Quarterly Drug Pipeline: April 2025 - Prime Therapeutics
Quarterly Drug Pipeline: April 2025
Clinical insights and competitive intelligence on anticipated drugs in development
Editor-in-chief's message
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 FDA review and those in phase 3 studies 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
In the first quarter of 2025, the agency has approved 7 novel drugs which is 30% less that the number of approvals about the same time last year. New options in oncology, rare disease and a new antibiotic for uncomplicated urinary tract infection for females are among some of this year’s novel approvals so far. One of the notable novel approvals in the first quarter of 2025 is suzetrigine (Journavx). This is a first-in-class oral non-opioid analgesic for the treatment of moderate to severe acute pain in adults.
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 (74%) and for orphan drugs (38%) continue to grow for agents with applications submitted to the FDA. Nine therapies are seeking FDA’s Accelerated Approval. FDA’s Accelerated Approval pathway allows for earlier approval of drugs to treat serious conditions, and fill an unmet medical need based on a surrogate endpoint. Confirmatory trials are needed to confirm clinical benefit for these drugs to remain on the market.
Notable anticipated approvals for second quarter 2025 include:
- A cell-based gene therapy for epidermolysis bullosa, a rare genetic skin disorder
- First medicine for Menkes disease, a rare genetic metabolic disorder
- A new cellular therapy for patients with advanced melanoma
- By the end of summer of 2025, FDA decision is expected for six new gene and cellular therapies, four of which are seeking Accelerated Approval.
- In the fall of 2025, several new indications are expected for existing GLP-1s such as:
- Injectable semaglutide (NN9931) for metabolic dysfunction-associated steatohepatitis (MASH)
- Injectable semaglutide (Wegovy) and tirzepatide (Zepbound) for heart failure with preserved ejection fraction (HFpEF) in patients with obesity
- Oral semaglutide (Rybelsus) for major adverse cardiovascular events (MACE) risk reduction in adults with type 2 diabetes (T2DM) and established CVD and/or CKD
- Moreover, we are closely monitoring the pipeline for oral GLP-1s. Visit Prime’s GLP-1 Pipeline Update to learn more.
We hope you enjoy the report!
Maryam Tabatabai
Associate Vice President, Clinical Information
Editorial team
Maryam Tabatabai, PharmD
Editor-In-ChiefAssociate Vice President, Clinical Information
Carole Kerzic, RPh
Executive Editor
Drug Information Pharmacist Principal
Nicole Kjesbo, PharmD, BCPS
Executive Editor
Clinical Program Development Director Senior
Consultant panel
Robert Greer, RPh, BCOP
Vice President, Clinical Strategy and Programs
Andrea Henry, PharmD, MBA, BCPS
Drug Information Pharmacist Principal
Danny Melson
Data Scientist Principal
All brand names are property of their respective owners.
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 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.
Deep dive
brensocatib oral
Manufacturer: Insmed / AstraZeneca
Proposed indications
Non-cystic fibrosis (CF) bronchiectasis (NCFB)
Clinical overview
Mechanism of action
The dipeptidyl peptidase 1 (DPP-1) enzyme activates neutrophil serine proteases (NSPs) in neutrophils when formed in the bone marrow. In chronic inflammatory lung diseases, neutrophils accumulate in the airways leading to an excess of active NSPs that cause inflammation and damage. Brensocatib is an oral, reversible DPP-1 inhibitor that may reduce damage to the lungs by reducing NSP activation.
Clinical trials
The randomized, double-blind, placebo-controlled, Phase 3 ASPEN (NCT04594369) study evaluated brensocatib in patients 12–85 years of age with NCFB confirmed by chest CT scan and at least two pulmonary exacerbations (PEs) in the prior 12 months. Enrolled patients did not have COPD, asthma or an immunodeficiency disorder and were not current smokers. Treatment with brensocatib 10 mg or 25 mg once-daily resulted in statistically significant reductions in the primary endpoint of annualized rate of PEs compared to placebo (placebo adjusted reduction, 21.1% [p=0.0019] with 10 mg dose and 19.4% [p=0.0046] with 25 mg dose). Statistically significant improvement in some secondary endpoints were also reported including prolongation of time to first PE compared to placebo (18.7% and 17.5%, respectively for each dose), increased odds of remaining exacerbation-free over 52 weeks (41.2% and 40%, respectively for each dose), and change from baseline in FEV1 (38 mL [p=0.0054] for 25 mg dose only). Brensocatib was generally well tolerated. Hyperkeratosis (≥ 5%) was a TEAE of special interest reported in the study.
Dosage and administration
In the ASPEN trial, brensocatib was administered in doses of 10 mg or 25 mg orally once daily for 52 weeks.
Place in therapy
Bronchiectasis is a chronic inflammatory lung condition caused by severe or recurrent lung infections. It is characterized by permanent dilatation and loss of elasticity of the bronchi resulting in mucus accumulation, chronic airway infection and persistent neutrophilic inflammation. While bronchiectasis is a common manifestation of the genetic conditions cystic fibrosis and alpha-1-antitrypsin deficiency, certain autoimmune diseases, such as RA, Sjogren’s syndrome, Crohn’s disease and ulcerative colitis and immunodeficient conditions, such as HIV-1 infection, may increase the risk of developing bronchiectasis. Patients with bronchiectasis experience persistent cough and mucopurulent sputum production and pulmonary exacerbations requiring antibiotic therapy and/or hospitalization. Dyspnea, wheezing and pleuritic chest pain may also be present. Lung function tests (e.g., FEV1, FVC) reveal obstructive impairment. Bronchiectasis is reported in approximately 1 in 150–350 individuals who are ≥ 75 years of age. An estimated 350,000 to 500,000 adults have bronchiectasis in the U.S. with more women affected than men.
Treatment of bronchiectasis is generally aimed at managing underlying conditions, removing excess mucus and preventing PEs and lung infections. This includes use of antibiotics (oral, IV), expectorants and decongestants as well as adequate hydration and chest physiotherapy (CPT). Surgery may be considered in extreme cases with bronchiectasis isolated to a section of lung or with excessive bleeding.
Brensocatib is a first-in-class DDP-1 inhibitor. If approved, it will be the first agent to treat the underlying pathology of NSPs associated with NCFB. In the ASPEN trial, once-daily oral brensocatib significantly reduced the annualized rate of PE and improved lung function (as measured by FEV₁) compared to placebo in patients with NCFB. Notably, ICER announced that it will assess the comparative clinical effectiveness and value of brensocatib for the treatment of NCFB and will discuss findings at a public meeting in September 2025.
FDA approval timeline
FDA designations: Breakthrough Therapy, Priority Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $109 |
| 2026 | $428 |
| 2027 | $836 |
| 2028 | $1,264 |
| 2029 | $1,684 |
copper histidinate (CUTX-101) SC
Manufacturer: Fortress / Zydus
Proposed indications
Menkes disease
Clinical overview
Mechanism of action
CUTX-101 is a copper replacement therapy to maintain serum copper levels.
Clinical trials
Data were combined from two long-term, open-label, single-arm, single-site studies, including a Phase 1/2 study (NCT00001262) and a Phase 3 study (NCT00811785), that evaluated CUTX-101 in patients with severe ATP7A genotype Menkes disease. Patients were categorized into two cohorts, an early treatment (ET) cohort (n=31) with patients who initiated CUTX-101 within 4 weeks of birth and a late treatment (LT) cohort (n=35) with patients who started CUTX-101 after 4 weeks of birth; age in both cohorts was adjusted for prematurity. Data in the ET and LT cohorts were compared to an untreated historical control (HC) group (n=18) with severe ATP7A genotype Menkes disease. The subjects in the HC group were asymptomatic for significant neurological symptoms approximately 4 weeks after birth, diagnosed with Menkes disease after 4 weeks of birth and survived for at least two weeks after diagnosis. Data analysis revealed a significant improvement in the primary endpoint of OS among patients in the ET cohort compared to the HC group (median OS, 177.1 months verses 16.1 months, respectively; p<0.0001). As a secondary endpoint, a significant improvement in OS was also reported among the LT group compared to the HC group (median OS, 62.4 versus 17.6 months, respectively; p<0.0001). In addition, the Phase 1/2 trial reported significant improvements in gross motor, fine motor/adaptive, personal-social and language neurodevelopment in ET subjects who initiated CUTX-101 prior to onset of symptoms. The most common adverse events reported among patients who received CUTX-101 were pneumonia, seizures, dehydration, failure to thrive and respiratory distress; however, none were considered related to CUTX-101.
Dosage and administration
In both trials, a CUTX-101 dose of 1,450 mcg (250 mcg elemental copper) was administered SC twice daily until 12 months of age, followed by 1,450 mcg once daily thereafter. CUTX-101 treatment was limited to a three-year duration due to the risk of nephrotoxicity and because brain myelination is typically completed by 24 months of age.
Place in therapy
Menkes disease is a rare, congenital, X-linked, metabolic disorder caused by mutations in the copper transporting ATP7A gene. Patients with Menkes disease cannot properly absorb copper from their diet leading to abnormally low levels of copper in the brain and liver. Onset of signs and symptoms of Menkes disease occur at about 6 to 8 weeks of age. Characteristic features include hypotonia, seizures, failure to thrive, progressive neurodegeneration and connective tissue dysfunction. Death usually occurs by 3 years of age. The estimated incidence of Menkes disease in the U.S. varies greatly. A 2020 study estimated 1 in 8,664 male births in the U.S., or 225 babies each year, will be affected by Menkes disease. Approximately 75% of cases involve infants born to mothers who are carriers of the ATP7A gene mutation.
Currently, there are no FDA-approved treatments for Menkes disease. Investigational parenteral copper replacement therapy has been used in clinical trials or expanded access/compassionate use programs and studies suggest that initiating parenteral copper replacement therapy by 4 weeks of age may improve clinical outcomes. Early diagnosis of Menkes disease is essential; however, there are no newborn screening programs available. For at-risk infants, diagnosis is typically based on detection of abnormal plasma levels of neurochemicals associated with copper metabolism, including dopamine, norepinephrine, dihydroxyphenylacetic acid and dihydroxyphenylglycol. ATP7A mutation testing is commercially available for infants suspected of having Menkes disease based on clinical and biochemical status.
If approved, CUTX-101 will be the first medicine available for Menkes disease. In clinical trials, it was associated with significant improvement in survival and neurological outcomes compared to an untreated historical control group.
FDA approval timeline
FDA designations: Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, RPD
Financial forecast (reported in millions)
The financial forecast for CUTX-101 is not currently available.deramiocel IV
Proposed indications
Duchenne muscular dystrophy (DMD) cardiomyopathy
Clinical overview
Mechanism of action
Deramiocel is created using healthy myocardial tissue from human donors. The tissue is cultured to produce cardiosphere-derived cells (CDCs) that secrete exosomes (extracellular vesicles). The exosomes contain non-coding microRNAs that alter gene expression of macrophages to reduce inflammation and stimulate tissue regeneration.
Clinical trial(s)
The double-blind, placebo-controlled, Phase 2 HOPE-2 (NCT04428476) trial evaluated deramiocel in male patients ≥ 10 years of age with genetically confirmed DMD. Enrolled patients were either late ambulatory or non-ambulatory, had moderate limb impairment (Performance of Upper Limb [PUL] motor function score 2–5) and received prior glucocorticoid therapy for ≥ 12 months. Twenty patients were randomized to deramiocel (n=8) or placebo (n=12). The study reported a 71% slowing of loss of function based on the primary efficacy outcome of change from baseline to 12 months in mid-level elbow dimension of PUL 1.2 score with deramiocel compared to placebo (-0.8 points versus -3.4 points, respectively; p=0.014). The change from baseline to 12 months in the secondary outcome of mid-level PUL 2.0 score just missed statistical significance (LSM difference, 29.6 points; p=0.059). Secondary outcomes for cardiac function revealed a significant difference in the mean change in LVEF at 12 months with deramiocel compared to placebo (+0.1 versus -3.9 percentage points, respectively; p=0.0022). In addition, deramiocel significantly improved indexed left ventricular end systolic volume (ESVI) (LSM difference, 53.1 mL/m²; p=0.013) and indexed left end diastolic volume (EDVI) (LSM difference, 47.8 mL/m²; p=0.031) compared to placebo. Infusion-related hypersensitivity reactions were observed in three patients who received deramiocel, with one patient discontinuing therapy due to a severe allergic reaction. To minimize the risk of hypersensitivity reactions, the study protocol was amended to include a pretreatment regimen comprising a glucocorticosteroid, histamine-1 blocker and histamine-2 blocker, after which deramiocel was generally well tolerated.
Five patients who received deramiocel and seven who received placebo in the HOPE-2 trial completed the open-label extension HOPE-2-OLE trial. After a gap phase of approximately 392 days when no treatment was administered, all 12 patients received deramiocel and remained on treatment through month 36. In a cohort-matched external comparator analysis, the extension study showed that treatment with deramiocel over 36 months resulted in a mean decline in PUL 2.0 total score (primary endpoint) of 3.46 points, compared to a 7.19-point decline in an external comparator group (p=0.019), translating to a 52% slowing of disease progression. Regarding secondary cardiac function measures, after 36 months, the median declines in LVEF and ESVI were 1.2 percentage points and 2.4 mL/m², respectively. Notably, greater cardiac benefit was seen in patients with LVEF > 45% at the end of the blinded HOPE-2 trial. This subgroup experienced median declines in LVEF and ESVI of 3.1 percentage points and 5.1 mL/m², respectively, after 36 months. No additional safety concerns were reported.
Topline data from the randomized, double-blind, placebo-controlled Phase 3 HOPE-3 (NCT05126758) trial are pending. The study is evaluating safety and efficacy of deramiocel in 104 ambulatory and non-ambulatory male patients ≥ 10 years of age with DMD. Primary completion of the study is estimated in December 2025
Dosage and administration
In the HOPE-2 trial, deramiocel 1.5 x 10⁸ CDCs was administered IV every three months for a total of four infusions in an outpatient setting. Every-three-month infusions were continued through month 36 of the HOPE-2-OLE study.
Place in therapy
DMD is a rare, X-linked neuromuscular disorder caused by mutations in the dystrophin (DMD) gene that result in a lack of functional dystrophin protein involved in maintaining muscle fiber integrity. This leads to progressive muscle degeneration, pulmonary dysfunction and cardiomyopathy. An estimated 400 to 600 boys are born with DMD each year in the U.S. Onset of DMD symptoms appear between 3 to 5 years of age. Loss of the ability to walk often occurs by 12 years of age and development of cardiomyopathy is typically seen around 14 years of age. Death due to respiratory or cardiac failure typically occurs by early 30s.
Cardiomyopathy is associated with myocardial fibrosis, left ventricular enlargement and left ventricular dysfunction. LVEF is a measure of cardiac pump function.
Treatment with ACEIs, ARBs and beta blockers can slow the of cardiac muscle deterioration associated with DMD. Use of select corticosteroids (e.g., prednisone, deflazacort [Emflaza], vamorolone [Agamree]) is the SOC pharmacotherapy for DMD to improve motor function, strength and pulmonary function and delay the loss of ambulation. Some studies suggest that corticosteroids may also delay onset of cardiomyopathy; however, corticosteroids are associated with side effects such as weight gain, slowed growth trajectories, bone fractures and cataracts. Other pharmacotherapies that may be used with SOC have been developed to target the genetic causes of the DMD. They include injectable exon skipping oligonucleotides (casimersen [Amondys 45], eteplirsen [Exondys 51], golodirsen [Vyondys 53] and viltolarsen [Viltepso]), the gene therapy delandistrogene moxeparvovec-rokl (Elevidys) and investigational oral ataluren. All three therapies deliver functional dystrophin protein but have not reported benefit for DMD cardiomyopathy. Ataluren is awaiting FDA approval in the third quarter of 2025. The oral histone deacetylase (HDAC) inhibitor givinostat (Duvyzat) slows disease progression, increases muscle mass, reduces the amount of fibrotic tissue and reduces muscle tissue necrosis; however, benefit for cardiomyopathy has also not been reported.
If approved, deramiocel will be the first cellular therapy available to treat DMD cardiomyopathy, a leading cause of death among patients with DMD. The off-the-shelf product is administered four times a year and has the potential for use for DMD cardiomyopathy regardless of the specific DMD-causing gene mutation. In the Phase 2 HOPE-2 trial and its extension study, deramiocel demonstrated improvement in skeletal muscle function and cardiac function for up to three years.
FDA approval timeline
Aug. 31, 2025
FDA designations: Orphan Drug, Priority Review, RMAT, RPD
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $10 |
| 2026 | $59 |
| 2027 | $148 |
| 2028 | $268 |
| 2029 | $346 |
dordaviprone (ONC201) oral
Proposed indications
Recurrent histone 3 lysine 27-to-methionine (H3 K27M)-mutant diffuse glioma
Clinical overview
Mechanism of action
Dordaviprone is a first-in-class oral imipridone. It is a selective antagonist of the dopamine receptor D2 (DRD2) and agonist of the mitochondrial protease caseinolytic proteolytic P (ClpP) subunit with potential for antiproliferative, cytotoxic, and pro-apoptotic effects against tumor cells. Overexpression of DRD2 is observed in glioma, particularly in midline regions where high levels of dopamine are produced and are therefore susceptible to DRD2 antagonism.
Clinical trial(s)
Dordaviprone was evaluated in three Phase 2 trials (NCT03295396; NCT02525692; NCT03134131), one Phase 1 trial (NCT03416530), and a compassionate use program. Pooled data from these programs were evaluated. The trials included 50 patients ≥ 2 years of age with measurable recurrent diffuse midline glioma (DMG) that harbored H3 K27M mutation who had received prior radiation therapy. Patients were evaluated by blinded independent central review (BICR). Based on the established response assessment criteria for gliomas Response Assessment in Neuro-Oncology (RANO) 2.0, monotherapy with dordaviprone resulted in an ORR of 28% with no complete responses. The median time to response was 4.6 months (range, 1.6–15.9), median DOR was 10.4 months (range, 7.4–15.4), and DCR was 40%. Safety data from 245 patients on various doses (125 mg–750 mg) showed dordaviprone was well tolerated. The most common TEAEs were mild nausea and dizziness.
Dordaviprone is also being studied in the double-blind, placebo-controlled, parallel-group, Phase 3 ACTION trial in adult and pediatric patients with newly diagnosed H3 K27M-mutant DMG. In this study, dordaviprone is given following radiotherapy, without waiting for recurrence of disease. Dual primary endpoints are OS and PFS. The primary completion of the study is anticipated in August 2026.
In clinical trials, dordaviprone was administered orally once weekly or every 3 weeks, according to each study design. The dose in adults was 625 mg and the dose in pediatrics was weight-based ranging from 125 mg to 625 mg.
Place in therapy
DMG is a high-grade (WHO Grade III or IV) glioma found in the midline structures of the brain (thalamus, brainstem, pons) and the spinal cord. It is commonly associated with H3 K27M mutation (reported prevalence, 40% to 90% of DMGs); H3 K27M mutation is rarely reported in non-midline gliomas. DMGs, particularly with H3 K27M mutation, are aggressive and have a poor prognosis with a median survival of about one year or less. DMG affects both children and adults. It is estimated that approximately 3,940 people in the U.S. are living with DMG and about 577 new cases are diagnosed each year.
Due to location, DMGs are typically inoperable. There are no pharmacotherapies approved specifically for H3 K27M-mutant glioma. Radiation therapy is the SOC but is usually not curative. IV bevacizumab (Avastin, biosimilars) is indicated for recurrent glioblastoma in adults. While it has shown prolonged PFS when given with lomustine, it had no effect on OS in clinical trials.
Dordaviprone is a first-in-class oral imipridone. It is the first systemic therapy that has shown benefit in treating recurrent H3 K27M-mutant DMG.
FDA approval timeline
Aug. 18, 2025
FDA designations: Fast Track, Orphan Drug, Priority Review, RPD, seeking Accelerated Approval
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $0 |
| 2026 | $12 |
| 2027 | $65 |
| 2028 | $121 |
| 2029 | $177 |
rebisufligene etisparvovec (UX111) IV
Manufacturer: Ultragenyx
Proposed indications
Sanfilippo A syndrome (Mucopolysaccharidosis III type A [MPS-IIIA])Clinical overview
Mechanism of actionUX111 is an in vivo gene therapy using a self-complementary AAV9 vector to deliver a functional copy of the N-sulfoglucosamine sulfohydrolase (SGSH) gene to cells.
Clinical trial(s)
The ongoing, open-label Phase 1/2/3 Transfer A trial (NCT02716246) is evaluating UX111 in patients ≥ 6 months of age with Sanfilippo A syndrome. The mITT group (n=17) included patients ≤ 2 years of age and patients > 2 years of age with a Bayley Scales of Infant and Toddler Development 3rd edition (Bayley-III) cognitive developmental quotient ≥ 60. At a mean follow-up of 36 months (range, up to 77 months), the study reported a 66% (p<0.0001) reduction in the primary endpoint of heparan sulfate exposure in the cerebrospinal fluid of patients in the mITT group. In addition, a significant improvement in cognitive function was reported with UX111 compared to natural history of untreated patients (Bayley-III raw cognitive score in patients 24–60 months of age, +16 points versus -6.8 points, respectively; p<0.0001). Numerical improvement in fine and gross motor scores were also reported. In addition, retention of meaningful functional abilities (e.g., communication, ambulation, feeding) were observed among 10 patients who received UX111 but were not in the mITT population due to older age or having more advanced disease. UX111 was generally well tolerated. Transient, mild to moderate elevations in liver enzymes were reported.
Dosage and administration
In the trial, patients in the mITT group received a one-time IV infusion of UX111 at a dose of 3 x 10¹³ vg/kg.
Place in therapy
Sanfilippo A syndrome is a rare, autosomal recessive, lysosomal storage disease. It is due to a mutation in the SGSH gene resulting in a deficiency of sulfamidase, causing an accumulation of heparan sulfate in lysosomes, which leads to cellular dysfunction, primarily in the CNS. In Sanfilippo A syndrome, developmental delay becomes evident by 2–5 years of age and progresses to intellectual and motor deterioration and behavioral disturbances. The average life expectancy of individuals with Sanfilippo A syndrome is about 15 years. The estimated prevalence of the condition in the U.S. is 0.52 per 1,000,000 live births.
There is no cure for Sanfilippo A syndrome. Current treatment is supportive. If approved, UX111 will be the first pharmacological treatment for patients with Sanfilippo A syndrome. The one-time gene therapy is designed to correct sulfamidase enzyme deficiency. Clinical trials demonstrated that UX111 significantly reduced heparan sulfate exposure and improved cognitive function. Study data also suggested that UX111 may improve communication and fine and gross motor skills compared to the non-treated historical control group.
FDA approval timeline
Jul. 22, 2025FDA designations: Breakthrough Therapy, Priority Review, seeking Accelerated Approval
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $23 |
| 2026 | $77 |
| 2027 | $145 |
| 2028 | $214 |
| 2029 | $294 |
rilzabrutinib oral
Proposed indications
Immune thrombocytopenic purpura (ITP)
Clinical overview
Mechanism of action
Rilzabrutinib is an oral, reversible, covalent Bruton’s tyrosine kinase (BTK) inhibitor.
Clinical trial(s)
The randomized, double-blind, placebo-controlled, Phase 3 LUNA 3 (NCT04562766) trial with an open-label extension evaluated rilzabrutinib in 232 patients ≥ 12 years of age with persistent or chronic primary ITP. Enrolled patients had two platelet counts < 30,000/µL taken at least five days apart during the screening period and no single platelet count > 35,000/µL within 14 days prior to the first dose of study drug. Patients had either a nondurable or insufficient response to SOC or had a contraindication or intolerance to SOC for ITP. The primary endpoint of durable platelet response is defined as the proportion of participants to achieve platelet counts ≥ 50,000/μL for at least eight out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy. In the trial, 23% of patients who received rilzabrutinib compared to 0% of those who received placebo (p<0.0001) achieved a durable platelet response. Based on pooled data from the double-blind and open-label periods, 29% of patients who received rilzabrutinib achieved a durable platelet response at data cutoff. In addition, significant improvement in bleeding was reported with rilzabrutinib compared to placebo as measured by the Immune Thrombocytopenic Purpura Bleeding Score (mean change from baseline at 25 weeks, -0.04 versus +0.05, respectively; p=0.0006). A 52% reduction in rescue therapy was also reported with rilzabrutinib compared to placebo (p=0.0007). The most common TEAEs reported with rilzabrutinib were mild or moderate diarrhea, nausea, headache and abdominal pain.
Dosage and administration
In the LUNA 3 trial, rilzabrutinib 400 mg was administered orally twice daily for up to 24 weeks in the double-blind period followed by 28 weeks in the open-label period.
Place in therapy
Primary ITP is an autoimmune disease characterized by thrombocytopenia due to impaired platelet production and increased platelet destruction in the body. Patients with ITP experience fatigue, bruising, petechiae and bleeding that is often unpredictable. Bleeding events may be serious, including menorrhagia, epistaxis, GI hemorrhage, hematuria and rarely, intracranial hemorrhage. ITP prevalence in the U.S. is approximately 8 per 100,000 in children and 12 per 100,000 in adults. The likelihood of remission is dependent on age. The reported one-year remission rates are 74% in children < 1 year of age, 67% in those 1–6 years of age and 62% in those 10–20 years of age.
Treatment of ITP should be individualized based on the patient’s clinical status and preferences and the estimated risk of bleeding. Platelet transfusions are usually reserved for cases where there is an immediate need to raise the platelet count, such as critical bleeding or an urgent need for an invasive procedure that is associated with a significant risk of bleeding. Combination therapy with IV-administered corticosteroids and immune globulin (IVIG) is used to treat critical bleeding, and either agent can be used alone for minor to severe bleeding, severe thrombocytopenia without bleeding or prior to elective surgery. Corticosteroids are generally preferred over IVIG due to cost and ease of outpatient administration; however, IVIG increases platelet count more rapidly and may be a preferred option because it lacks the side effects associated with corticosteroid therapy. Anti-D immune globulins are an alternative to IVIG in patients whose blood type is RhD-positive and have not undergone a splenectomy. Second-line therapy includes thrombopoietin receptor agonists (TPO-RA; oral avatrombopag [Doptelet], oral eltrombopag [Promacta], SC romiplostim [Nplate]) and off-label rituximab (Rituxan, biosimilars). TPO-RAs may require regular use as maintenance therapy and are associated with bone marrow fibrosis (reversible), hepatotoxicity and increased risk of thrombosis and thromboembolism. The oral splenic tyrosine kinase inhibitor fostamatinib (Tavalisse) is indicated for use in patients with insufficient response to prior treatment and has been primarily studied in the third-line setting. Splenectomy is also a option, particularly in patients who desire a potentially curative treatment, but the procedure is usually delayed for at least one year from diagnosis to allow an opportunity for ITP remission to occur.
If approved, rilzabrutinib will be the first BTK inhibitor indicated for the treatment of ITP and will provide another oral option for the condition. In the Phase 3 clinical trial, rilzabrutinib demonstrated a durable platelet response, reduced bleeding and reduced need for rescue therapy in patients ≥ 12 years of age with persistent or chronic ITP who experienced a prior nondurable or insufficient response or intolerance to SOC.
FDA approval timeline
Aug. 29, 2025
FDA designation: Fast Track, Orphan Drug
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $20 |
| 2026 | $101 |
| 2027 | $135 |
| 2028 | $171 |
| 2029 | $179 |
vatiquinone oral
Proposed indications
Friedreich's ataxia (FA)
Clinical overview
Mechanism of action
Vatiquinone is a first-in-class selective 15-Lipoxygenase (15-LO) inhibitor designed to alleviate cellular inflammation, oxidative stress due to FA-associated mitochondrial dysfunction and promote neuronal survival.
The Phase 2/3 MOVE-FA trial (NCT04577352) evaluated vatiquinone in patients ≥ 7 years of age with genetically confirmed FA. The trial contained a 72-week double-blind period that randomized patients to vatiquinone or placebo, followed by a 72-week open-label extension period where all patients received vatiquinone. Enrolled patients had a modified Friedreich Ataxia Rating Scale (mFARS) score between 20 and 70 and were able to walk ≥ 10 feet in 1 minute at baseline. The primary endpoint of change from baseline in the overall mFARS score with vatiquinone compared to placebo did not reach statistical significance (difference relative to placebo, -1.61 points; p=0.14) in the primary analysis population at 72 weeks. However, a statistically significant effect was reported on the pre-specified endpoint of mFARS upright stability subscale (difference relative to placebo, -1.26 points; p=0.021). The open-label, long-term extension phase reported a 3.7-point improvement in the mFARS score compared to a matched natural history cohort, which translated to a 50% slowing in disease progression over 3 years. Vatiquinone was generally well tolerated. No serious TEAEs were reported.
Dosage and administration
In the MOVE-FA trial, vatiquinone was administered orally three times a day. The dose was 200 mg in patients < 12 years of age weighing < 25 kg and 400 mg in those ≥ 12 years of age weighing ≥ 25 kg.
Place in therapy
FA is a genetic, progressive, neurodegenerative movement disorder caused by mutations in the frataxin (FXN) gene that codes for frataxin, a protein necessary for proper mitochondrial function. FA is characterized by progressive loss of muscle coordination and control (ataxia) resulting in an unsteady gait and fine limb movement. Affected individuals often develop slurred speech, foot deformities, scoliosis and cardiomyopathy. Onset of FA typically occurs between 10 and 15 years of age, but later onset has been reported. Later onset usually is slower to develop compared to earlier onset. Prevalence of FA is estimated at 1 in 50,000 people in the U.S.
There is no cure for FA. Supportive measures are used to maximize QOL. They include walking aids, physical therapy and surgical and non-surgical orthopedic interventions to maintain patient mobility, speech therapy to maximize verbal communication, and pharmacotherapy to manage cardiomyopathy. In 2023, the FDA approved oral once daily omaveloxolone (Skyclarys) for patients ≥ 16 years of age with FA. In a 48-week trial, omaveloxolone resulted in a significant improvement in mFARS score compared to placebo (difference relative to placebo, -2.41; p=0.0138) among patients ≥ 16 years of age with FA.
If approved, vatiquinone will be the first therapy for pediatric patients 7 to < 16 years of age with FA and will provide an alternative option to omaveloxolone for those ≥ 16 years of age. While the MOVE-FA trial failed to demonstrate a statistically significant improvement in overall mFARS with 72 weeks of vatiquinone therapy, a significant improvement may be achieved after three years of use.
FDA approval timeline
Aug. 19, 2025
FDA designations: Fast Track, Orphan Drug, Priority Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $5 |
| 2026 | $10 |
| 2027 | $16 |
| 2028 | $28 |
| 2029 | $35 |
vusolimogene oderparepvec (RP1) intratumoral
Proposed indications
Advanced melanoma for use in combination with nivolumab
Clinical overview
Mechanism of action
RP1 is a proprietary strain of the herpes simplex virus type 1 (HSV-1) engineered with a fusogenic protein (GALV-GP R⁻) and GM-CSF to maximize its tumor killing ability and increase immunogenic tumor cell death.
Clinical trial(s)
The ongoing, open-label, Phase 2 IGNYTE (NCT03767348) trial is evaluating safety (primary endpoint) and efficacy (secondary endpoints) of RP1 in adults with advanced and/or refractory solid tumors. The primary analysis included data from 140 patients with cutaneous melanoma that had failed anti-PD-1 therapy. Enrolled patients received RP1 plus nivolumab. The study revealed that after a median follow-up of 15.4 months, the ORR was 33.6% by modified RECIST 1.1 criteria and CRR was 15%. The median DOR was 21.6 months (range, 1.2 to 43.5). In addition, among patients who responded, most (85%) injected and non-injected lesions were reduced in size by at least 30%. The median OS had not been reached. Survival rates at 1-, 2- and 3-years were 75.3%, 63.3%, and 54.8%, respectively. RP1 plus nivolumab was generally well tolerated. Most adverse events were Grade 1 or 2 and included fatigue, chills, pyrexia, GI symptoms, injection site pain, headache, rash and pruritus. Grade 4 events consisted of one event each of elevated lipase, CRS, myocarditis, hepatic cytolysis and splenic rupture.
The ongoing confirmatory Phase 3 IGNYTE-3 (NCT06264180) trial is evaluating RP1 in combination with nivolumab compared to physicians’ choice of treatment (e.g., nivolumab/relatlimab-rmbw [Opdualag], chemotherapy [dacarbazine, temozolomide, paclitaxel/nab-paclitaxel], anti-PD-1 monotherapy) in patients ≥ 12 years of age with advanced cutaneous melanoma (Stage IIIb–IV) who progressed on anti-PD-1 and anti-CTLA-4 therapy. Primary study completion is anticipated in 2029.
In the IGNYTE trial, RP1 is administered by intratumoral injection at a dose of 1 x 10⁶ plaque-forming units (pfu)/mL in Cycle 1 followed by 1 x 10⁷ pfu/mL every two weeks in Cycles 2–8. Multiple tumors may be injected and injections should be made from largest to smallest lesions. Nivolumab 240 mg is administered IV every two weeks during cycles 2–9 and as 480 mg every four weeks during cycles 10–30. Patients were allowed to restart RP1 beyond eight cycles if they met protocol-specific criteria.
Place in therapy
It is estimated that 104,960 new cases of melanoma will be diagnosed and about 8,430 deaths will occur due to the condition in the U.S. in 2025.
Most cutaneous melanoma cases are diagnosed at an early stage when surgical excision is curative. Checkpoint inhibitor immunotherapy, including PD-1 inhibitors (e.g., nivolumab [Opdivo], pembrolizumab [Keytruda]), CTLA-4 inhibitors (e.g., ipilimumab [Yervoy]) and LAG-3 inhibitors (e.g., relatlimab-rmbw [component of Opdualag]), are the primary systemic treatment for advanced or metastatic disease (Stage III-IV) and in select patients with Stage II disease to reduce the risk of recurrence. Targeted therapy with BRAF plus MEK inhibitors (e.g., dabrafenib [Tafinlar] plus trametinib [Mekinist]) is recommended for advanced or metastatic disease with BRAF V600 mutation-positive disease, which is present in about half of melanoma cases. However, duration of response to BRAF plus MEK inhibitors is limited due to development of acquired resistance.
Treatment options are limited if melanoma progresses on or after immunotherapy. Combinations of nivolumab with ipilimumab or relatlimab-rmbw may be considered but are associated with Grade 3/4 TEAEs. Other second-line or subsequent therapies include IV-administered lifileucel (Amtagvi) or aldesleukin (Proleukin); however, both carry boxed warnings for severe infection and other adverse events. In addition, intralesional injection of the modified oncolytic HSV-1 therapy talimogene laherparepvec (T-VEC) vaccine (Imlygic) and systemic chemotherapy are also recommended in select patients but have not demonstrated improvement in overall survival. In addition, chemotherapy via isolated limb perfusion or infusion has reported good response rates, but its use is limited due to the complexity of the procedure.
If approved, RP1 would be used in combination with nivolumab. This regimen could compete with lifileucel as second-line treatment for advanced melanoma in patients who failed anti-PD-1.
FDA approval timeline
June 17, 2025
FDA designations: Fast Track, Orphan Drug
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $85 |
| 2026 | $152 |
| 2027 | $236 |
| 2028 | $311 |
| 2029 | $352 |
zopapogene imadenovec (PRGN-2012) sc
Proposed indications
Recurrent respiratory papillomatosis (RRP)
Clinical overview
Mechanism of action
PRGN-2012 is an off-the-shelf AdenoVerse gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11.
Clinical trial(s)
A non-randomized, single-center, single-arm, open-label, Phase 1/2 trial (NCT04724980) evaluated PRGN-2012 in adults with pathologically confirmed RRP. Enrolled patients required at least three interventions in the year prior to study enrollment. Among the 35 patients who received the Phase 2 dosage for PRGN-2012, 51% achieved the primary endpoint of complete response, defined as requiring no surgeries to control RRP in the 12 months after treatment. The median duration of complete response was 20 months at the data cutoff date. In addition, 86% of patients experienced a significant decrease in surgical interventions in the 12 months after treatment compared to the 12 months prior to treatment. PRGN-2012 induced T cell responses specific to HPV 6 and HPV 11 with a significantly greater expansion of peripheral HPV-specific T cells observed in responders compared with non-responders. The most common TEAEs (> 65%) were injection site reaction, fatigue, chills and fever, all of which were mild in severity.
An ongoing, single-arm, open-label, Phase 3 confirmatory trial (NCT06538480) continues to evaluate the safety and efficacy of PRGN-2012 in adults with RRP (estimated n=42); the primary completion of the study is estimated in early 2027.
In the Phase 2 portion of the NCT04724980 study, PRGN-2012 was administered SC at a dose of 5 x 10¹¹ particle units. The first dose was administered on Day 1 following surgical debulking of respiratory papillomatosis lesions with subsequent doses on Days 15, 43 and 85.
Place in therapy
RRP is a rare condition caused by the human papilloma virus (HPV), which is estimated to be present in 75%–80% of adults who have not received an HPV vaccine. Two specific virus subtypes, HPV 6 and HPV 11, account for more than 90% of RRP cases. Respiratory papillomatosis is characterized by the development of wart-like lesions (papillomas) in the respiratory tract. Lesions are most often found in the larynx and vocal cords, but other areas of the respiratory tract may be affected. While the papillomas are benign, they can recur after surgical removal and have rarely become malignant. Symptoms depend on the location of the papillomas, and include dysphonia, stridor (noisy breathing), chronic cough, dysphagia and dyspnea. If left untreated, RRP may lead to acute respiratory distress.
RRP can occur at any age. The estimated incidence of RRP in the U.S. is approximately 2 per 100,000 adults and 4 per 100,000 children with approximately 1,000 new pediatric cases reported each year. However, with increased uptake of the HPV vaccine (Gardasil-9) the number of new cases is declining. Juvenile-onset RRP is typically diagnosed between the ages of 2 and 4 years of age. In adults, RRP is most often diagnosed in the third or fourth decades of life, although, a second peak has been reported around 60 years of age.
There is currently no cure for RRP. Surgical removal of the papillomas is the SOC and the frequency of procedures varies among individuals. Surgical procedures include cold excision, micro-debridement and pulsed dye or carbon dioxide lasers. Medicines such as antivirals (e.g., acyclovir, ribavirin, cidofovir) and interferon have been used off-label to delay papilloma recurrence. While cidofovir has demonstrated partial or complete response against RRP in some patients, a small clinical trial found that the antiviral may be associated with nephrotoxicity and a low risk of malignant transformation of the lesions. It is important to note that the use of Gardasil-9 in boys and girls before they are exposed to HPV has dramatically decreased the incidence of RRP in some countries; however, its role in the treatment of RRP has not been established.
PRGN-2012 is an off-the-shelf therapy that targets HPV-6 and HPV-11. If approved, PRGN-2012 will be the first and only FDA-approved pharmacologic treatment that addresses the underlying cause of RRP. In a Phase 1/2 clinical trial, 51% of adults who received PRGN-2012 required no surgical intervention to control RRP during the 12 months following treatment (complete response). AdenoVerse gene therapies like PRGN-2012 have been shown to generate high-level and durable antigen-specific T cell immune responses and an ability to boost responses with repeat administration
FDA approval timeline
Aug. 27, 2025
FDA designations: Breakthrough Therapy, Orphan Drug, Priority Review, seeking Accelerated Approval
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $16 |
| 2026 | $133 |
| 2027 | $303 |
| 2028 | $433 |
| 2029 | $477 |
Keep on your radar
| Drug generic name | Therapeutic category | Apr 2025 pipeline - Total U.S. sales for 2029 (Dollars in millions) |
|---|---|---|
| aficamten | Cardiovascular | $1,384 |
| anitocabtagene autoleucel | Oncology - Gene Theraopy | $565 |
| apitegromab | Musculoskeletal | $412 |
| botaretigene sparoparvovec | Ophthalmology - Gene therapy | $131 |
| clemidsogene lanparvovec (RGX-121) | Metabolic - Gene therapy | $34 |
| cretostimogene grenadenorepvec (CG0070) | Oncology - Gene Theraopy | $693 |
| depemokimab | Respiratory | $591 |
| donidalorsen | Hematology | $287 |
| orforglipron | Endocrine | $5,877 |
| paltusotine | Endocrine | $465 |
| pariglasgene brecaparvovec (DTX401) | Metabolic - Gene therapy | $129 |
| plozasiran | Cardiovascular | $286 |
| relacorilant | Endocrine | $1,608 |
| telisotuzumab vedotin | Oncology | $346 |
| tolebrutinib | Neurology | $595 |
Drug list
Gene and cellular therapies
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| prademagene zamikeracel (EB-101) | Abeona | Epidermolysis bullosa | Surgical application | BLA; Breakthrough Therapy, Orphan Drug, RMAT, RPD | 04/29/2025 |
| clemidsogene lanparvovec (RGX-121) | Regenxbio | Mucopolysaccharidosis II (Hunter syndrome) | Intrathecal | BLA; Fast Track, Orphan Drug, RMAT, RPD seeking Accelerated Approval | Jul-Dec 2025 |
| vusolimogene oderparepvec (RP1) | Replimune | Melanoma (advanced, in combination with nivolumab, prior anti-PD1 therapy) | Intratumoral | BLA; Breakthrough Therapy, Priority Review, seeking Accelerated Approval | 07/22/2025 |
| rebisufligene etisparvovec (UX111) | Ultragenyx | Sanfilippo A syndrome (Mucopolysaccharidosis III type A) | IV | BLA; Fast Track, Orphan Drug, Priority Review, RMAT, RPD, seeking Accelerated Approval | 08/18/2025 |
| zopapogene imadenovec (PRGN-2012) | Precigen | Recurrent respiratory papillomatosis | SC | BLA; Breakthrough Therapy, Orphan Drug, Priority Review, seeking Accelerated Approval | 08/27/2025 |
| deramiocel | Nippon Shinyaku, Capricor | DMD cardiomyopathy | IV | BLA; Orphan Drug, Priority Review, RMAT, RPD | 08/31/2025 |
| etuvetidigene autotemcel | Fondazione Telethon | Wiskott-Aldrich syndrome | IV | BLA; Orphan Drug, RMAT, RPD | Nov 2025–Mar 2026 |
None
| 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 (CAN-2409) | Candel | Prostate cancer | Transrectal injection | BLA; Fast Track | TBD |
| bidridistrogene xeboparvovec (SRP-9003) | Sarepta | Limb-girdle muscular dystrophy | IV | BLA; Fast Track, Orphan Drug, RPD, may seek Accelerated Approval | TBD |
| cretostimogene grenadenorepvec (CG0070) | CG Oncology | Bladder cancer | Intravesical | BLA; Breakthrough Therapy, Fast Track | TBD |
| giroctocogene fitelparvovec | Pfizer | Hemophilia A | IV | BLA; Fast Track, Orphan Drug, RMAT | TBD |
| marnetegragene autotemcel (Kresladi) | Rocket | Leukocyte adhesion deficiency type I (LAD-I) | IV | BLA; Fast Track, Orphan Drug, RMAT, RPD | TBD |
| orca-T (allogeneic T-cell immunotherapy) | Orca | ALL; AML; Myelodysplastic syndrome | IV | BLA; Orphan Drug, RMAT | TBD |
| pariglasgene brecaparvovec (DTX401) | Ultragenyx | Glycogen storage disease (type 1a) | IV | BLA; Fast Track, Orphan Drug | TBD |
| rexlemestrocel-L (Revascor) | Mesoblast | Chronic HFrEF | IM | BLA; Orphan Drug, RMAT, RPD may seek Accelerated Approval | TBD |
| rilparencel (ReACT) | Prokidney | CKD | Percutaneous (contralateral kidney) | BLA; RMAT | TBD |
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| onasemnogene abeparvovec-xioi (Zolgensma) | Novartis | Spinal muscular atrophy | Intrathecal | sBLA; Breakthrough Therapy, Fast Track, Orphan Drug | TBD |
Biosimilars
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| insulin aspart (biosimilar to Novo Nordisk's Novolog) | Amphastar | T1DM; T2DM | SC | BLA | Apr–Jun 2025 |
| ustekinumab (biosimilar to Janssen's Stelara) | Hikma, Bio-Thera Solutions | PSO; PsA; CD; UC | SC | BLA | May-Jun 2025 |
| ranibizumab (biosimilar to Genentech's Lucentis) | Stada, Xbrane | Choroidal neovascularization; Macular edema from RVO; Wet AMD | Intravitreal | BLA | June 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Teva | Osteoporosis; Bone Cancer | SC | BLA | Aug-Sep 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Organon / Henlius | Osteoporosis; Bone Cancer | IV | BLA | Aug-Sep 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Biocon | Osteoporosis; Bone Cancer | SC | BLA | Aug-Oct 2025 |
| aflibercept (biosimilar to Regeneron's Eylea) | Alvotech, Teva | DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD | Intravitreal | BLA | Oct–Dec 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Mabxience, Amneal | Osteoporosis; Bone Cancer | SC | BLA | Oct–Dec 2025 |
| golimumab (biosimilar to Janssen's Simponi) | Alvotech, Teva | RA; AS; PsA; UC | SC | BLA | Oct–Dec 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Dr. Reddy's, Alvotech | Osteoporosis; Bone Cancer | SC | BLA | December 2025 |
| pertuzumab (biosimilar to Genentech's Perjeta) | Organon, Henlius | Breast cancer | IV | BLA | December 2025 |
None
None
Specialty
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| gemcitabine-releasing intravesical system (TAR-200) | Taris | Bladder cancer (BCG-unresponsive, high-risk non-muscle-invasive, with CIS) | Intravesical | NDA; Breakthrough Therapy, Fast Track, RTOR | 2025 |
| elamipretide | Stealth, AstraZeneca | Barth syndrome | SC | NDA; Fast Track, Orphan Drug, Priority Review, RPD | 04/29/2025 |
| nipocalimab | Janssen | Myasthenia gravis (generalized, anti-AChR, anti-MuSK, anti-LRP4) | IV | BLA; Fast Track, Orphan Drug, Priority Review | 04/29/2025 |
| pegzilarginase | Immedica | Arginase 1 deficiency (ARG1-D) | IV | BLA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, RPD | 05/05/2025 |
| sodium dichloroacetate (SL-1009) | Saol | Pyruvate dehydrogenase complex deficiency (PDCD) | Oral | NDA; Fast Track, Orphan Drug, Priority Review, RPD | 05/27/2025 |
| garadacimab | CSL | HAE | SC | BLA; Fast Track, Orphan Drug | June 2025 |
| sodium chlorite (NP001) | Neuvivo | ALS | IV | NDA; Fast Track, Orphan Drug, seeking Accelerated Approval | March 2025 |
| clesrovimab | Merck | RSV prevention (infants) | IM | BLA | 06/10/2025 |
| mitomycin (UGN-102) | Urogen | Bladder cancer (low-grade intermediate-risk non-muscle invasive) | Intravesical | 505(b)(2) NDA | 06/13/2025 |
| sebetralstat | Kalvista | HAE (on-demand, ages ≥ 12 years) | Oral | NDA; Fast Track, Orphan Drug | 06/17/2025 |
| taletrectinib | Nuvation | NSCLC (advanced ROS1-positive, line-agnostic) | Oral | NDA; Breakthrough Therapy, Orphan Drug, Priority Review | 06/23/2025 |
| avutometinib + defactinib | Verastem, Pfizer | Ovarian cancer (recurrent KRAS mutant, low-grade, serous, in combination with defactinib, ≥ 1 prior systemic therapy) | Oral | NDA; Breakthrough Therapy, Orphan Drug, Priority Review, seeking Accelerated Approval | 06/30/2025 |
| copper histidinate | Fortress, Zydus | Menkes disease | SC | NDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, RPD | 06/30/2025 |
| ataluren (Translarna) | PTC | DMD (nonsense mutation) | Oral | NDA; Fast Track, Orphan Drug | Jul–Sept 2025 |
| zongertinib | Boehringer Ingelheim | NSCLC (unresectable or metastatic, HER2+, prior systemic therapy) | Oral | NDA; Breakthrough Therapy, Fast Track, Priority Review | Jul-Sept 2025 |
| sunvozertinib | Dizal (Jiangsu) | NSCLC (locally advanced or metastatic, EGFR exon 20 insertion mutation, prior platinum-based chemotherapy) | Oral | NDA; Fast Track, Priority Review | 07/07/2025 |
| linvoseltamab | Regeneron | Multiple myeloma (R/R, 4th-line) | IV | BLA; Fast Track | 07/10/2505 |
| delgocitinib | LEO | Chronic hand eczema (corticosteroid failure/contraindication) | Topical | NDA; Fast Track | 07/23/2025 |
| sepiapterin | PTC | Follicular lymphoma (R/R) | IV | BLA; Fast Track, Orphan Drug | 07/29/2025 |
| odronextamab | Regeneron | Follicular lymphoma (R/R) | IV | BLA; Fast Track, Orphan Drug | 07/30/2025 |
| doxecitine / doxribtimine | UCB | Thymidine kinase 2 (TK2) deficiency | Oral | NDA; Breakthrough Therapy, Orphan Drug, Priority Review | August 2025 |
| brensocatib | Insmed, AstraZeneca | Bronchiectasis (non-cystic fibrosis) | Oral | NDA; Breakthrough Therapy, Priority Review | 08/12/2025 |
| troriluzole | Biohaven | Spinocerebellar ataxia | Oral | 505(b)(2) NDA; Fast Track, Orphan Drug, Priority Review | 08/16/2025 |
| dordaviprone (ONC201) | Chimerix | Glioma (diffuse, recurrent H3 K27M-mutant) | Oral | NDA; Fast Track, Orphan Drug, Priority Review, RPD, seeking Accelerated Approval | 08/18/2025 |
| vatiquinone | PTC | Friedreich's ataxia | Oral | NDA; Fast Track, Orphan Drug, Priority Review | 08/19/2025 |
| donidalorsen | Ionis | HAE (prophylaxis to prevent attack, ages ≥ 12 years) | SC | BLA; Orphan Drug | 08/21/2025 |
| bevacizumab-vikg | Outlook | Wet AMD | Intravitreal | BLA | 08/27/2025 |
| leuprolide mesylate (Camcevi) 3-month | Intas | Prostate cancer (palliative treatment) | SC | 505(b)(2) NDA | 08/29/2025 |
| rilzabrutinib | Sanofi | Immune thrombocytopenic purpura (ITP) | Oral | NDA; Fast Track, Orphan Drug | 08/29/2025 |
| lecanemab-irmb (Leqembi) SC | Eisai | Alzheimer's disease (mild; weekly maintenance dosing) | SC | BLA | 08/31/2025 |
| sodium pyruvate | Emphycorp | Idiopathic pulmonary fibrosis (IPF) | Intranasal | NDA | 09/12/2025 |
| apitegromab | Scholar Rock | Spinal muscular atrophy | IV | NDA; Fast Track, Orphan Drug, Priority Review, RPD | 09/22/2025 |
| pembrolizumab / hyaluronidase | Merck | All solid tumor indications as IV pembrolizumab | SC | BLA | 09/23/2025 |
| paltusotine | Crinetics | Acromegaly | Oral | NDA; Orphan Drug | 09/25/2025 |
| aficamten | Cytokinetics | Obstructive hypertrophic cardiomyopathy | Oral | NDA; Breakthrough Therapy, Orphan Drug | 09/26/2025 |
| telisotuzumab vedotin (Teliso-V) | Abbvie | NSCLC (locally advanced or metastatic, EGFR wild type, nonsquamous, c-Met protein overexpression) | IV | BLA; Breakthrough Therapy, seeking Accelerated Approval | 09/26/2025 |
| tolebrutinib | Sanofi | MS (non-relapsing secondary progressive) | Oral | NDA; Breakthrough Therapy, Priority Review | 09/28/2025 |
| riboflavin 5’-phosphate (Epioxa) | Glaukos | Keratoconus | Ophthalmic | NDA; Orphan Drug | 10/20/2025 |
| molgramostim | Savara | Acute pulmonary alveolar proteinosis | Inhaled | BLA; Breakthrough Therapy, Fast Track, Orphan Drug | Nov 2025–Mar 2026 |
| sibeprenlimab | Otsuka | IgA nephropathy (Berger's disease) | IV, SC | BLA; Breakthrough Therapy | Nov 2025–Mar 2026 |
| ziftomenib | Kura | AML (R/R, NPM 1 mutation) | Oral | NDA; Breakthrough Therapy, Fast Track, Orphan Drug | Nov 2025–Mar 2026 |
| plozasiran | Arrowhead | Familial chylomicronemia syndrome (FCS) | SC | NDA; Breakthrough Therapy, Fast Track, Orphan Drug | 11/18/2025 |
| lerodalcibep | LIB | LDL-C reduction (with ASCVD or high/very high risk for ASCVD); HeFH; HoFH | SC | BLA | 12/12/2025 |
| depemokimab | GlaxoSmithKline | Asthma (ages ≥ 12 years, type 2 inflammation); Chronic rhinosinusitis | SC | BLA | 12/16/2025 |
| fibrinogen | Grifols | Fibrinogen deficiency (acquired and congenital) | IV | BLA | 12/27/2025 |
| relacorilant | Corcept | Cushing's syndrome | Oral | NDA; Orphan Drug | 12/30/2025 |
| navepegritide | Ascendis | Achondroplasia (children) | SC | NDA; Orphan Drug | 03/31/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| ranibizumab port delivery system (Susvimo) | Genentech | Diabetic retinopathy | Intravitreal implant | sBLA | May–Jun 2025 |
| mepolizumab (Nucala) | GlaxoSmithKline | COPD (eosinophilic phenotype, add-on maintenance) | SC | sBLA | 05/07/2025 |
| belzutifan (Welireg) | Merck | Pheochromocytoma and paraganglioma (advanced, unresectable or metastatic, ages ≥ 12 years) | Oral | sNDA; Priority Review | 05/26/2025 |
| concizumab-mtci (Alhemo) | Novo Nordisk | Hemophilia A and B (without inhibitors) | SC | sBLA; Breakthrough Therapy | 05/31/2025 |
| dasatinib (Dasynoc) | Xspray | CML | Oral | sNDA; Orphan Drug | Jun–Oct 2025 |
| lenacapavir (Sunlenca) | Gilead | HIV-1 infection pre-exposure prophylaxis (PrEP) | Oral, SC | sNDA; Priority Review | 06/19/2025 |
| dupilumab (Dupixent) | Sanofi, Regeneron | Bullous pemphigoid | SC | sBLA; Orphan Drug, Priority Review | 06/20/2025 |
| pembrolizumab (Keytruda) | Merck | SCCHN (resectable locally advanced, as neoadjuvant followed by adjuvant treatment) | IV | sBLA; Priority Review | 06/23/2025 |
| emapalumab-lzsg (Gamifant) | Swedish Orphan Biovitrum | Hemophagocytic lymphohistiocytosis / macrophage activation syndrome (MAS) in Still's disease (inadequate response or intolerance to glucocorticoids, or recurrent MAS) | IV | sBLA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, RPD | 06/27/2025 |
| retifanlimab-dlwr (Zynyz) | Incyte | Anal cancer (squamous cell, advanced or metastatic) | IV | sBLA | Jul–Dec 2025 |
| tafasitamab-cxix (Monjuvi) | Incyte | Follicular lymphoma; Marginal zone lymphoma | IV | sBLA; Orphan Drug | Jul–Dec 2025 |
| upadacitinib (Rinvoq) | Abbvie | Giant cell arteritis | Oral | sNDA; Orphan Drug | 07/11/2025 |
| datopotamab deruxtecan-dlnk (Datroway) | Daiichi Sankyo, AstraZeneca | NSCLC (locally advanced or metastatic, nonsquamous, ≥ 2-line) | IV | sBLA; Breakthrough Therapy, Priority Review, seeking Accelerated Approval | 07/12/2025 |
| glofitamab-gxbm (Columvi) | Genentech | DLBCL (R/R, in combination with gemcitabine and oxaliplatin, prior therapy, ineligible for autologous SCT) | IV | sBLA | 07/20/2025 |
| darolutamide (Nubeqa) | Bayer | Prostate cancer (metastatic, hormone-sensitive, in combination with androgen deprivation therapy [ADT]) | Oral | sNDA; Fast Track | 07/25/2025 |
| pegcetacoplan (Empaveli) | Apellis | C3 Glomerulopathy (C3G) | SC | sNDA; Orphan Drug, Priority Review | 07/28/2025 |
| daratumumab / hyaluronidase-fihj (Darzalex Faspro) | Janssen | Multiple myeloma (newly diagnosed, in combination with D-VRd, autologous SCT deferred or ineligible); Multiple myeloma (high-risk, smoldering) | SC | sBLA | 07/30/2025 |
| lonapegsomatropin-tcgd (Skytrofa) | Ascendis | Growth hormone deficiency (adults) | SC | sBLA; Orphan Drug | 07/30/2025 |
| sparsentan (Filspari) | Travere | IgA nephropathy (Berger's disease) | Oral | sNDA; Orphan Drug, Priority Review | 08/25/2025 |
| sparsentan (Filspari) | Travere | Focal segmental glomerulosclerosis | Oral | sNDA; Orphan Drug | Sept–Dec 2025 |
| guselkumab (Tremfya) | Janssen | UC (SC induction regimen) | SC | sBLA | 09/22/2025 |
| obinutuzumab (Gazyva) | Genentech | Lupus nephritis | IV | sBLA; Breakthrough Therapy | October 2025 |
| fremanezumab-vfrm (Ajovy) | Teva | Migraine prevention (ages 6–17 years) | SC | sBLA; Fast Track | Oct-Dec 2025 |
| guselkumab (Tremfya) | Janssen | PSO (ages 6–17 years); PsA (ages 5–17 years) | SC | sBLA | 10/02/2025 |
| golimumab (Simponi) | Janssen | UC (ages 2–17 years) | SC | sBLA; Orphan Drug | 10/16/2025 |
| nusinersen (Spinraza) | Biogen | Spinal muscular atrophy (high dose) | Intrathecal | sNDA; Fast Track, Orphan Drug | 11/21/2025 |
| brexpiprazole (Rexulti) | Otsuka | PTSD (in combination with sertraline) | Oral | sNDA | Pending |
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| amlitelimab | Sanofi | Atopic dermatitis | SC | BLA | TBD |
| amniotic suspension allograft (ReNu) | Organogenesis | Osteoarthritis (knee) | Intra-articular | BLA; RMAT | TBD |
| anitocabtagene autoleucel | Arcellx, Gilead | Multiple myeloma | IV | BLA; Fast Track | TBD |
| apraglutide | Ironwood | Short bowel syndrome (parenteral support-dependent) | SC | NDA; Orphan Drug | TBD |
| astegolimab | Genentech, Amgen | COPD | IV | BLA | TBD |
| atacicept | Vera, Bristol Myers Squibb | IgA nephropathy (Berger's disease) | SC | BLA; Breakthrough Therapy, Orphan Drug | TBD |
| azetukalner | Xenon | Focal onset seizures | Oral | NDA | TBD |
| bepirovirsen | GlaxoSmithKline | HBV treatment | SC | NDA; Fast Track | TBD |
| bevacizumab (HLX04-O) | Henlius | Wet AMD | Intravitreal | BLA | TBD |
| blarcamesine | Anavex | Alzheimer's disease; Rett syndrome | Oral | NDA; Fast Track, RPD, may seek Accelerated Approval | TBD |
| botaretigene sparoparvovec | Janssen | Retinitis pigmentosa | Subretinal | BLA; Fast Track, Orphan Drug | TBD |
| buntanetap | Annovis | Alzheimer's disease; Parkinson's disease | Oral | NDA | TBD |
| camizestrant | AstraZeneca | Breast cancer (HR+/HER2-) | Oral | NDA | TBD |
| cetuximab sarotalocan | Rakuten | SCCHN | IV | BLA; Fast Track | TBD |
| cobolimab | GlaxoSmithKline | NSCLC (2nd-line) | IV | BLA | TBD |
| denecimig | Novo Nordisk | Hemophilia A | SC | BLA; Orphan Drug | TBD |
| depemokimab | GlaxoSmithKline | Eosinophilic granulomatosis with polyangiitis; Hypereosinophilic syndrome | SC | BLA | TBD |
| donaperminogene seltoplasmid | Helixmith | PAD | IM | BLA | TBD |
| fenebrutinib | Genentech | MS | Oral | NDA | TBD |
| fianlimab | Regeneron | Melanoma | IV | BLA; Fast Track | TBD |
| frexalimab | Sanofi | MS | IV, SC | BLA | TBD |
| garetosmab | Regeneron | Fibrodysplasia ossificans progressiva | IV | BLA; Fast Track, Orphan Drug | TBD |
| gedatolisib | Celcuity, Pfizer | Breast cancer (HR+/HER2-) | IV | NDA; Breakthrough Therapy, Fast Track | TBD |
| giredestrant | Genentech | Breast cancer (HR+/HER2-) | Oral | NDA; Fast Track | TBD |
| gold nanocrystal | Clene | ALS | Oral | NDA; Orphan Drug | TBD |
| govorestat | Applied | Sorbitol dehydrogenase (SORD) deficiency | Oral | NDA; Orphan Drug | TBD |
| GYM329/RG6237 | Genentech | Spinal muscular atrophy | SC | BLA | TBD |
| hydromethylthionine mesylate | Taurx | Alzheimer's disease (mild-moderate) | Oral | NDA | TBD |
| hydroxypropyl beta cyclodextrin | Cyclo | Niemann-Pick disease (type C) | IV | NDA; Fast Track, Orphan Drug, RPD | TBD |
| ianalumab | Novartis | Autoimmune hepatitis; ITP; Sjogren's syndrome | SC | BLA; Fast Track; Orphan Drug | TBD |
| icotrokinra | Genentech | PSO | Oral | NDA | TBD |
| imsidolimab | Anaptysbio | Generalized pustular psoriasis (GPP) | IV, SC | BLA; Orphan Drug | TBD |
| inaxaplin | Vertex | Focal segmental glomerulosclerosis | Oral | NDA; Breakthrough Therapy, may seek Accelerated Approval | TBD |
| iron sucrose / stannus protoporphyrin (RBT-1) | Renibus | Reduce risk of post-operative complications associated with cardiothoracic surgery | IV | NDA; Breakthrough Therapy, Fast Track | TBD |
| itepekimab | Regeneron, Sanofi | COPD | SC | BLA; Fast Track | TBD |
| itolizumab | Equillium | GVHD treatment | IV | BLA; Fast Track, Orphan Drug | TBD |
| ixoberogene soroparvovec | Adverum | Wet AMD | Intravitreal | BLA; Fast Track, RMAT | TBD |
| ketamine (NRX100) | Hope | Bipolar disorder (suicidal depression) | IV | 505(b)(2) NDA; Fast Track | TBD |
| lanifibranor | Inventiva | MASH | Oral | NDA; Breakthrough Therapy, Fast Track may seek Accelerated Approval | TBD |
| latozinemab | Alector, GlaxoSmithKline | Frontotemporal dementia | IV | BLA; Breakthrough Therapy, Fast Track, Orphan Drug | TBD |
| linerixibat | GlaxoSmithKline | Cholangitis pruritus | Oral | NDA; Orphan Drug | TBD |
| narsoplimab | Omeros | Transplant-associated thrombotic microangiopathy (TA-TMA) | IV, SC | BLA; Breakthrough Therapy, Orphan Drug | TBD |
| nemvaleukin alfa | Mural Oncology | Ovarian cancer (platinum-resistant, in combination with pembrolizumab) | IV | BLA; Fast Track | TBD |
| nerandomilast | Boehringer Ingelheim | Idiopathic pulmonary fibrosis | Oral | NDA; Breakthrough Therapy, Orphan Drug | TBD |
| nexiguran ziclumeran | Intellia, Regeneron | Transthyretin amyloid cardiomyopathy | IV | BLA; Orphan Drug, RMAT | TBD |
| nilotinib | Xspray | CML | Oral | NDA; Orphan Drug | TBD |
| nipocalimab | Janssen | Autoimmune hemolytic anemia | IV | BLA; Fast Track, Orphan Drug | TBD |
| NTLA-2002 | Intellia | HAE | IV | BLA; Orphan Drug, RMAT | TBD |
| obefazimod | Abivax | UC | Oral | NDA | TBD |
| OCU400 | Ocugen | Retinitis pigmentosa | Subretinal | BLA; Orphan Drug, RMAT | TBD |
| pabinafusp alfa | JCR | Mucopolysaccharidosis II (Hunter syndrome) | IV | BLA; Breakthrough Therapy, Fast Track, Orphan Drug | TBD |
| palazestrant | Olema | Breast cancer (HR+/HER2-) | Oral | NDA; Fast Track | TBD |
| pegadricase | Swedish Orphan Biovitrum | Gout | IV | BLA; Fast Track | TBD |
| pegargiminase | Polaris | Mesothelioma | IM | BLA; Fast Track, Orphan Drug | TBD |
| pelabresib | Novartis | Myelofibrosis | Oral | NDA; Fast Track, Orphan Drug | TBD |
| pelacarsen | Novartis | Dyslipidemia | SC | NDA; Fast Track | TBD |
| PL-9643 | Palatin | DED | Ophthalmic | NDA | TBD |
| potravitug | Memo | BK polyomavirus (BKV) infection | IV | BLA; Fast Track | TBD |
| recombinant human glutamic acid decarboxylase (rhGAD65) | Diamyd | T1DM (newly diagnosed, ages 12–28 years, HLA DR3-DQ2 haplotype) | Intralymphatic | BLA; Fast Track, Orphan Drug, may seek Accelerated Approval | TBD |
| REGN-5713-5714-5715 | Regeneron | Birch allergy | SC | BLA | TBD |
| remibrutinib | Novartis | MS; Spontaneous urticaria | Oral | NDA | TBD |
| resiniferatoxin | Grunenthal | Osteoarthritis (knee) | Intra-articular | NDA; Breakthrough Therapy | TBD |
| riliprubart | Sanofi | Chronic inflammatory demyelinating polyneuropathy (CIDP) | IV, SC | BLA; Orphan Drug | TBD |
| rusfertide | Takeda | Polycythemia vera | SC | NDA; Fast Track, Orphan Drug | TBD |
| savolitinib | AstraZeneca | NSCLC (MET+, 2nd-line) | Oral | NDA; Fast Track | TBD |
| sefaxersen | Roche | IgA nephropathy (Berger's disease) | SC | NDA | TBD |
| serplulimab | Henlius | SCLC | IV | BLA; Orphan Drug | TBD |
| suramin | PaxMedica | Human African sleeping sickness | IV | NDA; Orphan Drug | TBD |
| tebipenem pivoxil | GlaxoSmithKline | UTI (complicated) | Oral | NDA; Fast Track, QIDP | TBD |
| tiragolumab | Genentech | Esophageal cancer; NSCLC (unresectable, Stage 3, in combination with Tecentriq, 1st-line) | IV | BLA; Breakthrough Therapy, Fast Track, Orphan Drug | TBD |
| tividenofusp alfa | Denali | Mucopolysaccharidosis II (Hunter syndrome) | IV | BLA; Breakthrough Therapy, Fast Track, Orphan Drug, RPD | TBD |
| veligrotug | Viridian | Thyroid eye disease | IV | BLA | TBD |
| viaskin peanut | DBV | Peanut allergy | Transdermal | BLA; Breakthrough Therapy, Fast Track, may seek Accelerated Approval (ages 1–3 years) | TBD |
| VRDN-003 | Viridian | Thyroid eye disease | SC | BLA | TBD |
| zilganersen | Ionis | Alexander disease | Intrathecal | NDA; Fast Track | TBD |
| zipalertinib | Taiho | NSCLC (EGFR exon 20 insertion mutations) | Oral | NDA; Breakthrough Therapy | TBD |
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| alpelisib (Piqray) | Novartis | Breast cancer (HER2+) | Oral | sNDA | TBD |
| aminolevulinic acid (Ameluz) | Biofrontera | Superficial basal cell carcinoma | Topical | sNDA | TBD |
| atezolizumab (Tecentriq) | Genentech, Bristol Myers Squibb | Breast cancer (triple-negative); Prostate cancer (metastatic, castration-resistant, in combination with cabozantinib) | IV | sBLA | TBD |
| brolucizumab-dbll (Beovu) | Novartis | Diabetic retinopathy | Intravitreal | sBLA | TBD |
| cemiplimab-rwlc (Libtayo) | Regeneron, Sanofi | Melanoma | IV | sBLA; Fast Track | TBD |
| crovalimab-akkz (Piasky) | Genentech | Hemolytic uremic syndrome | IV, SC | sBLA | TBD |
| eplontersen (Wainua) | Ionis | Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary) | SC | sNDA; Fast Track, Orphan Drug | TBD |
| inebilizumab-cdon (Uplizna) | Amgen | Myasthenia gravis | IV | sBLA; Orphan Drug | TBD |
| iptacopan (Fabhalta) | Novartis | Hemolytic uremic syndrome | Oral | sNDA | TBD |
| mitapivat (Pyrukynd) | Agios | SCD | Oral | sNDA; Orphan Drug | TBD |
| mosunetuzumab-axgb (Lunsumio) | Genentech | DLBCL (2nd-line, in combination with polatuzumab vedotin-piiq) | SC | sBLA | TBD |
| nogapendekin alfa inbakicept-pmln (Anktiva) | Immunitybio | NSCLC | IV, SC | sBLA | TBD |
| obinutuzumab (Gazyva) | Genentech | Membranous nephropathy; NHL (indolent); SLE | IV | sBLA | TBD |
| olezarsen (Tryngolza) | Akcea | Dyslipidemia | SC | sNDA | TBD |
| ropeginterferon alfa-2b-njft (Besremi) | PharmaEssentia | Essential thrombocythemia | 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 |
| tezepelumab-ekko (Tezspire) | Amgen | Chronic rhinosinusitis; Nasal polyposis | SC | sBLA | TBD |
| tislelizumab-jsgr (Tevimbra) | Beigene | HCC | IV | sBLA; Orphan Drug | TBD |
| venetoclax (Venclexta) | Abbvie | Myelodysplastic syndrome | Oral | sNDA; Breakthrough Therapy, Orphan Drug | TBD |
| zanidatamab-hrii (Ziihera) | Jazz | Gastroesophageal adenocarcinoma (HER2+) | IV | sBLA; Fast Track, Orphan Drug | TBD |
Traditional
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| dihydroergotamine | Shin Nippon | Acute migraine | Intranasal | NDA | 04/30/2025 |
| hydrocortisone oral solution (ET-400) | Eton | Adrenocortical insufficiency | Oral | NDA | 05/28/2025 |
| acoltremon | Aerie | DED | Ophthalmic | NDA | 05/30/2025 |
| COVID-19 vaccine (mRNA-1283) | Moderna | COVID-19 prevention | IM | BLA; Priority Review | 05/30/2026 |
| elinzanetant | Bayer | Vasomotor symptoms associated with menopause | Oral | NDA | 08/01/2025 |
| oxylanthanum carbonate | Unicycive | Hyperphosphatemia (in patients with CKD on dialysis) | Oral | 505(b)(2) NDA | 06/28/2025 |
| semaglutide (NN9931) | Novo Nordisk | MASH | SC | NDA; Breakthrough Therapy | Aug 2025–Feb 2026 |
| telmisartan / amlodipine / indapamide | George Medicines | Hypertension (including initiation of treatment) | Oral | 505(b)(2) NDA | 08/06/2025 |
| aceclidine | Lenz | Presbyopia | Ophthalmic | NDA | 08/08/2025 |
| cyclobenzaprine | Tonix | Fibromyalgia | SL | 505(b)(2) NDA; Fast Track | 08/15/2025 |
| bumetanide | Corstasis | Edema (associated with CHF, liver disease, & kidney disease) | Intranasal | 505(b)(2) NDA | 09/14/2025 |
| seasonal influenza/COVID-19 vaccine (mRNA-1083) | Moderna | Seasonal influenza & COVID-19 immunization (ages ≥ 50 years) | IM | BLA; Fast Track | Oct–Dec 2025 |
| tradipitant | Vanda, Eli Lilly | Motion sickness | Oral | NDA | Oct–Dec 2025 |
| atropine | Sydnexis | Myopia (pediatric) | Ophthalmic | NDA | 10/23/2025 |
| milsaperidone (Bysanti) | Vanda | Bipolar I disorder; Schizophrenia | Oral | NDA | 03/31/2026 |
| epinephrine | Aquestive | Anaphylaxis | SL | 505(b)(2) NDA; Fast Track | 04/01/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| semaglutide (Ozempic) | Novo Nordisk | PAD (in patients with T2DM) | SC | sNDA | 2025 |
| dasiglucagon (Zegalogue) | Novo Nordisk | Congenital hyperinsulinism (dosing up to 3 weeks) | SC | sNDA; Orphan Drug, RPD | Apr–Jun 2025 |
| roflumilast (Zoryve) 0.3% foam | Arcutis, AstraZeneca | PSO (scalp and body, ages ≥ 12 years) | Topical | sNDA | 05/22/2025 |
| meningococcal vaccine (Menquadfi) | Sanofi | Meningococcal immunization (ages 6 weeks to 23 months) | IM | sBLA | 05/23/2025 |
| ruxolitinib (Opzelura) | Incyte | Atopic dermatitis (pediatrics) | Topical | sNDA | Jul–Oct 2025 |
| RSV vaccine (mRESVIA) | Moderna | RSV immunization (high-risk adults, ages 18-59 years) | IM | sBLA; Priority Review | 07/11/2025 |
| risperidone ER (Uzedy) | Teva | Bipolar I disorder (maintenance, adults) | SC | sNDA | Sept–Oct 2025 |
| mitapivat (Pyrukynd) | Agios | Thalassemia (alpha or beta, non-transfusion-dependent and transfusion-dependent) | Oral | sNDA; Orphan Drug | 09/07/2025 |
| finerenone (Kerendia) | Bayer | Chronic HF (LVEF ≥ 40% or preserved LVEF) | Oral | sNDA; Priority Review | 09/10/2025 |
| tirzepatide (Zepbound) | Eli Lilly | HFpEF (in patients with obesity) | SC | sNDA | 09/16/2025 |
| semaglutide (Wegovy) | Novo Nordisk | HFpEF (in patients with obesity) | SC | sNDA | Oct–Dec 2025 |
| lumateperone (Caplyta) | Janssen, Bristol Myers Squibb | MDD (adjunct to antidepressants, adults) | SC | sNDA | 10/03/2025 |
| roflumilast (Zoryve) 0.05% cream | Arcutis, AstraZeneca | Atopic dermatitis (ages 2–5 years) | Topical | sNDA | 10/13/2025 |
| semaglutide (Rybelsus) | Novo Nordisk | MACE risk reduction (in adults with T2DM and established CVD and/or CKD) | Oral | sNDA | 10/31/2025 |
| celecoxib (Elyxyb) ready-to-use oral solution | Scilex | Acute pain | Oral | sNDA | 01/21/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| aroxybutynin / atomoxetine | Apnimed | OSA | Oral | NDA; Fast Track | TBD |
| baclofen / naltrexone / sorbitol | Pharnex | Charcot-Marie-Tooth disease | Oral | NDA; Fast Track, Orphan Drug | TBD |
| basimglurant | Noema | Trigeminal neuralgia | Oral | NDA; Fast Track | TBD |
| brilaroxazine | Reviva | Schizophrenia | Oral | NDA | TBD |
| cagrilintide / semaglutide | Novo Nordisk | Obesity; T2DM | SC | NDA | TBD |
| camlipixant | GlaxoSmithKline | Chronic cough | Oral | NDA | TBD |
| cebranopadol | Tris | Acute pain | Oral | NDA | TBD |
| centanafadine | Otsuka | ADHD | Oral | NDA | TBD |
| cytisinicline | Achieve | Smoking cessation | Oral | NDA; Breakthrough Therapy | TBD |
| d-cycloserine / lurasidone | Alvogen | Bipolar disorder (suicidal) | Oral | NDA; Breakthrough Therapy, Fast Track | TBD |
| dipalmitoyl hydroxyproline (QRX003) | Quoin | Congenital ichthyosis | Topical | NDA | TBD |
| ensitrelvir | Shionogi | COVID-19 post-exposure prophylaxis | Oral | NDA; Fast Track | TBD |
| esreboxetine | Axsome, Pfizer | Fibromyalgia | Oral | NDA | TBD |
| gepotidacin | GlaxoSmithKline | Urogenital gonorrhea | Oral | NDA; QIDP | TBD |
| Lyme disease vaccine | Valneva, Pfizer | Lyme disease immunization | IM | BLA; Fast Track | TBD |
| navacaprant | Neumora | MDD | Oral | NDA | TBD |
| obicetrapib | New Amsterdam | Dyslipidemia | Oral | NDA | TBD |
| orforglipron | Eli Lilly | T2DM; Obesity; OSA (in patients with obesity) | Oral | NDA | TBD |
| oveporexton | Takeda | Narcolepsy | Oral | NDA; Breakthrough Therapy | TBD |
| purified vero rabies vaccine (SP0087) | Sanofi | Rabies (post-exposure treatment) | IM | BLA | TBD |
| quadrivalent influenza mRNA vaccine (mRNA-1010) | Moderna | Seasonal influenza vaccination | IM | BLA | TBD |
| ralinepag | United Therapeutics | PAH | Oral | NDA; Orphan Drug | TBD |
| retatrutide | Eli Lilly | Obesity; T2DM | SC | NDA | TBD |
| RSV live attenuated vaccine (SP0125) | Sanofi | RSV immunization | Intranasal | BLA | TBD |
| semaglutide / insulin icodec | Novo Nordisk | T2DM | SC | NDA | TBD |
| survodutide | Zealand, Boehringer Ingelheim | MASH; Obesity | SC | NDA | TBD |
| tavapadon | Abbvie | Parkinson's disease | Oral | NDA | TBD |
| ulixacaltamide | Praxis | Essential tremor | Oral | NDA | TBD |
| valiltramiprosate | Alzheon | Alzheimer's disease | Oral | NDA; Fast Track | TBD |
| venglustat | Sanofi | Fabry's disease; Gaucher's disease | Oral | NDA; Fast Track, Orphan Drug | TBD |
| vidofludimus | Immunic | MS | Oral | NDA | TBD |
| zoliflodacin | Innoviva | Gonorrhea (uncomplicated) | Oral | NDA; Fast Track, QIDP | TBD |
| Name | Manufacturer | Clinical use | Dosage form | Development status | FDA decision |
|---|---|---|---|---|---|
| dextromethorphan / bupropion (Auvelity) | Axsome | Alzheimer’s disease-related neuropsychiatric symptoms | Oral | sNDA; Breakthrough Therapy, Fast Track | TBD |
| finerenone (Kerendia) | Bayer | Chronic HFrEF | Oral | sNDA | TBD |
| semaglutide (Ozempic) | Novo Nordisk | Alzheimer's disease; Diabetic retinopathy | SC | sNDA | TBD |
| semaglutide (Rybelsus) | Novo Nordisk | Alzheimer's disease; Obesity | Oral | sNDA | TBD |
| semaglutide (Wegovy) | Novo Nordisk | Osteoarthritis (of knee, with obesity) | SC | sNDA | TBD |
| treprostinil sodium (Tyvaso) | Ferrer, GlaxoSmithKline | Idiopathic pulmonary fibrosis | Inhaled | sNDA; Orphan Drug | TBD |
| Name | Manufacturer | Clinical use | Dosage form | Development status |
|---|---|---|---|---|
| camrelizumab | Jiangsu Hengrui | HCC (unresectable, 1st-line, in combination with rivoceranib) | IV | CRL |
| condoliase | Ferring | Radicular leg pain associated with lumbar disc herniation | Intervertebral disc injection | CRL |
| etripamil (Cardamyst) | Milestone | Paroxysmal supraventricular tachycardia | Intranasal | CRL |
| reproxalap | Aldeyra | DED | Ophthalmic | CRL |
| rivoceranib | Elevar | HCC (unresectable, 1st-line, in combination with camrelizumab) | Oral | CRL |
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
AChR Acetylcholine Receptor
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
APOE4 Apolipoprotein E4 gene variant
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
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
c-MET C-Mesenchymal-Epithelial Transition
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
CFTR Cystic Fibrosis Transmembrane Conductance Regulator
CHF Congestive Heart Failure
CI Confidence Interval
CIS Carcinoma in Situ
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
CT scan Computed Tomography scan
CTLA-4 Cytotoxic T-Lymphocyte-Associated Protein 4
CV Cardiovascular
CVD Cardiovascular Disease
CYP3A4 Cytochrome P-450 3A4
CYP450 Cytochrome P-450
D-VRd Bortezomib, Lenalidomide and Dexamethasone
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
ERK Extracellular signal-Regulated Kinase
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
GALV-GP-R⁻ Gibbon Ape Leukemia Virus GlycoProtein
G-CSF Granulocyte Colony Stimulating Factor
GERD Gastroesophageal Reflux Disease
GGT Gamma-Glutamyl Transferase
GI Gastrointestinal
GIST Gastrointestinal Stromal Tumor
GLP-1 Glucagon-Like Peptide-1 Receptor Agonist
GM-CSF Granulocyte-Macrophage Colony Stimulating Factor
GVHD Graft Versus Host Disease
H Half
H3 K27M Histone 3 Lysine 27-to-Methionine
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 Hormone Receptor
HR- Hormone Receptor-negative
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
LAG-3 Lymphocyte-Activation Gene 3
LAMA Long-Acting Muscarinic Antagonist
LDL-C Low-Density Lipoprotein Cholesterol
LPAD Limited Population Pathway for Antibacterial and Antifungal Drugs
LRP4 Low density Lipoprotein-Related Protein 4
LSM Least Square Mean
LVEF Left Ventricular Ejection Fraction
mAb Monoclonal Antibody
MACE Major Adverse Cardiovascular Events
MADRS Montgomery – Åsberg Depression Rating Scale
MAPK Mitogen-Activated Protein Kinase
MASH Metabolic Dysfunction-Associated Steatohepatitis
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
MET Mesenchymal Epithelial Transition
MI Myocardial Infarction
mITT modified Intent-To-Treat
MRI Magnetic Resonance Imaging
mRNA messenger Ribonucleic Acid
MRSA Methicillin-Resistant Staphylococcus Aureus
MS Multiple Sclerosis
MSI-H Microsatellite Instability-High
mTOR mechanistic Target of Rapamycin
MuSK Muscle-Specific tyrosine Kinase
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-Hodg
NIH National Institutes of Health
NPM 1 Nucleophosmin 1
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
OSA Obstructive Sleep Apnea
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
RAF Rapidly Accelerated Fibrosarcoma
RARA Retinoic Acid Receptor alpha
RAS Ras Protein Superfamily
RBC Red Blood Cell
RCC Renal Cell Carcinoma
RECIST Response Evaluation Criteria in Solid Tumors
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