | TBD |
rusfertide | Takeda | Polycythemia vera | SC | NDA; Fast Track, Orphan Drug | TBD |
sefaxersen | Ionis/Roche | IgA nephropathy (Berger’s disease) | SC | NDA | TBD |
sepiapterin | PTC | Phenylketonuria (PKU) | Oral | NDA; Orphan Drug | TBD |
serplulimab | Henlius | SCLC | IV | BLA; Orphan Drug | TBD |
soticlestat | Takeda | Dravet syndrome; Lennox-Gastaut syndrome | Oral | NDA; Orphan Drug | TBD |
sozinibercept | Opthea | Wet AMD | Intravitreal | BLA; Fast Track | TBD |
sunvozertinib | Dizal (Jiangsu) | NSCLC | Oral | NDA; Breakthrough Therapy | TBD |
tamibarotene | Syros | Myelodysplastic syndrome | Oral | NDA; Fast Track, Orphan Drug | TBD |
telisotuzumab vedotin | Abbvie | NSCLC | IV | BLA; Breakthrough Therapy | TBD |
tiragolumab | Genentech | Esophageal cancer; NSCLC | IV | BLA; Breakthrough Therapy, Orphan Drug | TBD |
tolebrutinib | Sanofi | MS | Oral | NDA | TBD |
vatiquinone | PTC | Friedreich’s ataxia | Oral | NDA; Fast Track, Orphan Drug | TBD |
venglustat | Sanofi | Fabry’s disease; GM2 gangliosidosis (Tay-Sachs disease, Sandhoff disease) | Oral | NDA; Fast Track, Orphan Drug | TBD |
vimseltinib | Ono | Pigmented villonodular synovitis | Oral | NDA; Fast Track | TBD |
zanidatamab | Jazz | Gastric cancer | IV | BLA; Fast Track, Orphan Drug | TBD |
zasocitinib | Takeda | PSO | Oral | NDA | TBD |
Phase 3 supplemental drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
alpelisib (Piqray®) | Novartis | Breast cancer (HER2+) | Oral | sNDA | TBD |
brolucizumab-dbll (Beovu®) | Novartis | Diabetic retinopathy | Intravitreal | sBLA | TBD |
cabozantinib (Cabometyx®/Cometriq®) | Exelixis | Prostate cancer | Oral | sNDA | TBD |
canakinumab (Ilaris®) | Novartis | NSCLC (adjuvant) | SC | sBLA | TBD |
cemiplimab-rwlc (Libtayo®) | Regeneron | Melanoma | IV | sBLA; Fast Track | TBD |
dupilumab (Dupixent®) | Sanofi | Bullous pemphigoid | SC | sBLA; Orphan Drug | TBD |
durvalumab (Imfinzi®) | AstraZeneca | Bladder cancer (1st-line); Breast cancer (TNBC) | IV | sBLA; Breakthrough Therapy | TBD |
eplontersen (Wainua™) | Ionis | Transthyretin amyloid cardiomyopathy | SC | sNDA; Fast Track, Orphan Drug | TBD |
ganaxolone (Ztalmy®) | Marinus | Tuberous sclerosis complex | IV, Oral | sNDA; Orphan Drug | TBD |
inebilizumab-cdon (Uplizna®) | Amgen | Autoimmune disorders (IgG4-related disease) | IV | sBLA | TBD |
iptacopan (Fabhalta®) | Novartis | C3 glomerulopathy (C3G); Hemolytic uremic syndrome | Oral | sNDA; Breakthrough Therapy, Orphan Drug, RPD | TBD |
mitapivat (Pyrukynd®) | Agios | SCD; Thalassemia | Oral | sNDA; Orphan Drug | TBD |
mosunetuzumab-axgb (Lunsumio™) | Genentech | DLBCL (2nd-line, in combination with polatuzumab vedotin) | SC | sBLA | TBD |
obinutuzumab (Gazyva®) | Genentech | Lupus nephritis; Membranous nephropathy; SLE | IV | sBLA; Breakthrough Therapy | TBD |
pozelimab (Veopoz™) | Regeneron | PNH | IV, SC | sBLA; Orphan Drug | TBD |
satralizumab-mwge (Enspryng®) | Genentech | Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) | SC | sBLA; Orphan Drug | TBD |
secukinumab (Cosentyx®) | Novartis | Giant cell arteritis; Lupus nephritis | SC | sBLA | TBD |
sotorasib (Lumakras®) | Amgen | CRC | Oral | sNDA; Orphan Drug | TBD |
sparsentan (Filspari®) | Travere | Focal segmental glomerulosclerosis | Oral | sNDA; Orphan Drug | TBD |
tislelizumab- jsgr (Tevimbra®) | Beigene | HCC | IV | sBLA; Orphan Drug | TBD |
venetoclax (Venclexta®) | Abbvie/Genentech | Myelodysplastic syndrome | Oral | sNDA; Breakthrough Therapy, Orphan Drug | TBD |
vutrisiran (Amvuttra®) | Alnylam | Transthyretin amyloid cardiomyopathy | SC | sNDA; Orphan Drug | TBD |
Submitted new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
narsoplimab | Omeros | Transplant-associated thrombotic microangiopathy | IV, SC | BLA; Breakthrough Therapy, Orphan Drug | Jul-Sep 2024 |
dasatinib (Dasynoc®) | Xspray | CML | Oral | 505(b)(2) NDA; Orphan Drug | 07/31/2024 |
deuruxolitinib | Sun | Alopecia areata (moderate to severe) | Oral | NDA; Breakthrough Therapy, Fast Track | 08/06/2024 |
midomafetamine | Lykos | PTSD | Oral | NDA; Breakthrough Therapy, Priority Review | 08/11/2024 |
denileukin diftitox (Lymphir™ ) | Citius | Cutaneous T cell lymphoma (R/R, ≥ 2nd-line) | IV | BLA; Orphan Drug | 08/13/2024 |
nemolizumab | Galderma | Prurigo nodularis | SC | BLA; Breakthrough Therapy, Priority Review | 08/14/2024 |
palopegteriparatide | Ascendis | Hypoparathyroidism | SC | NDA; Orphan Drug | 08/14/2024 |
seladelpar | Gilead | Primary biliary cholangitis (including pruritus, ursodeoxycholic acid inadequate response/intolerance) | Oral | NDA; Breakthrough Therapy, Orphan Drug, Priority Review | 08/14/2024 |
vorasidenib | Les Laboratoires Servier | Glioma (diffuse, IDH-mutant) | Oral | NDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review | 08/20/2024 |
linvoseltamab | Regeneron | Multiple myeloma (4th-line) | IV | BLA; Fast Track, Priority Review | 08/22/2024 |
axatilimab | Syndax | Chronic GVHD (≥ 2 prior lines of systemic therapy, ages ≥ 6 years) | IV | BLA; Fast Track, Orphan Drug, Priority Review | 08/28/2024 |
ocrelizumab/hyaluronidase (Ocrevus® SC) | Genentech | MS | SC | BLA | 09/13/2024 |
atezolizumab/hyaluronidase (Tecentriq® SC) | Genentech | Alveolar soft part sarcoma; HCC; Melanoma; NSCLC; SCLC; Urothelial cancer | SC | BLA | 09/15/2024 |
arimoclomol | Zevra | Niemann-Pick disease type C | Oral | NDA; Breakthrough Therapy, Fast Track, Orphan Drug, RPD | 09/21/2024 |
N-acetyl-L-leucine | Intrabio | Niemann-Pick disease type C | Oral | NDA; Fast Track, Orphan Drug, Priority Review, RPD | 09/24/2024 |
revumenib | Syndax/Abbvie | AML (R/R KMT2A-rearranged) | Oral | NDA; Breakthrough Therapy, Orphan Drug, Priority Review, RTOR | 09/26/2024 |
garadacimab | CSL | HAE (prophylactic treatment) | SC | BLA; Fast Track, Orphan Drug | Oct-Dec 2024 |
marstacimab | Pfizer | Hemophilia A and B (without factor VIII or IX inhibitors) | SC | BLA; Fast Track, Orphan Drug | Oct-Dec 2024 |
paliperidone palmitate ER | Luye | Schizophrenia | IM | 505(b)(2) NDA | 10/09/2024 |
octreotide (Oclaiz™) | Camurus | Acromegaly | SC | NDA | 10/21/2024 |
treosulfan | Medexus | Allogeneic HSCT (preparation, in combination with fludarabine) | IV | NDA; Orphan Drug | 10/30/2024 |
docetaxel | Zhuhai Beihai | Breast cancer; Gastric cancer; NSCLC; Prostate cancer | IV | 505(b)(2) NDA | 11/01/2024 |
zolbetuximab | Astellas | Gastric cancer (unresectable or metastatic, HER2-, CLDN18.2+, 1st-line) | IV | BLA; Orphan Drug | 11/09/2024 |
inavolisib | Genentech | Breast cancer (HR+/HER2-, 1st-line, in combination with palbociclib & fulvestrant) | Oral | NDA; Breakthrough Therapy, Priority Review | 11/27/2024 |
govorestat | Applied Therapeutics | Classic galactosemia | Oral | NDA; Fast Track, Orphan Drug, Priority Review, RPD | 11/28/2024 |
acoramidis | Bridgebio/AstraZeneca | Transthyretin amyloid cardiomyopathy (ATTR-CM) | Oral | NDA; Orphan Drug | 11/29/2024 |
zanidatamab | Jazz/Beigene | Biliary tract cancer (unresectable, locally advanced or metastatic, HER2+, previously-treated) | IV | BLA; seeking Accelerated Approval, Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review | 11/29/2024 |
nemolizumab | Galderma | Atopic dermatitis (adults and adolescents, moderate to severe) | SC | BLA | 12/14/2024 |
revakinagene taroretcel | Neurotech | Macular telangiectasia type 2 | Implant | BLA; Fast Track, Orphan Drug, Priority Review | 12/17/2024 |
irinotecan liposome | CSPC | Pancreatic cancer | IV | 505(b)(2) NDA | 12/18/2024 |
olezarsen | Akcea | Familial chylomicronemia syndrome | SC | NDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review | 12/19/2024 |
datopotamab deruxtecan | Daiichi Sankyo/AstraZeneca | NSCLC (locally advanced or metastatic, nonsquamous, ≥ 2-line) | IV | BLA | 12/20/2024 |
glepaglutide | Zealand | Short bowel syndrome (dependent on parenteral support) | SC | NDA; Orphan Drug | 12/20/2024 |
nivolumab/hyaluronidase (Opdivo® SC) | Bristol-Myers Squibb | Bladder cancer; Melanoma; RCC; Solid tumors | SC | BLA | 12/27/2024 |
ustekinumab (biosimilar to Janssen’s Stelara®) | Biocon/Janssen | PSO; PsA; CD; UC | IV, SC | BLA | 12/27/2024 |
ensartinib | Xcovery | NSCLC (metastatic, ALK+) | Oral | NDA | 12/28/2024 |
crinecerfont | Neurocrine | Congenital adrenal hyperplasia | Oral | NDA; Breakthrough Therapy, Orphan Drug, Priority Review | 12/29/2024 |
cosibelimab | Checkpoint | Cutaneous squamous cell carcinoma (metastatic or locally advanced, ineligible for curative surgery or radiation) | IV | BLA | 01/02/2025 |
vanzacaftor/tezacaftor/deutivacaftor | Vertex | CF (ages ≥ 6 years) | Oral | NDA; Orphan Drug, Priority Review | 01/02/2025 |
zenocutuzumab | Merus | NSCLC (NRG1+); Pancreatic cancer (NRG1+) | IV | BLA; Breakthrough Therapy, Orphan Drug, Priority Review | 01/06/2025 |
datopotamab deruxtecan | Daiichi Sankyo/AstraZeneca | Breast cancer (HR+/HER2-, IHC 0, IHC 1+ or IHC 2+/ISH-, unresectable or metastatic) | IV | BLA | 01/29/2025 |
elamipretide | Stealth | Barth syndrome | SC | NDA; Fast Track, Orphan Drug, Priority Review, RPD | 01/29/2025 |
fitusiran | Sanofi | Hemophilia A and B (with or without inhibitors) | SC | NDA; Fast Track, Orphan Drug | 03/25/2025 |
condoliase | Ferring | Radicular leg pain associated with lumbar disc herniation | Intrathecal | BLA | 05/14/2025 |
sebetralstat | Kalvista | HAE | Oral | NDA; Fast Track, Orphan Drug | 06/18/2025 |
mirdametinib | Springworks | Neurofibromatosis type 1- associated plexiform neurofibromas (NF1-PN) | Oral | NDA; Fast Track, Orphan Drug, RPD | 07/01/2025 |
Submitted supplemental drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
iptacopan (Fabhalta®) | Novartis | IgA nephropathy (Berger’s disease) | Oral | sNDA; seeking Accelerated Approval, Priority Review | Jul-Nov 2024 |
benralizumab (Fasenra®) | AstraZeneca | Eosinophilic granulomatosis with polyangiitis | SC | sBLA; Orphan Drug | Jul-Dec 2024 |
fam-trastuzumab deruxtecan-nxki (Enhertu®) | Daiichi Sankyo/AstraZeneca | Breast cancer (HER2+, 3rd-line) | IV | sBLA; Breakthrough Therapy, Fast Track | Jul-Dec 2024 |
olaparib (Lynparza®) | AstraZeneca | Endometrial cancer (1st-line, in combination with durvalumab) | Oral | sNDA | Jul-Dec 2024 |
dostarlimab-gxly (Jemperli) | GlaxoSmithKline | Endometrial cancer (advanced or recurrent, in combination with carboplatin & paclitaxel) | IV | sBLA; Breakthrough Therapy, Priority Review | 08/23/2024 |
sodium oxybate (Lumryz™) | Avadel | Narcolepsy (pediatrics) | Oral | sNDA; Orphan Drug | 09/06/2024 |
dupilumab (Dupixent®) | Genentech | Nasal polyposis (add-on maintenance treatment for adolescents aged 12 to 17 years with inadequately controlled chronic rhinosinusitis with nasal polyposis (CRSwNP)) | SC | sBLA | 09/15/2024 |
amivantamab-vmjw (Rybrevant®) | Janssen | NSCLC (locally advanced or metastatic, EGFR exon 19 deletion or L858R substitution, disease progression on/after osimertinib) | IV | sBLA; Breakthrough Therapy | 09/20/2024 |
pembrolizumab (Keytruda®) | Merck | Mesothelioma (pleural, unresectable or metastatic malignant, 1st-line, in combination with chemotherapy) | IV | sBLA; Priority Review | 09/25/2024 |
dupilumab (Dupixent®) | Genentech | COPD | SC | sBLA; Breakthrough Therapy | 09/27/2024 |
isatuximab-irfc (Sarclisa®) | Sanofi/Abbvie | Multiple myeloma (transplant ineligible, newly diagnosed, in combination with bortezomib, lenalidomide and dexamethasone [VRd]) | IV | sBLA; Orphan Drug, Priority Review | 09/27/2024 |
bimekizumab (Bimzelx®) | UCB | AS; nr-axSpA; PsA; HS | SC | sBLA | Oct-Dec 2024 |
osimertinib (Tagrisso®) | AstraZeneca | NSCLC (unresectable, stage III EGFR-mutated, after chemoradiotherapy) | Oral | sBLA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review | Oct-Dec 2024 |
nivolumab (Opdivo®) | Bristol-Myers Squibb | NSCLC (resectable stage IIA to IIIB, neoadjuvant with chemotherapy, and adjuvant) | IV | sBLA; Breakthrough Therapy, Fast Track | 10/08/2024 |
avacincaptad pegol (Izervay™) | Astellas | Dry AMD-related geographic atrophy | Intravitreal | sNDA; Breakthrough Therapy, Fast Track | 11/19/2024 |
daratumumab/hyaluronidase-fihj (Darzalex Faspro® SC) | Janssen | Multiple myeloma (newly diagnosed, in combination with bortezomib, lenalidomide and dexamethasone for induction and consolidation, and with lenalidomide [VRd] for maintenance) | SC | sBLA | 11/28/2024 |
ribociclib (Kisqali®) | Novartis | Breast cancer (adjuvant) | Oral | sNDA; Breakthrough Therapy | December 2024 |
tislelizumab-jsgr (Tevimbra®) | Beigene | Gastric or gastroesophageal junction adenocarcinoma (locally advanced unresectable or metastatic, in combination with fluoropyrimidine- and platinum-containing chemotherapy) | IV | sBLA | December 2024 |
guselkumab (Tremfya®) | Janssen/Novartis | UC (moderate to severe) | SC | sBLA | 01/10/2025 |
lecanemab-irmb (Leqembi®) | Eisai | Alzheimer’s disease (mild, monthly maintenance dosing) | IV | sBLA; Breakthrough Therapy, Fast Track | 01/25/2025 |
mirikizumab-mrkz (Omvoh™) | Eli Lilly | CD | IV, SC | sBLA | 01/31/2025 |
cobicistat/darunavir (Prezcobix®) | Janssen | HIV-1 infection (ages ≥ 6 years of age, weighing ≥ 25 kg) | Oral | sNDA | 04/04/2025 |
guselkumab (Tremfya®) | Janssen/Novartis | CD (moderate to severe) | IV, SC | sBLA | 04/18/2025 |
ranibizumab port delivery system (Susvimo®) | Genentech | DME; Diabetic retinopathy | Intravitreal implant | sBLA | May-Jun 2025 |
upadacitinib (Rinvoq®) | Abbvie | Giant cell arteritis | Oral | sNDA | 7/11/2025 |
Phase 3 new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
abiraterone | Tavanta | Prostate cancer | Oral | NDA | TBD |
aficamten | Cytokinetics | Hypertrophic cardiomyopathy | Oral | NDA; Breakthrough Therapy, Orphan Drug | TBD |
anti-betv1 antibody (REGN-5713-5714-5715) | Regeneron | Birch allergy | SC | BLA | TBD |
anti-BK polyomavirus | Memo | BK polyomavirus infection (renal transplant recipients) | IV | BLA; Fast Track | TBD |
apraglutide | Ironwood | Short bowel syndrome | SC | NDA; Orphan Drug | TBD |
astegolimab | Genentech | COPD | IV | BLA | TBD |
ataluren | PTC | DMD | Oral | NDA; Fast Track, Orphan Drug | TBD |
atrasentan | Novartis | IgA nephropathy (Berger’s disease) | Oral | NDA | TBD |
avutometinib | Verastem | Ovarian cancer (recurrent KRAS mutant, low-grade, serous, in combination with defactinib, ≥ 1 prior systemic therapy) | Oral | NDA; seeking Accelerated Approval, Breakthrough Therapy, Orphan Drug | TBD |
bentracimab | Serb | Ticagrelor (Brilinta®) reversal | IV | BLA; Breakthrough Therapy | TBD |
cannabidiol gel | Harmony | Fragile X syndrome | Topical | NDA; Fast Track, Orphan Drug | TBD |
cetuximab sarotalocan | Rakuten | SCCHN | IV | BLA; Fast Track | TBD |
clesrovimab | Merck | RSV prevention | IM | BLA | TBD |
cobolimab | GlaxoSmithKline | NSCLC | IV | BLA | TBD |
crovalimab | Genentech | Hemolytic uremic syndrome | IV, SC | BLA | TBD |
CTX-009 | Compass | Biliary tract cancer | IV | BLA; Fast Track | TBD |
defactinib | Verastem | Ovarian cancer (KRAS mutant, low-grade, serous, ≥ 1 prior systemic therapy, in combination with avutometinib) | Oral | NDA; seeking Accelerated Approval, Breakthrough Therapy, Orphan Drug | TBD |
deoxythymidine/deoxycytidine | UCB | Thymidine kinase 2 (TK2) deficiency | Oral | BLA; Breakthrough Therapy, Orphan Drug | TBD |
depemokimab | GlaxoSmithKline | Asthma; Chronic rhinosinusitis | SC | BLA | TBD |
dersimelagon | Mitsubishi Tanabe | Porphyria | Oral | NDA; Fast Track, Orphan Drug | TBD |
dinutuximab beta | EUSA | Neuroendocrine tumors | IV | BLA; Orphan Drug | TBD |
donidalorsen | Ionis | HAE | SC | NDA; Orphan Drug | TBD |
efruxifermin | Akero | NASH | SC | BLA; Breakthrough Therapy; Fast Track | TBD |
fazirsiran | Arrowhead | Alpha-1 antitrypsin deficiency | SC | NDA; Breakthrough Therapy, Fast Track, Orphan Drug | TBD |
fenebrutinib | Genentech | MS | Oral | NDA | TBD |
fianlimab | Regeneron | Melanoma | IV | BLA; Fast Track | TBD |
garetosmab | Regeneron | Fibrodysplasia ossificans progressiva | IV | BLA; Fast Track, Orphan Drug | TBD |
giredestrant | Genentech | Breast cancer (HR+/HER2-) | Oral | NDA; Fast Track | TBD |
gold nanocrystal | Clene | ALS | Oral | NDA; Orphan Drug | TBD |
hydroxypropyl beta cyclodextrin | Cyclo | Niemann-Pick disease type C | IV | NDA; Fast Track, Orphan Drug, RPD | TBD |
ianalumab | Novartis | Autoimmne hepatitis; Sjogren’s syndrome; ITP | SC | BLA; Fast Track | TBD |
imlifidase | Sarepta | Kidney transplant rejection | IV | BLA; Fast Track, Orphan Drug | TBD |
imsidolimab | Anaptysbio | Generalized pustular psoriasis | IV, SC | BLA; Orphan Drug | TBD |
itepekimab | Regeneron | COPD | SC | BLA; Fast Track | TBD |
JDQ-443 | Novartis | NSCLC | Oral | NDA | TBD |
ketamine | Hope | Bipolar disorder (suicidal); MDD (suicidal) | IV | 505(b)(2) NDA; Fast Track | TBD |
latozinemab | Alector | Frontotemporal dementia | IV | BLA; Breakthrough Therapy, Fast Track, Orphan Drug | TBD |
lerodalcibep | LIB | Dyslipidemia/hypercholesterolemia; HeFH; HoFH | SC | BLA | TBD |
leukocyte interleukin | CEL-SCI | SCCHN | SC | BLA; Orphan Drug | TBD |
ligelizumab | Novartis | Food allergies | SC | BLA | TBD |
linerixibat | GlaxoSmithKline | Primary biliary cholangitis-related pruritus | Oral | NDA; Orphan Drug | TBD |
mepolizumab (Nucala®) | GlaxoSmithKline | COPD | SC | sBLA | TBD |
molgramostim | Savara | Pulmonary alveolar proteinosis | Inhaled | BLA; Breakthrough Therapy, Fast Track, Orphan Drug | TBD |
navepegritide | Ascendis | Achondroplasia | SC | NDA; Orphan Drug | TBD |
nipocalimab | Janssen | Myasthenia gravis | IV | BLA; Fast Track, Orphan Drug | TBD |
obicetrapib | New Amsterdam | Dyslipidemia/hypercholesterolemia | Oral | NDA | TBD |
pabinafusp alfa | JCR | Mucopolysaccharidosis II (Hunter syndrome) | IV | BLA; Orphan Drug | TBD |
paltusotine | Crinetics | Acromegaly | Oral | NDA; Orphan Drug | TBD |
pegadricase | Swedish Orphan Biovitrum | Gout | IV | BLA | TBD |
pegargiminase | Polaris | Mesothelioma | IM | BLA; Fast Track, Orphan Drug | TBD |
pegzilarginase | Immedica | Arginase 1 deficiency | IV | BLA; Breakthrough Therapy, Fast Track, Orphan Drug, RPD | TBD |
pelabresib | Novartis | Myelofibrosis | Oral | NDA; Fast Track, Orphan Drug | TBD |
pelacarsen | Novartis | CVD (elevated lipoprotein[a]) | SC | NDA; Fast Track | TBD |
plozasiran | Arrowhead | Familial chylomicronemia syndrome | SC | NDA; Fast Track, Orphan Drug | TBD |
QRX003 | Quoin | Netherton syndrome | Topical | NDA | TBD |
remibrutinib | Novartis | MS; Urticaria | Oral | NDA | TBD |
resiniferatoxin | Grunenthal | Osteoarthritis pain (knee) | Intra-articular | NDA; Breakthrough Therapy | TBD |
rilzabrutinib | Sanofi | ITP | Oral | NDA; Fast Track, Orphan Drug | TBD |
rusfertide | Takeda | Polycythemia vera | SC | NDA; Fast Track, Orphan Drug | TBD |
sefaxersen | Ionis/Roche | IgA nephropathy (Berger’s disease) | SC | NDA | TBD |
sepiapterin | PTC | Phenylketonuria (PKU) | Oral | NDA; Orphan Drug | TBD |
serplulimab | Henlius | SCLC | IV | BLA; Orphan Drug | TBD |
soticlestat | Takeda | Dravet syndrome; Lennox-Gastaut syndrome | Oral | NDA; Orphan Drug | TBD |
sozinibercept | Opthea | Wet AMD | Intravitreal | BLA; Fast Track | TBD |
sunvozertinib | Dizal (Jiangsu) | NSCLC | Oral | NDA; Breakthrough Therapy | TBD |
tamibarotene | Syros | Myelodysplastic syndrome | Oral | NDA; Fast Track, Orphan Drug | TBD |
telisotuzumab vedotin | Abbvie | NSCLC | IV | BLA; Breakthrough Therapy | TBD |
tiragolumab | Genentech | Esophageal cancer; NSCLC | IV | BLA; Breakthrough Therapy, Orphan Drug | TBD |
tolebrutinib | Sanofi | MS | Oral | NDA | TBD |
vatiquinone | PTC | Friedreich’s ataxia | Oral | NDA; Fast Track, Orphan Drug | TBD |
venglustat | Sanofi | Fabry’s disease; GM2 gangliosidosis (Tay-Sachs disease, Sandhoff disease) | Oral | NDA; Fast Track, Orphan Drug | TBD |
vimseltinib | Ono | Pigmented villonodular synovitis | Oral | NDA; Fast Track | TBD |
zanidatamab | Jazz | Gastric cancer | IV | BLA; Fast Track, Orphan Drug | TBD |
zasocitinib | Takeda | PSO | Oral | NDA | TBD |
Phase 3 supplemental drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
alpelisib (Piqray®) | Novartis | Breast cancer (HER2+) | Oral | sNDA | TBD |
brolucizumab-dbll (Beovu®) | Novartis | Diabetic retinopathy | Intravitreal | sBLA | TBD |
cabozantinib (Cabometyx®/Cometriq®) | Exelixis | Prostate cancer | Oral | sNDA | TBD |
canakinumab (Ilaris®) | Novartis | NSCLC (adjuvant) | SC | sBLA | TBD |
cemiplimab-rwlc (Libtayo®) | Regeneron | Melanoma | IV | sBLA; Fast Track | TBD |
dupilumab (Dupixent®) | Sanofi | Bullous pemphigoid | SC | sBLA; Orphan Drug | TBD |
durvalumab (Imfinzi®) | AstraZeneca | Bladder cancer (1st-line); Breast cancer (TNBC) | IV | sBLA; Breakthrough Therapy | TBD |
eplontersen (Wainua™) | Ionis | Transthyretin amyloid cardiomyopathy | SC | sNDA; Fast Track, Orphan Drug | TBD |
ganaxolone (Ztalmy®) | Marinus | Tuberous sclerosis complex | IV, Oral | sNDA; Orphan Drug | TBD |
inebilizumab-cdon (Uplizna®) | Amgen | Autoimmune disorders (IgG4-related disease) | IV | sBLA | TBD |
iptacopan (Fabhalta®) | Novartis | C3 glomerulopathy (C3G); Hemolytic uremic syndrome | Oral | sNDA; Breakthrough Therapy, Orphan Drug, RPD | TBD |
mitapivat (Pyrukynd®) | Agios | SCD; Thalassemia | Oral | sNDA; Orphan Drug | TBD |
mosunetuzumab-axgb (Lunsumio™) | Genentech | DLBCL (2nd-line, in combination with polatuzumab vedotin) | SC | sBLA | TBD |
obinutuzumab (Gazyva®) | Genentech | Lupus nephritis; Membranous nephropathy; SLE | IV | sBLA; Breakthrough Therapy | TBD |
pozelimab (Veopoz™) | Regeneron | PNH | IV, SC | sBLA; Orphan Drug | TBD |
satralizumab-mwge (Enspryng®) | Genentech | Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) | SC | sBLA; Orphan Drug | TBD |
secukinumab (Cosentyx®) | Novartis | Giant cell arteritis; Lupus nephritis | SC | sBLA | TBD |
sotorasib (Lumakras®) | Amgen | CRC | Oral | sNDA; Orphan Drug | TBD |
sparsentan (Filspari®) | Travere | Focal segmental glomerulosclerosis | Oral | sNDA; Orphan Drug | TBD |
tislelizumab- jsgr (Tevimbra®) | Beigene | HCC | IV | sBLA; Orphan Drug | TBD |
venetoclax (Venclexta®) | Abbvie/Genentech | Myelodysplastic syndrome | Oral | sNDA; Breakthrough Therapy, Orphan Drug | TBD |
vutrisiran (Amvuttra®) | Alnylam | Transthyretin amyloid cardiomyopathy | SC | sNDA; Orphan Drug | TBD |
Traditional
Submitted new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
galantamine | Alpha Cognition | Alzheimer’s disease (mild to moderate) | Oral | 505(b)(2) NDA | 07/27/2024 |
carbidopa/levodopa ER | Amneal | Parkinson’s disease | Oral | 505(b)(2) NDA | 08/08/2024 |
tradipitant | Vanda/Eli Lilly | Gastroparesis | Oral | NDA | 09/18/2024 |
xanomeline/trospium (KarXT) | Bristol-Myers Squibb | Schizophrenia | Oral | NDA | 09/26/2024 |
epinephrine | ARS | Anaphylaxis | Intranasal | 505(b)(2) NDA; Fast Track | 10/02/2024 |
sulopenem etzadroxil/probenecid | Iterum | UTI (uncomplicated, adult women) | Oral | NDA; Fast Track, QIDP | 10/25/2024 |
minocycline HCl 40 mg | Journey | Rosacea | Oral | 505(b)(2) NDA | 11/04/2024 |
vicagrel | Jiangsu Vcare | Thrombotic cardiovascular and cerebrovascular diseases | Oral | NDA | 12/28/2024 |
chikungunya vaccine | Bavarian | Chikungunya virus immunization (ages ≥ 12 years) | IM | BLA; Breakthrough Therapy, Fast Track | Jan-Jun 2025 |
lamotrigine oral suspension | OWP | Bipolar disorder; Seizure disorder | Oral | 505(b)(2) NDA | 01/03/2025 |
diazoxide choline | Soleno | Prader-Willi syndrome (ages ≥ 4 years with hyperphagia) | Oral | 505(b)(2) NDA; Breakthrough Therapy, Fast Track, Orphan Drug | Feb-Jun 2025 |
meningococcal vaccine (GSK3536819A) | GlaxoSmithKline | Meningococcal immunization | IM | BLA | 02/14/2025 |
hydrocortisone oral solution (ET-400) | Eton | Adrenocortical insufficiency | Oral | NDA | 02/28/2025 |
etripamil nasal spray | Milestone | Paroxysmal supraventricular tachycardia | Intranasal | NDA | 03/28/2025 |
mRNA Coronavirus vaccine (Spikevax) | Moderna | COVID-19 immunization (SARS-CoV-2 variant JN.1) | IM | BLA; Fast Track | 04/07/2025 |
chenodiol | Mirum | Cerebrotendinous xanthomatosis | Oral | NDA; Orphan Drug | 06/28/2025 |
Submitted supplemental drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
semaglutide (Wegovy®) | Novo Nordisk | Chronic HFpEF | SC | sNDA; Priority Review | July 2024 |
tislelizumab-jsgr (Tevimbra®) | Beigene | Esophageal squamous cell carcinoma (unresectable, recurrent, locally advanced, or metastatic, 1st-line) | IV | sBLA; Orphan Drug | July 2024 |
ACAM2000 | Emergent | Mpox immunization | IM | sBLA | 09/06/2024 |
vibegron (Gemtesa®) | Sumitovant | Overactive bladder (in men receiving BPH pharmacotherapy) | Oral | sNDA | Oct-Dec 2024 |
dasiglucagon (Zegalogue®) | Novo Nordisk | Congenital hyperinsulinism (CHI) | SC | sNDA; Orphan Drug, RPD | 10/08/2024 |
anacaulase-bcdb (Nexobrid®) | Vericel | Burn injury (pediatrics) | Topical | sBLA; Orphan Drug | 11/08/2024 |
tirzepatide (Zepbound®) | Eli Lilly | Sleep apnea with obesity | SC | sNDA; Fast Track | December 2024 |
tapinarof (Vtama®) | Dermavant | Atopic dermatitis (ages ≥ 2 years) | Topical | sNDA | 12/13/2024 |
sotagliflozin (Inpefa®) | Lexicon | T1DM (with CKD) | Oral | sNDA | 12/20/2024 |
semaglutide (Ozempic®) | Novo Nordisk | Diabetic nephropathy | SC | sNDA | January 2025 |
smallpox and monkeypox vaccine (Jynneos®) – freeze-dried formulation | Bavarian | Mpox immunization; Smallpox immunization | SC | sBLA | Jan-Mar 2025 |
brexpiprazole (Rexulti®) | Otsuka | PTSD (in combination with sertraline) | Oral | sNDA | 02/08/2025 |
Phase 3 new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
aceclidine | Lenz | Presbyopia | Ophthalmic | NDA | TBD |
AR-15512 | Aerie | DED | Ophthalmic | NDA | TBD |
aroxybutynin/atomoxetine | Apnimed | Sleep apnea | Oral | NDA; Fast Track | TBD |
azetukalner | Xenon | Partial/focal seizures | Oral | NDA | TBD |
baclofen/naltrexone/sorbitol | Pharnext | Charcot-Marie-Tooth disease | Oral | NDA; Fast Track, Orphan Drug | TBD |
bemnifosbuvir | Atea | COVID-19 | Oral | NDA; Fast Track | TBD |
blarcamesine | Anavex | Alzheimer’s disease | Oral | NDA | TBD |
brensocatib | Insmed | Bronchiectasis | Oral | NDA; Breakthrough Therapy | TBD |
brilaroxazine | Reviva | Schizophrenia | Oral | NDA | TBD |
cadisegliatin | vTv | T1DM | Oral | NDA; Breakthrough Therapy | TBD |
cagrilintide/semaglutide | Novo Nordisk | Obesity/overweight | SC | NDA | TBD |
camlipixant | GlaxoSmithKline | Chronic cough | Oral | NDA | TBD |
carbachol/brimonidine | Visus | Presbyopia | Ophthalmic | 505(b)(2) NDA | TBD |
COVID-19 vaccine (SP0253) | Sanofi | COVID-19 | IM | BLA | TBD |
cytisinicline | Achieve Life Sciences | Smoking cessation | Oral | NDA | TBD |
dengue tetravalent vaccine, live, attenuated | Takeda | Dengue fever (ages 4-60 years) | SC | BLA; Fast Track | TBD |
elinzanetant | Bayer | Menopausal vasomotor symptoms | Oral | NDA | TBD |
ensitrelvir fumaric acid | Shionogi | COVID-19 | Oral | BLA; Fast Track | TBD |
epinephrine (sublingual) | Aquestive | Anaphylaxis | SL | 505(b)(2) NDA; Fast Track | TBD |
esreboxetine | Axsome | Fibromyalgia | Oral | NDA | TBD |
gepotidacin | GlaxoSmithKline | UTI (uncomplicated); Urogenital gonorrhea | Oral | NDA; QIDP | TBD |
navacaprant | Neumora | MDD | Oral | NDA | TBD |
orforglipron | Eli Lilly | Obesity/overweight; T2DM | Oral | NDA | TBD |
piclidenoson | Can-Fite | PSO | Oral | NDA | TBD |
PL-9643 | Palatin | DED | Ophthalmic | NDA | TBD |
purified vero rabies vaccine (SP0087) | Sanofi | Rabies (post-exposure treatment) | IM | BLA | TBD |
quadrivalent influenza mRNA vaccine (mRNA-1010) | Moderna | Seasonal influenza immunization | IM | BLA | TBD |
ralinepag | United Therapeutics | PAH | Oral | NDA; Orphan Drug | TBD |
relacorilant | Corcept | Cushing’s syndrome | Oral | NDA; Orphan Drug | TBD |
retatrutide | Eli Lilly | Obesity/overweight; T2DM | SC | NDA | TBD |
RSV live attenuated vaccine | Sanofi | RSV immunization | Intranasal | BLA | TBD |
survodutide | Boehringer Ingelheim | Obesity/overweight; T2DM | SC | NDA | TBD |
suzetrigine | Vertex | Acute pain (moderate to severe) | Oral | NDA; Breakthrough Therapy, Fast Track | TBD |
tebipenem pivoxil | GlaxoSmithKline | UTI (complicated) | Oral | NDA; Fast Track, QIDP | TBD |
tiratricol | Rare Thyroid Therapeutics | Resistance to thyroid hormone type beta (RTH-b) | Oral | NDA; Fast Track, Orphan Drug, RPD | TBD |
tradipitant | Vanda | Emesis | Oral | NDA | TBD |
tramiprosate | Alzheon | Alzheimer’s disease | Oral | NDA; Fast Track | TBD |
ulixacaltamide | Praxis | Essential tremor | Oral | NDA | TBD |
ulotaront | Sumitomo | Schizophrenia | Oral | NDA; Breakthrough Therapy | TBD |
VHX-896 | Vanda | Bipolar disorder; Schizophrenia | Oral | NDA | TBD |
visomitin | Mitotech | DED | Ophthalmic | NDA | TBD |
zoliflodacin | Innoviva | Gonorrhea (uncomplicated) | Oral | NDA; Fast Track, QIDP | TBD |
Phase 3 supplemental drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
dexmedetomidine (IgalmiT™) | Bioxcel | Alzheimer’s disease-related neuropsychiatric symptoms | Oral | sNDA; Breakthrough Therapy | TBD |
ibrexafungerp (Brexafemme®) | GSK plc | Fungal infections (systemic, hospital indications) | Oral | sNDA; Fast Track, Orphan Drug, QIDP | TBD |
lumateperone (Caplyta®) | Intra-Cellular Therapies | MDD | Oral | sNDA | TBD |
phentolamine 0.75% (Ryzumvi™) | Viatris | Presbyopia | Ophthalmic | sNDA | TBD |
roflumilast (Zoryve®) | Arcutis | PSO | Topical | sNDA | TBD |
semaglutide (Rybelsus®) | Novo Nordisk | Obesity | Oral | sNDA | TBD |
Submitted new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
galantamine | Alpha Cognition | Alzheimer’s disease (mild to moderate) | Oral | 505(b)(2) NDA | 07/27/2024 |
carbidopa/levodopa ER | Amneal | Parkinson’s disease | Oral | 505(b)(2) NDA | 08/08/2024 |
tradipitant | Vanda/Eli Lilly | Gastroparesis | Oral | NDA | 09/18/2024 |
xanomeline/trospium (KarXT) | Bristol-Myers Squibb | Schizophrenia | Oral | NDA | 09/26/2024 |
epinephrine | ARS | Anaphylaxis | Intranasal | 505(b)(2) NDA; Fast Track | 10/02/2024 |
sulopenem etzadroxil/probenecid | Iterum | UTI (uncomplicated, adult women) | Oral | NDA; Fast Track, QIDP | 10/25/2024 |
minocycline HCl 40 mg | Journey | Rosacea | Oral | 505(b)(2) NDA | 11/04/2024 |
vicagrel | Jiangsu Vcare | Thrombotic cardiovascular and cerebrovascular diseases | Oral | NDA | 12/28/2024 |
chikungunya vaccine | Bavarian | Chikungunya virus immunization (ages ≥ 12 years) | IM | BLA; Breakthrough Therapy, Fast Track | Jan-Jun 2025 |
lamotrigine oral suspension | OWP | Bipolar disorder; Seizure disorder | Oral | 505(b)(2) NDA | 01/03/2025 |
diazoxide choline | Soleno | Prader-Willi syndrome (ages ≥ 4 years with hyperphagia) | Oral | 505(b)(2) NDA; Breakthrough Therapy, Fast Track, Orphan Drug | Feb-Jun 2025 |
meningococcal vaccine (GSK3536819A) | GlaxoSmithKline | Meningococcal immunization | IM | BLA | 02/14/2025 |
hydrocortisone oral solution (ET-400) | Eton | Adrenocortical insufficiency | Oral | NDA | 02/28/2025 |
etripamil nasal spray | Milestone | Paroxysmal supraventricular tachycardia | Intranasal | NDA | 03/28/2025 |
mRNA Coronavirus vaccine (Spikevax) | Moderna | COVID-19 immunization (SARS-CoV-2 variant JN.1) | IM | BLA; Fast Track | 04/07/2025 |
chenodiol | Mirum | Cerebrotendinous xanthomatosis | Oral | NDA; Orphan Drug | 06/28/2025 |
Submitted supplemental drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
semaglutide (Wegovy®) | Novo Nordisk | Chronic HFpEF | SC | sNDA; Priority Review | July 2024 |
tislelizumab-jsgr (Tevimbra®) | Beigene | Esophageal squamous cell carcinoma (unresectable, recurrent, locally advanced, or metastatic, 1st-line) | IV | sBLA; Orphan Drug | July 2024 |
ACAM2000 | Emergent | Mpox immunization | IM | sBLA | 09/06/2024 |
vibegron (Gemtesa®) | Sumitovant | Overactive bladder (in men receiving BPH pharmacotherapy) | Oral | sNDA | Oct-Dec 2024 |
dasiglucagon (Zegalogue®) | Novo Nordisk | Congenital hyperinsulinism (CHI) | SC | sNDA; Orphan Drug, RPD | 10/08/2024 |
anacaulase-bcdb (Nexobrid®) | Vericel | Burn injury (pediatrics) | Topical | sBLA; Orphan Drug | 11/08/2024 |
tirzepatide (Zepbound®) | Eli Lilly | Sleep apnea with obesity | SC | sNDA; Fast Track | December 2024 |
tapinarof (Vtama®) | Dermavant | Atopic dermatitis (ages ≥ 2 years) | Topical | sNDA | 12/13/2024 |
sotagliflozin (Inpefa®) | Lexicon | T1DM (with CKD) | Oral | sNDA | 12/20/2024 |
semaglutide (Ozempic®) | Novo Nordisk | Diabetic nephropathy | SC | sNDA | January 2025 |
smallpox and monkeypox vaccine (Jynneos®) – freeze-dried formulation | Bavarian | Mpox immunization; Smallpox immunization | SC | sBLA | Jan-Mar 2025 |
brexpiprazole (Rexulti®) | Otsuka | PTSD (in combination with sertraline) | Oral | sNDA | 02/08/2025 |
Phase 3 new drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
aceclidine | Lenz | Presbyopia | Ophthalmic | NDA | TBD |
AR-15512 | Aerie | DED | Ophthalmic | NDA | TBD |
aroxybutynin/atomoxetine | Apnimed | Sleep apnea | Oral | NDA; Fast Track | TBD |
azetukalner | Xenon | Partial/focal seizures | Oral | NDA | TBD |
baclofen/naltrexone/sorbitol | Pharnext | Charcot-Marie-Tooth disease | Oral | NDA; Fast Track, Orphan Drug | TBD |
bemnifosbuvir | Atea | COVID-19 | Oral | NDA; Fast Track | TBD |
blarcamesine | Anavex | Alzheimer’s disease | Oral | NDA | TBD |
brensocatib | Insmed | Bronchiectasis | Oral | NDA; Breakthrough Therapy | TBD |
brilaroxazine | Reviva | Schizophrenia | Oral | NDA | TBD |
cadisegliatin | vTv | T1DM | Oral | NDA; Breakthrough Therapy | TBD |
cagrilintide/semaglutide | Novo Nordisk | Obesity/overweight | SC | NDA | TBD |
camlipixant | GlaxoSmithKline | Chronic cough | Oral | NDA | TBD |
carbachol/brimonidine | Visus | Presbyopia | Ophthalmic | 505(b)(2) NDA | TBD |
COVID-19 vaccine (SP0253) | Sanofi | COVID-19 | IM | BLA | TBD |
cytisinicline | Achieve Life Sciences | Smoking cessation | Oral | NDA | TBD |
dengue tetravalent vaccine, live, attenuated | Takeda | Dengue fever (ages 4-60 years) | SC | BLA; Fast Track | TBD |
elinzanetant | Bayer | Menopausal vasomotor symptoms | Oral | NDA | TBD |
ensitrelvir fumaric acid | Shionogi | COVID-19 | Oral | BLA; Fast Track | TBD |
epinephrine (sublingual) | Aquestive | Anaphylaxis | SL | 505(b)(2) NDA; Fast Track | TBD |
esreboxetine | Axsome | Fibromyalgia | Oral | NDA | TBD |
gepotidacin | GlaxoSmithKline | UTI (uncomplicated); Urogenital gonorrhea | Oral | NDA; QIDP | TBD |
navacaprant | Neumora | MDD | Oral | NDA | TBD |
orforglipron | Eli Lilly | Obesity/overweight; T2DM | Oral | NDA | TBD |
piclidenoson | Can-Fite | PSO | Oral | NDA | TBD |
PL-9643 | Palatin | DED | Ophthalmic | NDA | TBD |
purified vero rabies vaccine (SP0087) | Sanofi | Rabies (post-exposure treatment) | IM | BLA | TBD |
quadrivalent influenza mRNA vaccine (mRNA-1010) | Moderna | Seasonal influenza immunization | IM | BLA | TBD |
ralinepag | United Therapeutics | PAH | Oral | NDA; Orphan Drug | TBD |
relacorilant | Corcept | Cushing’s syndrome | Oral | NDA; Orphan Drug | TBD |
retatrutide | Eli Lilly | Obesity/overweight; T2DM | SC | NDA | TBD |
RSV live attenuated vaccine | Sanofi | RSV immunization | Intranasal | BLA | TBD |
survodutide | Boehringer Ingelheim | Obesity/overweight; T2DM | SC | NDA | TBD |
suzetrigine | Vertex | Acute pain (moderate to severe) | Oral | NDA; Breakthrough Therapy, Fast Track | TBD |
tebipenem pivoxil | GlaxoSmithKline | UTI (complicated) | Oral | NDA; Fast Track, QIDP | TBD |
tiratricol | Rare Thyroid Therapeutics | Resistance to thyroid hormone type beta (RTH-b) | Oral | NDA; Fast Track, Orphan Drug, RPD | TBD |
tradipitant | Vanda | Emesis | Oral | NDA | TBD |
tramiprosate | Alzheon | Alzheimer’s disease | Oral | NDA; Fast Track | TBD |
ulixacaltamide | Praxis | Essential tremor | Oral | NDA | TBD |
ulotaront | Sumitomo | Schizophrenia | Oral | NDA; Breakthrough Therapy | TBD |
VHX-896 | Vanda | Bipolar disorder; Schizophrenia | Oral | NDA | TBD |
visomitin | Mitotech | DED | Ophthalmic | NDA | TBD |
zoliflodacin | Innoviva | Gonorrhea (uncomplicated) | Oral | NDA; Fast Track, QIDP | TBD |
Phase 3 supplemental drugs
Name | Manufacturer | Clinical Use | Dosage Form | Development Status | FDA Decision |
dexmedetomidine (IgalmiT™) | Bioxcel | Alzheimer’s disease-related neuropsychiatric symptoms | Oral | sNDA; Breakthrough Therapy | TBD |
ibrexafungerp (Brexafemme®) | GSK plc | Fungal infections (systemic, hospital indications) | Oral | sNDA; Fast Track, Orphan Drug, QIDP | TBD |
lumateperone (Caplyta®) | Intra-Cellular Therapies | MDD | Oral | sNDA | TBD |
phentolamine 0.75% (Ryzumvi™) | Viatris | Presbyopia | Ophthalmic | sNDA | TBD |
roflumilast (Zoryve®) | Arcutis | PSO | Topical | sNDA | TBD |
semaglutide (Rybelsus®) | Novo Nordisk | Obesity | Oral | sNDA | TBD |
Complete response letter
Name | Manufacturer | Clinical Use | Dosage Form | Development Status |
camrelizumab | Jiangsu Hengrui | HCC (unresectable, 1st-line, in combination with rivoceranib) | IV | CRL |
foscarbidopa/foslevodopa | Abbvie | Parkinson’s disease | SC infusion | CRL |
hepatitis B vaccine (recombinant), adjuvanted (Heplisav-B®) | Dynavax | Hepatitis B prevention | IM | CRL |
insulin icodec (Awiqli) | Novo Nordisk | T1DM; T2DM | SC | CRL |
marnetegragene autotemcel (Kresladi™) | Rocket | Leukocyte adhesion deficiency-I | IV | CRL |
naloxone (high-dose) (OX124) | Orexo | Opioid overdose | Intranasal | CRL |
patritumab deruxtecan | Merck | NSCLC (locally advanced or metastatic, EGFR-mutated, ≥ 2 prior systemic therapies) | IV | CRL |
prademagene zamikeracel | Abeona | Epidermolysis bullosa (recessive dystrophic) | Topical | CRL |
ranibizumab (biosimilar to Genentech’s Lucentis®) | STADA Arzneimittel | Diabetic retinopathy; DME; Myopic choroidal neovascularization; Macular edema following RVO; Wet AMD | Intravitreal | CRL |
rituximab (biosimilar to Genentech’s Rituxan®) | Dr. Reddy’s | CLL; Granulomatosis with polyangiitis/microscopic polyangiitis; NHL; Mature B cell NHL/mature B cell acute leukemia; Pemphigus vulgaris; RA | IV | CRL |
rivoceranib | Elevar | HCC (unresectable, 1st-line, in combination with camrelizumab) | Oral | CRL |
Glossary of terms
Glossary of terms
5-FU 5-Fluorouracil
6MWD 6 Minute Walking Distance
6MWT 6 Minute Walking Test
ABSSSI Acute Bacterial Skin and Skin Structure Infection
ACC American College of Cardiology
ACEI Angiotensin-Converting Enzyme Inhibitor
ACR20 American College of Rheumatology 20% Improvement
ACR50 American College of Rheumatology 50% Improvement
ACR70 American College of Rheumatology 70% Improvement
ADC Antibody-Drug Conjugate
ADHD Attention Deficit Hyperactivity Disorder
ADL Activities of Daily Living
ALK Anaplastic Lymphoma Kinase
ALK+ Anaplastic Lymphoma Kinase-positive
ALL Acute Lymphoblastic Leukemia
ALS Amyotrophic Lateral Sclerosis
ALSFRS-R Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised
ALT Alanine Transaminase
AMD Age-Related Macular Degeneration
AML Acute Myeloid Leukemia
ANCA Antineutrophil Cytoplasmic Antibodies
ARB Angiotensin II Receptor Blocker
ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor
AS Ankylosing Spondylitis
ASCVD Atherosclerotic Cardiovascular Disease
AST Aspartate Aminotransferase
BCG Bacillus Calmette-Guérin
BCL-1
BCVA Best Corrected Visual Acuity
BLA Biologics License Application
BMI Body Mass Index
BMT Bone Marrow Transplant
BP Blood Pressure
BPH Benign Prostatic Hyperplasia
BRAF V-Raf Murine Sarcoma Viral Oncogene Homolog B1
BTK Bruton’s Tyrosine Kinase
BSA Body Surface Area
BsUFA Biosimilar User Fee Act
CABP Community Acquired Bacterial Pneumonia
CAP Community Acquired Pneumonia
CAR T Chimeric Antigen Receptor T-Cell
CD Crohn’s Disease
CD3 Cluster of Differentiate 3
CD19 Cluster of Differentiate 19
CD20 Cluster of Differentiate 20
CD38 Cluster of Differentiate 38
CD79b Cluster of Differentiate 79b
CDC Centers for Disease Control and Prevention
CF Cystic Fibrosis
CHF Congestive Heart Failure
CI Confidence Interval
CKD Chronic Kidney Disease
CLDN18.2+ Claudin-18.2-positive
CLL Chronic Lymphocytic Leukemia
CML Chronic Myeloid Leukemia
CMS Centers for Medicare & Medicaid Services
CNS Central Nervous System
COPD Chronic Obstructive Pulmonary Disease
COVID-19 Coronavirus Disease 2019
CRC Colorectal Cancer
CRL Complete Response Letter
CRR Complete Response Rate
CRS Cytokine Release Syndrome
CSF Colony Stimulating Factor
CTLA-4 Cytotoxic T-Lymphocyte-Associated Protein 4
CV Cardiovascular
CVD Cardiovascular Disease
CYP3A4 Cytochrome P-450 3A4
CYP450 Cytochrome P-450
DAS28-CRP Disease Activity Score-28 with C Reactive Protein
DBP Diastolic Blood Pressure
DCR Disease Control Rate
DEA Drug Enforcement Administration
DED Dry Eye Disease
DLBCL Diffuse Large B Cell Lymphoma
DMARD Disease Modifying Antirheumatic Drug
DMD Duchenne Muscular Dystrophy
DME Diabetic Macular Edema
dMMR DNA mismatch repair
DMT Disease Modifying Therapy
DNA Deoxyribonucleic Acid
DOR Duration of Response
DPP-4 Dipeptidyl Peptidase 4
DR Delayed-Release
DSM-5 Diagnostic and Statistical Manual of Mental Disorders, 5th edition
EASI-75 Eczema Area and Severity Index ≥ 75% Reduction
ECOG Eastern Cooperative Oncology Group
EDSS Expanded Disability Status Scale
eGFR estimated Glomerular Filtration Rate
EGFR Epidermal Growth Factor Receptor
ER Extended-Release
ERA Endothelin Receptor Agonist
ESA Erythropoietin Stimulating Agent
ESRD End-Stage Renal Disease
EUA Emergency Use Authorization
FDA Food and Drug Administration
FEV1 Force Expiratory Volume in 1 Second
FH Familial Hypercholesterolemia
FLT3 FMS-Like Tyrosine Kinase-3
FMS Feline McDonough Sarcoma
FVC Forced Vital Capacity
GABA-A Gamma-Aminobutyric Acid Receptor Type A
G-CSF Granulocyte Colony Stimulating Factor
GERD Gastroesophageal Reflux Disease
GGT Gamma-Glutamyl Transferase
GI Gastrointestinal
GIST Gastrointestinal Stromal Tumor
GLP-1RA Glucagon-Like Peptide-1 Receptor Agonist
GM-CSF Granulocyte-Macrophage Colony Stimulating Factor
GVHD Graft Versus Host Disease
H Half
HAE Hereditary Angioedema
HAM-A Hamilton Anxiety Rating Scale
HAM-D Hamilton Depression Rating Scale
HAMD-17 Hamilton Depression Rating Scale
HAP Healthcare-Associated Pneumonia
Hb Hemoglobin
HbA1c Hemoglobin A1c
HBV Hepatitis B Virus
HCC Hepatocellular Carcinoma
HCP Healthcare Professional
HCV Hepatitis C Virus
HDRS-17 Hamilton Depression Rating Scale
HeFH Heterozygous Familial Hypercholesterolemia
HER Human Epidermal Growth Factor Receptor
HER2 Human Epidermal Growth Factor Receptor 2
HER2- Human Epidermal Growth Factor Receptor 2-negative
HER2+ Human Epidermal Growth Factor Receptor 2-positive
HF Heart Failure
HFA Hydrofluoroalkane
HFpEF Heart Failure with preserved Ejection Fraction
HFrEF Heart Failure with reduced Ejection Fraction
HIT Heparin Induced Thrombocytopenia
HIV Human Immunodeficiency Virus
HIV-1 Human Immunodeficiency Virus-1
HR Hazard Ratio
HR+ Hormone Receptor-positive
HoFH Homozygous Familial Hypercholesterolemia
HS Hidradenitis Suppurativa
HSCT Hematopoietic Stem Cell Transplant
HSV Herpes Simplex Virus
HTN Hypertension
IBS Irritable Bowel Syndrome
IBS-C Irritable Bowel Syndrome, Constipation Predominant
ICER Institute for Clinical and Economic Review
ICS Inhaled Corticosteroid
IDH Isocitrate Dehydrogenase
IGA Investigator’s Global Assessment
IgA Immunoglobulin A
IgG Immunoglobulin G
IgG1 Immunoglobulin G1
IgG4 Immunoglobulin G4
IHC Immunohistochemistry
IL-4 Interleukin-4
IL-5 Interleukin-5
IL-8 Interleukin-8
IL-12 Interleukin-12
IL-13 Interleukin-13
IL-17 Interleukin-17
IL-23 Interleukin-23
IL-31 Interleukin-31
IM Intramuscular
IR Immediate-Release
IRB Institutional Review Board
ISH In Situ Hybridization
ITP Immune Thrombocytopenic Purpura
ITT Intent-To-Treat
IV Intravenous
JAK Janus Kinase Inhibitor
JIA Juvenile Idiopathic Arthritis
KIT c-KIT Proto-Oncogene
KMT2A Lysine (K)-Specific Methyltransferase 2A
KRAS Kirsten Rat Sarcoma viral oncogene homolog
LABA Long-Acting Beta Agonist
LAMA Long-Acting Muscarinic Antagonist
LDL-C Low-Density Lipoprotein Cholesterol
LPAD Limited Population Pathway for Antibacterial and Antifungal Drugs
LS Least Square
LVEF Left Ventricular Ejection Fraction
mAb Monoclonal Antibody
MACE Major Adverse Cardiovascular Events
MADRS Montgomery – Åsberg Depression Rating Scale
MDD Major Depressive Disorder
MDI Metered Dose Inhaler
MDR Multi-Drug Resistant
MECP2 Methyl-CpG Binding Protein 2
MEK Mitogen-Activated Extracellular Signal-Regulated Kinase
MI Myocardial Infarction
mITT modified Intent-To-Treat
MRI Magnetic Resonance Imaging
MRSA Methicillin-Resistant Staphylococcus Aureus
MS Multiple Sclerosis
MSI-H Microsatellite Instability-High
mTOR mechanistic Target of Rapamycin
N/A Not Applicable
NAFLD Nonalcoholic Fatty Liver Disease
NASH Non-Alcoholic Steatohepatitis
NCCN National Comprehensive Cancer Network
NCT National Clinical Trials
NDA New Drug Application
NHL Non-Hodgkin Lymphoma
NIH National Institutes of Health
nr-axSpA Non-Radiographic Axial Spondyloarthritis
NRAS Neuroblastoma RAS Proto-Oncogene
NRG1+ Neuregulin-1-positive
NSAID Non-Steroidal Anti-Inflammatory Drug
NSCLC Non-Small Cell Lung Cancer
NTRK Neurotrophic Tyrosine Receptor Kinase
NYHA New York Heart Association
ODT Orally Disintegrating Tablet
OR Odds Ratio
ORR Objective Response Rate
OS Overall Survival
OTC Over-the-Counter
PAD Peripheral Arterial Disease
PAH Pulmonary Arterial Hypertension
PARP Poly (ADP-Ribose) Polymerase
PAS Prior Approval Supplement
PASI Psoriasis Area and Severity Index
PASI 50 Psoriasis Area and Severity Index 50% Reduction
PASI 75 Psoriasis Area and Severity Index 75% Reduction
PASI 90 Psoriasis Area and Severity Index 90% Reduction
PASI 100 Psoriasis Area and Severity Index 100% Reduction
PCI Percutaneous Coronary Intervention
PCSK9 Proprotein Convertase Subtilisin Kexin 9
PD-1 Programmed Death Protein 1
PD-L1 Programmed Death-Ligand 1
PDE3 Phosphodiesterase 3
PDE4 Phosphodiesterase 4
PDE5 Phosphodiesterase 5
PDUFA Prescription Drug User Fee Application
PFS Progression-Free Survival
PGA Physician Global Assessment
PHI Primary Humoral Immunodeficiency
PI3K Phosphatidylinositol-3-kinase
PNH Paroxysmal Nocturnal Hemoglobinuria
PsA Psoriatic Arthritis
PSO Plaque Psoriasis
PTCA Percutaneous Transluminal Coronary Angioplasty
PTSD Post-Traumatic Stress Disorder
Q Quarter
QIDP Qualified Infectious Diseases Product
QOL Quality of Life
R/R Relapsed or Refractory
R-CHOP Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, Prednisone
RA Rheumatoid Arthritis
RAS Ras Protein Superfamily
RBC Red Blood Cell
RCC Renal Cell Carcinoma
REMS Risk Evaluation and Mitigation Strategy
RMAT Regenerative Medicine Advanced Therapy
RNA Ribonucleic Acid
ROS1 ROS Proto-Oncogene 1
RPD Rare Pediatric Disease
RRR Relative Risk Reduction
RSV Respiratory Syncytial Virus
RTOR Real-Time Oncology Review
RVO Retinal Vein Occlusion
SARS-CoV-2 Severe Acute Respiratory Syndrome-Associated Coronavirus-2
sBLA supplemental Biologics License Application
SBP Systolic Blood Pressure
SC Subcutaneous
SCCHN Squamous Cell Cancer of the Head and Neck
SCD Sickle Cell Disease
SCLC Small Cell Lung Cancer
SCT Stem Cell Transplant
SGLT2 Sodium-Glucose Co-Transporter 2
SL Sublingual
SLE Systemic Lupus Erythematosus
SLL Small Lymphocytic Lymphoma
sNDA supplemental New Drug Application
SNRI Serotonin and Norepinephrine Reuptake Inhibitor
SOC Standard of Care
SOD-1 Superoxide Dismutase – Type 1
sPGA static Physician Global Assessment
SR Sustained-Release
SSRI Selective Serotonin Reuptake Inhibitor
SSSI Skin and Skin Structure Infection
T1DM Type 1 Diabetes Mellitus
T2DM Type 2 Diabetes Mellitus
TBD To Be Determined
TEAE Treatment-Emergent Adverse Event
TKI Tyrosine Kinase Inhibitor
TNBC Triple Negative Breast Cancer
TNF Tumor Necrosis Factor
TNFα Tumor Necrosis Factor-alpha
UA Unstable Angina
UC Ulcerative Colitis
ULN Upper Limit of Normal
U.S. United States
UTI Urinary Tract Infection
VAS Visual Analog Scale
VEGF Vascular Endothelial Growth Factor
VTE Venous Thromboembolism
WBC White Blood Cell
WHO World Health Organization
XR Extended-Release
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