Quarterly Drug Pipeline: July 2025 - Prime Therapeutics
Quarterly Drug Pipeline: July 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.
The 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 for select agents.
Reflection
Thus far in 2025, the agency has approved 20 novel drugs, close to the number of approvals about the same time last year. Of note, most of the approvals so far in 2025 use at least one of the FDA’s expedited approval programs.
Some of the noteworthy approvals in the second quarter of 2025 include a new option for ovarian cancer, an autologous cell sheet-based gene therapy for a rare genetic skin disorder, a new ophthalmic for dry eye disease, an oral agent for Berger’s disease (a kidney immune disease) and a new monoclonal antibody for respiratory syncytial virus (RSV) prophylaxis in infants.
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 (68%) and for orphan drugs (37%) continue to grow for agents with applications submitted to the FDA. Five therapies, all for Orphan conditions, 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 third quarter 2025 include:
- First SC version of Keytruda in the oncology setting
- First treatment for non-relapsing secondary progressive multiple sclerosis
- An off-the shelf gene therapy for a rare genetic condition caused by human papilloma virus
- A first-in-class agent for bronchiectasis, a chronic inflammatory lung condition
- A new mechanism of action for spinal muscular atrophy
A hot topic in the drug pipeline that continues to garner significant attention is GLP-1s. Visit Prime’s GLP-1 Pipeline Update to learn more. Select highlights include:
- Injectable semaglutide (Wegovy) indication for metabolic dysfunction-associated steatohepatitis (MASH) – FDA decision Aug. 18, 2025
- Oral semaglutide (Wegovy) formulation for weight loss – FDA decision in fourth quarter 2025
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
Samantha Decker, PharmD
Medical Pharmacy Clinical Pharmacist
Natalee Felten, PharmD, BCPS
Medical Pharmacy Clinical Pharmacist
Andrea Henry, PharmD, MBA, BCPS
Drug Information Pharmacist Principal
Katie Lockhart
Clinical Outcomes, Analytics, and Research
Danny Melson
Data Scientist Principal
Olivia Pane, PharmD, CDCES
Drug Information Pharmacist
Devon Trumbower, PharmD, BCPS
Medical Pharmacy Clinical Pharmacist
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
apitegromab IV
Proposed indications
Spinal muscular atrophy (SMA)
Clinical overview
Mechanism of action
Apitegromab is a selective latent myostatin inhibitor designed to improve motor function in people living with SMA.
The double-blind, placebo-controlled, Phase 3 SAPPHIRE (NCT05156320) trial evaluated apitegromab for the treatment of non-ambulatory patients with type 2 or type 3 SMA who were receiving current SOC (nusinersen or risdiplam). The study enrolled 156 patients 2–12 years of age. Patients were randomized 1:1:1 to receive apitegromab 10 mg/kg, apitegromab 20 mg/kg or placebo added to SOC therapy. The trial reported that apitegromab achieved the primary endpoint of statistically significant improvement compared to placebo in motor function from baseline to month 12, as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) (LSM difference: 1.8 points [p=0.0192] with both doses combined, 1.4 points nonsignificant p=0.1149] with apitegromab 20 mg/kg and 2.2 points [nominal p=0.0121] with apitegromab 10 mg/kg). Approximately 30.4% of patients in the apitegromab groups achieved a clinically meaningful (≥ 3-point) improvement in HFMSE score compared to 12.5% in the placebo group. Apitegromab was well tolerated. Pneumonia and dehydration were serious adverse events reported in the apitegromab group but not the placebo group. Similar efficacy and safety results were reported in an exploratory population of patients 13–21 years of age with type 2 or type 3 SMA who received apitegromab 20 mg/kg (LSM difference compared to placebo in change in HFMSE, 1.8 points).
The open-label, Phase 2 TOPAZ (NCT03921528) trial evaluated apitegromab in 58 patients with type 2 or type 3 SMA in three study cohorts. Cohort 1 stratified ambulatory patients 5–21 years of age into two arms (apitegromab 20 mg/kg alone or in combination with nusinersen), cohort 2 evaluated apitegromab 20 mg/kg combined with nusinersen in non-ambulatory patients 5–21 years of age and cohort 3 randomized patients ≥ 2 years of age to apitegromab 2 mg/kg or 20 mg/kg in combination with nusinersen in a blinded manner. At month 12, the mean change from baseline in the primary endpoint of HFMSE score in cohort 1 was -0.3 points in patients who received apitegromab plus nusinersen and -0.4 points in patients who received apitegromab alone. The mean change in HFMSE in cohort 2 was +0.6 points. In cohort 3, the mean change in HFMSE was +5.3 and +7.1 points for the 2 mg/kg and 20 mg/kg arms, respectively.
Dosage and administration
In the SAPPHIRE and TOPAZ trials, apitegromab was administered via IV infusion every 4 weeks for 12 months.
Place in therapy
Spinal muscular atrophy (SMA) is a rare, debilitating, hereditary disease characterized by progressive motor function decline and muscular atrophy that spares cognitive abilities. Most cases are caused by mutations in the survival motor neuron (SMN)-1 and -2 genes, leading to a lack of SMN protein that is responsible for normal motor neuron function. Several types of SMA have been identified and vary by age of onset, severity and prognosis. Complications depend on the degree of muscle weakness and include respiratory infections, scoliosis and joint contractures.
Approximately 25,000 people in the U.S. have SMA. Type 1 SMA is the most common phenotype, accounting for about 60% of cases. Onset for type 1 occurs soon after birth to 6 months of age. Patients with type 1 typically do not achieve motor milestones and require nutritional support and possibly respiratory support by 12 months of age. SMA type 2 accounts for about 20% of SMA cases. Symptoms of type 2 present between 6 to 18 months of age and lead to an inability to stand or walk without assistance. Type 3 SMA accounts for about 20% to 30% of cases. The onset of type 3 symptoms occurs after 18 months of age. Affected individuals achieve independent mobility but lose the ability to stand and walk over time. Type 4 accounts for < 5% of SMA cases. It is a milder form of SMA with onset in adulthood.
There is no cure for SMA. Disease modifying pharmacological agents include nusinersen (Spinraza) administered via lumbar puncture and oral risdiplam (Evrysdi), both of which increase the production of the SMN protein. The gene therapy onasemnogene abeparvovec-xioi (Zolgensma) delivers a functional SMN1 gene and is reserved for patients under 2 years of age with type 1 SMA.
If approved, apitegromab will provide a new mechanism of action to treat SMA. It improves muscle strength by inhibiting myostatin, a protein associated with muscle wasting. The SAPPHIRE and TOPAZ trials demonstrated enhanced motor function when apitegromab was added to SOC in non-ambulatory patients as compared to placebo. Data from the TOPAZ trial also suggest that greater gains may be achieved in younger patients with a shorter duration of SMA disease.
ICER released a revised evidence report assessing the comparative clinical effectiveness and value of treatments for SMA, including apitegromab. ICER stated that the addition of apitegromab to SOC in patients 2–12 years of age with type 2 or type 3 SMA likely provides comparable or incremental benefits compared with no additional therapy, but that there is some possibility of substantial benefit with long-term use as well as some possibility of net harm. The final evidence report will be reviewed by the Midwest Comparative Effectiveness Public Advisory Council (CEPAC) on Aug. 1, 2025.
FDA approval timeline
Sept. 22, 2025
FDA designations: Fast Track, Orphan Drug, Priority Review, RPD
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $37 |
| 2026 | $173 |
| 2027 | $277 |
| 2028 | $369 |
| 2029 | $457 |
clemidsogene lanparvovec (RGX-121) intracisternal
Manufacturer: Nippon Shinyaku, Regenxbio
Proposed indications
Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome
Clinical overview
Mechanism of action
RGX-121 is a gene therapy with an adeno-associated virus 9 (AAV9) vector that delivers the iduronate-2-sulfatase (IDS) gene to the CNS to provide a continuous source of iduronate-2-sulfatase (I2S). A lack of I2S results in accumulation of mucopolysaccharides in many organ systems in the body, including the CNS.
Clinical trials
The two-part, 104-week, open-label, Phase 1/2/3 CAMPSIITE (NCT03566043) trial evaluated RGX-121 in boys 4 months to 5 years of age with neuronopathic MPS II. The study allowed SOC enzyme replacement therapy (ERT) which could be discontinued after 24 weeks. Part 1 of the study determined the pivotal dose of RGX-121 of 2.9 x 10¹¹ genome copies/g brain mass, which was administered to 10 patients in Part 2 of the study. Neuronopathic forms of MPS II exhibit elevated concentrations of the glycosaminoglycans (GAG) heparan sulfate (HS) in the brain that leads to CNS abnormalities and neurocognitive impairment. In the study, CSF levels of D2S6, an HS disaccharide, were used to monitor I2S enzyme activity as a surrogate endpoint to predict clinical benefit. A January 2024 interim report found that eight of the 10 patients who received the pivotal dose of RGX-121 in Part 2 achieved reductions in CSF D2S6 to below the maximum attenuated levels (100 ng/mL) at week 16; the remaining two patients also exhibited robust reductions in CSF D2S6 (55%, 85%). A total of 14 patients in both parts of the study received the pivotal dose of RGX-121, among whom an 86% reduction in the CSF D2S6 level was reported. Notably, dose-dependent reductions in CSF D2S6 were demonstrated for up to 4 years among patients enrolled in Part 1 of the study. In addition, most participants (at all doses) in Part 1 demonstrated continued acquisition or stability of skills. RGX-121 was well tolerated.
Dosage and administration
In the CAMPSIITE trial, a one-time dose of RGX-121 was administered intracisternally (into the cisterna magna) using guided imagery. The pivotal dose was 2.9 x 10¹¹ genome copies/g brain mass.
Place in therapy
MPS II is a rare, genetic, metabolic condition that is almost exclusively diagnosed in boys, although cases in girls have been reported. It affects approximately 1 in 100,000 to 170,000 male births. In MPS II, gene mutations result in a lack of the I2S enzyme leading to an accumulation of mucopolysaccharides that causes tissue damage in many organ systems in the body. There are two MPS II phenotypes, neuropathic and non-neuropathic, based on neurologic impact and rate of progression. Patients with the more severe type, neuronopathic MPS II, primarily experience neurologic manifestations, including cognitive decline. Other organ systems affected include the airway, heart, liver, spleen, eyes, skin, bones and joints. Onset of symptoms of neuronopathic MPS II occurs around 2 to 4 years of age. Death due to upper airway disease or cardiovascular failure occurs between 10 to 20 years of age. Patients with non-neuropathic MPS II experience little if any CNS involvement, although other organ systems are affected. Diagnosis is often made after 10 years of age and affected individuals typically have normal intellectual development. Premature death after the age of 50 years can occur due to respiratory or cardiac complications.
Pharmacologic treatment of MPS II consists of weekly IV infusions of the ERT idursulfase (Elaprase), a recombinant human I2S. In clinical trials, it led to significant increases in 6MWT and FVC (combined endpoint), measures of cardiac and respiratory function, respectively, compared to placebo at 53 weeks. HSCT may also provide sufficient enzyme activity to slow or stop the progression of the disease but is associated with an elevated risk of morbidity and mortality, and its long-term efficacy is uncertain.
If approved, RGX-121 will be the first gene therapy to treat MPS II. RGX-121 is administered as a one-time intracisternal injection to deliver a functional copy of the I2S enzyme directly to the brain. Interim clinical trial data demonstrated RGX-121 led to reductions in D2S6 in the CSF and may lead to attainment or stabilization of developmental skills. Conversely, the currently available ERT Elaprase does not deliver I2S across the blood-brain-barrier and therefore, Elaprase does not alleviate the neurodegeneration experienced by patients with MPS II.
Denali is seeking Accelerated Approval for the CNS-penetrant ERT tividenofusp alfa for the treatment of MPS II. The FDA granted a Priority Review with a decision expected by early January 2026.
FDA approval timeline
FDA designations: Fast Track, Orphan Drug, Priority Review, RPD, RMAT, seeking Accelerated Approval
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $7 |
| 2026 | $14 |
| 2027 | $22 |
| 2028 | $29 |
| 2029 | $37 |
etuvetidigene autotemcel IV
Manufacturer: Fondazione Telethon
Proposed indications
Wiskott-Aldrich syndrome (WAS)Clinical overview
Mechanism of actionMutations in the WAS gene lead to a deficiency in the WAS protein (WASP), which is essential for proper functioning of certain blood cells. Immune cells (T cells and B cells) that lack WASP have compromised immunological synapse formation, cell migration and cytotoxicity. Etuvetidigene autotemcel is comprised of autologous CD34-positive hematopoietic stem and progenitor cells modified with a lentiviral vector with a functional copy of the WAS gene, which is delivered to the bone marrow to restore WASP expression.
Clinical trial(s)
Etuvetidigene autotemcel was studied in boys with WAS who do not have a suitable matched donor for allogeneic HSCT. Complete data from eight boys from the open-label, single-arm Phase 1/2 TIGET-WAS (NCT01515462) clinical trial and nine boys who received etuvetidigene autotemcel as part of an expanded access program (EAP) were analyzed. Data revealed WASP expression was restored 3 months after administration of etuvetidigene autotemcel, as evidenced by increases in the fraction of WASP-positive lymphocytes and platelets, and response was maintained throughout the study follow-up. As platelet counts improved, frequency and severity of bleeding events declined. All subjects achieved platelet transfusion-independence and absence of severe bleeding by 9 months after the etuvetidigene autotemcel dose and all evaluable patients discontinued immunoglobulin supplementation at a median of 0.9 years (range, 0.2–5 years) after the dose. At a median follow-up of 8.4 years (trial range, 5.2–10.5 years; EAP range, 0.4–4.9 years), overall survival was 94% – one patient in the EAP group died 4.5 months after the etuvetidigene autotemcel dose due to an unrelated underlying condition. No TEAEs related to etuvetidigene autotemcel have been reported; however, mild to moderate TEAEs due to conditioning therapy were reported in most patients.
An ongoing, open-label, single-arm Phase 3 trial (NCT03837483) is evaluating a cryopreserved formulation of etuvetidigene autotemcel in patients with WAS who do not have a matching donor for HSCT. Study completion is anticipated in 2027 (primary completion, September 2025).
Dosage and administration
In clinical trials, etuvetidigene autotemcel was administered via a single IV infusion at a target dose of 5–10 × 10⁶ CD34+ cells per kg, with an acceptable range of 2–20 × 10⁶ cells per kg. All subjects received rituximab and reduced-intensity conditioning with busulfan and fludarabine prior to etuvetidigene autotemcel treatment. Patients who participated in the clinical trial were hospitalized for a median of 52 days (range, 36–82 days).
Place in therapy
WAS is a rare, X-linked, genetic disorder characterized by thrombocytopenia, eczema, immunodeficiency, and increased risk of autoimmunity and lymphoma/leukemia. It is estimated that fewer than 5,000 boys in the U.S. have the condition. WAS is caused by mutations in the WAS gene leading to a decreased ability of immune cells to respond to the environment, fight infection and form functional platelets.
Current treatment options are supportive and include administration of immunoglobulins, platelet transfusions or eltrombopag (Alvaiz, Promacta) to increase platelet counts, antibiotic and antiviral to prevent infection, topical products to manage eczema, and medications (e.g., rituximab) to manage autoimmunity. HSCT, preferably within the first 2 years of life, is the SOC for WAS. It is a potential curative treatment if an adequate donor is available. Without HSCT, life expectancy of patients with WAS is approximately 15 years.
If approved, etuvetidigene autotemcel will be the first gene therapy to treat WAS in patients who are eligible for allogeneic HSCT but do not have a matching donor. Data from the clinical trial and the EAP group demonstrate etuvetidigene autotemcel restores WASP expression, reduces the need for platelet transfusions and immunoglobulin supplementation and improves overall survival.
FDA approval timeline
November 2025–March 2026 FDA designations: Orphan Drug, RPD, RMAT
Financial forecast (reported in millions)
The financial forecast for etuvetidigene autotemcel is not currently available.relacorilant oral
Manufacturer: Corcept
Proposed indications
Cushing syndrome
Clinical overview
Mechanism of action
Relacorilant is a selective glucocorticoid receptor (GR) modulator. It competes with cortisol for binding to the GR to modulate cortisol activity.
Clinical trials
Relacorilant was evaluated in the placebo-controlled, 2-part Phase 3 GRACE (NCT03697109) trial in adults with endogenous Cushing syndrome and uncontrolled hypertension and/or hyperglycemia (T2DM or impaired glucose tolerance [IGT]). During the open-label period, all 152 enrolled patients received treatment with relacorilant. Among patients with hypertension, relacorilant led to significant improvements from baseline in SBP (LSM change, -7.9 mm Hg; p<0.0001) and DBP (LSM change, -5.1 mm Hg; p<0.0001) at 22 weeks. Among all patients with hyperglycemia, significant improvement from baseline in glucose metabolism (glucose area under the curve [AUCglucose]) was seen with relacorilant (LSM change, -2.7 h*mmol/L, p<0.0001) at week 22. Of the 152 patients enrolled, 62.5% completed the open-label period of the study, 66.7% (n=44) achieved hypertension control and 57.5% (n=42) achieved hyperglycemia control. At week 22, patients (n=62) who met the predefined hyperglycemia and/or hypertension response criteria entered the randomized withdrawal phase and were randomized to continue relacorilant or switch to placebo for 12 weeks. Patients who switched to placebo were 83% more likely to lose blood pressure control compared to those who continued relacorilant (odds ratio, 0.17; p=0.02). In addition, patients who switched to placebo experienced significant increases in AUCglucose and HbA1c compared to those who continued relacorilant. Relacorilant was well tolerated and was not associated with hypokalemia, endometrial hypertrophy or related vaginal bleeding or adrenal insufficiency.
The randomized, double-blind, placebo-controlled Phase 3 GRADIENT (NCT04308590) trial evaluated relacorilant in 137 adults with cortisol-secreting adrenal adenoma or hyperplasia associated with T2DM, IGT and/or uncontrolled systolic hypertension. There was no significant difference between relacorilant and placebo in the primary endpoint of mean change from baseline in SBP based on 24-hour ambulatory blood pressure monitoring (ABPM) (-6.6 versus -2.1 mm Hg, respectively; p=not significant [NS]) at 22 weeks. In addition, no significant change from baseline in DBP were reported with relacorilant or placebo (-4.1 versus -1.7 mm Hg, respectively; p=NS). Among patients with hyperglycemia, significant improvements from baseline to week 22 were observed regarding fasting glucose, HbA1c and AUCglucose, with significant differences compared to placebo for each parameter (LSM difference, -22.2 mg/dL [p=0.0022], -0.3% [p=0.0188], -2.6 h*mmoll/L [p=0.0459], respectively). TEAEs reported (≥ 20%) with relacorilant included back pain, fatigue and upper abdominal pain.
Dosage and administration
In clinical trials, once-daily oral relacorilant was initiated at a dose of 100 mg and titrated to 400 mg.
Place in therapy
Cushing syndrome is also known as endogenous hypercortisolism. Cortisol is a glucocorticoid released by the adrenal gland that regulates the body’s stress response and plays a pivotal role in metabolism. The persistently elevated cortisol activity in Cushing syndrome is often associated with HTN and hyperglycemia. Most cases of endogenous Cushing syndrome are caused by adrenocorticotropin (ACTH)-secreting pituitary adenomas. Other causes include ectopic ACTH syndrome and adrenal tumors. Cushing syndrome occurs in an estimated 10 to 15 people per million people each year. Although it can occur at any age, it is most common in adults between the ages of 20 and 50 years and affects women more frequently than men.
Treatment for Cushing syndrome depends on the specific cause of cortisol excess and may include surgery, radiation and chemotherapy (mitotane [Lysodren]). Use of cortisol-inhibiting drugs is often required when surgery is delayed, contraindicated or unsuccessful, or for tumor recurrence. FDA-approved medications for Cushing syndrome include oral agents – the cortisol receptor blocker mifepristone (Korlym and generics), cortisol synthesis inhibitors osilodrostat (Isturisa) and levoketoconazole (Recorlev) – and the SC-administered somatostatin analog pasireotide diaspartate (Signifor) (for rapid normalization of cortisol). Off-label use of ketoconazole, metyrapone, cabergoline and etomidate have been reported in the literature.
If approved, relacorilant may be an alternative to once-daily mifepristone to normalize cortisol action in patients with Cushing syndrome. It differentiates itself from mifepristone, particularly in females, as it has no affinity for the progesterone receptor; therefore, it does not cause antiprogesterone side effects including endometrial hypertrophy, irregular vaginal bleeding and risk of pregnancy termination.
In addition, Corcept recently submitted relacorilant to the FDA for the treatment of platinum-resistant ovarian cancer. The FDA decision is expected to occur by July 2026.
FDA approval timeline
FDA designations: Orphan Drug
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $0 |
| 2026 | $625 |
| 2027 | $1,181 |
| 2028 | $1,514 |
| 2029 | $1,774 |
semaglutide (Wegovy) SC
Proposed indications
Metabolic dysfunction-associated steatohepatitis (MASH), formerly known as non-alcoholic steatohepatitis (NASH)
Clinical overview
Mechanism of action
Semaglutide is a glucagon-like peptide-1 (GLP-1) receptor agonist.
Clinical trial(s)
The ongoing two-part, randomized, double-blind, placebo-controlled Phase 3 ESSENCE (NCT04822181) trial is evaluating once-weekly injectable semaglutide 2.4 mg for the treatment of adults with biopsy-confirmed MASH and fibrosis stage F2/F3 (significant to advanced fibrosis without cirrhosis). The primary outcome is focused on histologic events for Part 1 of the study and clinical events for Part 2. Among 800 patients in the planned interim analysis for Part 1, the mean BMI was 34.6 kg/m² and 55.5% of patients had T2DM at baseline. The Part 1 analysis revealed that significantly more patients in the semaglutide group achieved the primary endpoint of resolution of steatohepatitis with no worsening of fibrosis compared to those in the placebo group (62.9% versus 34.1%, respectively; p<0.0001) at 72 weeks. Likewise, significantly more patients who received semaglutide exhibited improvement in liver fibrosis with no worsening of steatohepatitis compared to those who received placebo (36.8% versus 22.4%, respectively; p<0.0001). Improvements in body weight and cardiometabolic parameters were also reported. GI adverse events occurred more often in the semaglutide group than in the placebo group. The incidence of serious adverse events was similar in both groups. Longer‐term data (total duration 240 weeks) will be used for the Part 2 analysis to evaluate the effect of semaglutide on cirrhosis-free survival. The ESSENCE trial is estimated to be completed in April 2029.
Dosage and administration
In the ESSENCE trial, the semaglutide dose is titrated over 16 weeks to a target dose of 2.4 mg administered once weekly by SC injection.
Place in therapy
MASH is an advanced form of metabolic-associated fatty liver disease (MAFLD), formerly known as non-alcoholic fatty liver disease (NAFLD). NIH estimates that 24% of adults in the U.S. have MAFLD and approximately 1.5% to 6.5% have MASH. MASH is characterized by accumulation of fat in the liver (steatosis) resulting in inflammation and hepatocyte injury (ballooning), with or without fibrosis, and ultimately cirrhosis. MASH is a leading cause of liver cancer and the second most common indication for liver transplantation. The condition is closely associated with metabolic disorders such as T2DM, obesity, metabolic syndrome and dyslipidemia. CVD is also closely associated with MASH and is a leading cause of death among patients with MASH, particularly in those with fibrosis stage ≥ 2. MASH can occur at any age but is typically diagnosed between the ages of 40 and 60 years, when irreversible liver damage becomes evident. Moreover, incidence of MASH is rising in pediatrics, due in part to an increase in childhood obesity.
Lifestyle management (e.g., weight loss, exercise, alcohol avoidance) as well as management of diabetes, hyperlipidemia and hypertension are important elements for the treatment of MASH. Resmetirom (Rezdiffra), an oral thyroid hormone receptor-beta (THR-beta) agonist, is the only FDA-approved drug for the condition. It received Accelerated Approval in 2024 for use in combination with diet and exercise in adults with noncirrhotic MASH with stages F2/F3 fibrosis based on surrogate endpoints of improvement of MASH and liver fibrosis, which are reasonably likely to predict clinical benefit in patients with MASH.
Semaglutide is currently FDA-approved for the treatment of T2DM (brand Rybelsus, Ozempic) and obesity or overweight (brand Wegovy). While a head-to-head trial comparing resmetirom and semaglutide for MASH is not available, indirect comparisons of the pivotal trials appear to show similar improvement in steatohepatitis and liver fibrosis. Resmetirom uses intrahepatic mechanisms to improve MASH by regulating fat synthesis, fatty acid oxidation, cholesterol metabolism and mitochondrial function and by reducing inflammation and fibrosis. Semaglutide affects metabolism using extrahepatic mechanisms. These agents have the potential to be used as complementary medicines in patients with MASH.
FDA approval timeline
Aug. 18, 2025
FDA designations: Breakthrough Therapy, Priority Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales estimated for MASH |
|---|---|
| 2025 | $0 |
| 2026 | $204 |
| 2027 | $642 |
| 2028 | $844 |
| 2029 | $1,015 |
semaglutide (Wegovy) Oral
Proposed indications
Chronic weight management in adults with obesity or overweight with at least one weight-related comorbid condition
To reduce the risk of major adverse cardiovascular events (MACE) in adults with established cardiovascular disease (CVD) and either obesity or overweight
Clinical overview
Mechanism of action
Semaglutide is a glucagon-like peptide-1 (GLP-1) receptor agonist.
Clinical trial(s)
The double-blind, placebo-controlled, Phase 3b OASIS-4 (NCT05564117) trial evaluated oral semaglutide 25 mg in 307 adults with obesity (BMI ≥ 30 kg/m²) or overweight (BMI ≥ 27 kg/m²) with one or more comorbidities. Patients with T2DM were excluded. Enrolled patients were randomized to once-daily oral semaglutide 25 mg or placebo, as an adjunct to lifestyle intervention, for 64 weeks. Baseline average bodyweight was 105.9 kg (233 lbs). After 64 weeks of treatment, mean weight loss was 13.6% in the semaglutide group compared to 2.2% in the placebo group. Mean weight loss was 16.6% and 2.7%, respectively, if all patients adhered to treatment. In addition, 79.2% of patients who received semaglutide compared to 31.1% who received placebo achieved at least 5% reduction of their body weight (p<0.0001). Significant improvements in cardiometabolic risk factors were also observed with semaglutide compared to placebo. The treatment discontinuation rate due to GI adverse events was 3.4% with semaglutide and 2% with placebo.
Dosage and administration
In the OASIS-4 trial, semaglutide tablets were administered orally once daily. The dose was titrated over 12 weeks to a target dose of 25 mg.
Place in therapy
Obesity is a chronic condition associated with a significant increase in mortality and multiple health risks. According to 2017–2018 data from the National Health and Nutrition Examination Survey (NHANES), 30.7% of adults in the U.S. have overweight, 42.4% have obesity and 9.2% have severe obesity.
Several medications are FDA-approved for weight management as adjuncts to lifestyle management (reduced-calorie diet, increased physical activity) in individuals with obesity or with overweight and weight-related comorbid conditions (e.g., T2DM, hypertension, dyslipidemia). These agents include the injectable GLP-1 receptor agonists semaglutide (Wegovy), liraglutide (Saxenda) and tirzepatide (Zepbound), and the oral agents orlistat (Xenical), phentermine (Adipex-P; short-term use), phentermine/topiramate (Qsymia) and naltrexone/bupropion (Contrave). Injectable semaglutide and tirzepatide appear to produce greater weight reduction compared with other agents. Notably, the open-label, Phase 3b SURMOUNT-5 trial demonstrated tirzepatide was superior to semaglutide, each at maximally tolerated doses, for weight reduction at 72 weeks (LSM percent change, -20.2% versus -13.7%, respectively; p<0.001) in patients with obesity but without T2DM. Also to note, oral semaglutide (25 mg) and injectable semaglutide (1.7 mg to 2.4 mg) led to similar percentage weight loss in non-head-to-head trials.
Semaglutide (Rybelsus) is currently the only oral GLP-1 receptor agonist available in the U.S. Rybelsus is indicated for the treatment of T2DM (in daily doses up 14 mg). If approved by the FDA, the Wegovy 25 mg tablet will be the first oral GLP-1 receptor agonist indicated for chronic weight management. This could be followed by the approval of the oral non-peptide GLP-1 agent orforglipron by Eli Lilly with planned submission to the FDA for weight management by the end of 2025.
FDA approval timeline
October-December 2025
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $0 |
| 2026 | $247 |
| 2027 | $787 |
| 2028 | $1,206 |
| 2029 | $1,584 |
sibeprenlimab SC
Proposed indications
Immunoglobulin A (IgA) nephropathy (IgAN)
Clinical overview
Mechanism of action
Sibeprenlimab is a monoclonal antibody that selectively inhibits the activity of A PRoliferation-Inducing Ligand (APRIL), which plays a key role in IgAN pathogenesis by promoting the production of pathogenic galactose-deficient (Gd)-IgA1 and immune complex formation.
Clinical trial(s)
The ongoing, randomized, double-blind, placebo-controlled Phase 3 VISIONARY (NCT05248646) trial evaluated sibeprenlimab for the treatment of adults (n=510) with IgAN who are receiving maximally tolerated SOC. The primary endpoint was reduction of proteinuria as measured by the change in 24-hour urine protein-to-creatinine ratio (UPCR) at 9 months. Interim analysis revealed that patients treated with sibeprenlimab achieved a 51.2% (p<0.0001) reduction in proteinuria at 9 months when compared to placebo. Sibeprenlimab demonstrated a favorable safety profile that was consistent with previously reported data in the dose-finding Phase 2 ENVISION trial (NCT04287985, n=155) which reported nasopharyngitis, upper respiratory tract infection, hypertension, diarrhea and muscle pain in > 5% of patients treated with IV-administered sibeprenlimab and more often than in patients who received placebo. Notably, the ENVISION trial reported an LSM difference in eGFR relative to placebo from baseline to month 12 of up to 7.6 mL/min/1.73 m² with IV-administered sibeprenlimab. The ongoing VISIONARY study will evaluate the annualized change in kidney function over 24 months as measured by eGFR as a key secondary endpoint and is expected to be completed in December 2026.
In the VISIONARY trial, sibeprenlimab was administered SC at a dose of 400 mg every 4 weeks and can be self-administered.
Place in therapy
IgAN, also known as Berger’s disease, is an autoimmune disorder characterized by the accumulation of IgA in the kidneys. This causes inflammation and damage to the kidneys and may eventually lead to ESRD. IgAN can occur at any age, but onset is typically seen between 10 to 40 years of age. The incidence is two-fold higher in men than women and more common among Asian and Caucasian populations.
Pharmacologic treatment of IgAN is focused on preventing or delaying ESRD with antihypertensive medications, such as ACEIs, ARBs, beta-blockers and CCBs. Patients at risk for disease progression may switch from an ACEI or ARB to the oral endothelin receptor antagonists (ERAs) sparsentan (Filspari) and atrasentan (Vanrafia; FDA approved in April 2025). Corticosteroids (e.g., oral budesonide DR [Tarpeyo]), immunosuppressive agents, an oral SGLT2 inhibitor or the oral complement inhibitor iptacopan (Fabhalta) may also be added in patients with IgAN at risk of rapid disease progression. While kidney transplantation is the treatment of choice in patients who progress to kidney failure, recurrent IgA accumulation after transplant is common.
If approved, sibeprenlimab will be a first-in-class APRIL inhibitor providing a new mechanism of action for treatment of IgAN when used in addition to supportive care. In the Phase 3 clinical trial, SC-administered sibeprenlimab significantly reduced proteinuria compared to placebo (51.2%) at 9 months and was well tolerated. Significant stabilization of eGFR decline was also reported in the Phase 2 trial with IV-administered sibeprenlimab; the effect on eGFR with SC-administered sibeprenlimab has yet to be reported. It appears that Otsuka is only pursuing FDA approval for SC administration of sibeprenlimab for the treatment of IgAN at this time.
Notably, other ERAs, including Filspari, carry boxed warnings for hepatotoxicity and/or embryo-fetal toxicity and require a REMS program. Monitoring of liver transaminase levels was not reported in the Phase 2 published data, and it remains to be seen if sibeprenlimab will avoid the need for any risk-mitigating measures.
FDA approval timeline
Nov. 28, 2025
FDA designations: Breakthrough Therapy, Priority Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $4 |
| 2026 | $84 |
| 2027 | $221 |
| 2028 | $380 |
| 2029 | $541 |
tolebrutinib oral
Proposed indications
To treat non-relapsing secondary progressive multiple sclerosis (SPMS) and to slow disability accumulation independent of relapse activity.
Clinical overview
Mechanism of action
Tolebrutinib is a Bruton’s tyrosine kinase (BTK) inhibitor that crosses the blood-brain barrier and regulates B-lymphocytes and disease-associated microglia within the CNS, thereby targeting smoldering neuroinflammation associated with MS progression.
Clinical trial(s)
The 48-month, double-blind, placebo-controlled, Phase 3 HERCULES (NCT04411641) trial evaluated tolebrutinib in 1,131 adults with non-relapsing SPMS. After a median follow-up of 133 weeks, the primary endpoint of confirmed disability progression that was sustained for at least 6 months was observed in a smaller proportion of patients in the tolebrutinib group compared to the placebo group (22.6% versus 30.7%, respectively; p=0.003). In addition, the mean annualized rate of new or enlarging lesions on transverse relaxation times (T2)-weighted MRI, a secondary endpoint, was 1.84 in the tolebrutinib group and 2.95 in the placebo group (p=0.01). Elevations in ALT greater than 3 × ULN occurred in 4% of patients who received tolebrutinib compared to 1.6% who received placebo. Four patients in the tolebrutinib group and no patients in the placebo group experienced ALT increases to more than 20 ˣ ULN, all within 90 days of the first treatment dose. One death occurred due to postoperative complications after a patient received a liver transplantation, with the underlying liver failure related to tolebrutinib. The study was subsequently amended to include weekly liver function monitoring, after which all cases of elevated liver enzymes resolved without sequelae.
Dosage and administration
In clinical trials, tolebrutinib 60 mg was administered orally once daily.
Place in therapy
Approximately 1 million people in the U.S. are living with multiple sclerosis (MS). The majority of cases are relapsing-remitting MS (RRMS), and while disease courses are highly variable, patients can eventually progress to secondary progressive multiple sclerosis (SPMS), experiencing a gradual worsening of neurological pathology. Evidence suggests that during SPMS an expansion of existing lesions occurs, rather than an increase in new active lesions.
Several DMTs are indicated to treat relapsing forms of MS, including active SPMS. They include but are not limited to the following:
Injectable CD20- or CD52-directed cytolytic antibodies: ocrelizumab (Ocrevus), ofatumumab (Kesimpta), ublituximab-xiiy (Briumvi) and alemtuzumab (Lemtrada)
Oral sphingosine 1-phosphate receptor modulators: fingolimod (Gilenya), ozanimod (Zeposia), ponesimod (Ponvory) and siponimod (Mayzent)
Oral fumarate products: dimethyl fumarate (Tecfidera), diroximel fumarate (Vumerity) and monomethyl fumarate (Bafiertam)
Oral pyrimidine synthesis inhibitor: teriflunomide (Aubagio)
Oral purine antimetabolite: cladribine (Mavenclad)
If approved, tolebrutinib will be the first BTK inhibitor for the treatment of MS and the first therapy specifically approved for non-relapsing SPMS. Tolebrutinib is differentiated from other agents by its ability to penetrate the brain to target B cells, versus existing medicines that target peripheral B and T cells. In clinical trials, tolebrutinib significantly delayed (~30%) time to onset of confirmed disability progression compared to placebo in patients with non-relapsing SPMS. In clinical trials, the risk of liver injury associated with tolebrutinib was mitigated with weekly monitoring of liver enzymes.
Notably, ICER released a final evidence report assessing the comparative clinical effectiveness and value of tolebrutinib for the treatment of SPMS. The report acknowledges that safe and effective treatment for non-relapsing SPMS remains a significant unmet health care need, as there is no FDA-approved agent for the condition. Treatment with tolebrutinib will likely require close monitoring of liver function tests, due to the risk of liver toxicity from the drug as reported in the clinical trial. An independent appraisal committee of medical evidence experts voted (12-1) that the current evidence for tolebrutinib is not adequate to demonstrate that the net health benefit for the drug is greater than that of best supportive care.
Tolebrutinib is also in Phase 3 trials for relapsing MS and primary progressive MS.
FDA approval timeline
Sept. 28, 2025
FDA designation: Breakthrough Therapy, Priority Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $32 |
| 2026 | $133 |
| 2027 | $238 |
| 2028 | $380 |
| 2029 | $537 |
Keep on your radar
| Drug generic name | Therapeutic category | Jul 2025 pipeline - Total U.S. sales for 2029 (Dollars in millions) |
|---|---|---|
| aficamten | Cardiovascular | $965 |
| anitocabtagene autoleucel | Oncology - Gene Theraopy | $1,257 |
| atacicept | Immunology | $802 |
| botaretigene sparoparvovec | Ophthalmology - Gene therapy | $89 |
| cretostimogene grenadenorepvec | Oncology – Gene therapy | $819 |
| depemokimab | Respiratory | $806 |
| imlunestrant | Oncology | $427 |
| orforglipron | Endocrine | $7,951 |
| paltusotine | Endocrine | $465 |
| pariglasgene brecaparvovec | Metabolic - Gene therapy | $157 |
| plozasiran | Cardiovascular | $286 |
| remibrutinib | Dermatology | $777 |
| tividenofusp alfa | Metabolic | $177 |
| ziftomenib | Oncology | $282 |
Drug list
Gene and cellular therapies
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| zopapogene imadenovec (PRGN-2012) | Precigen | Recurrent respiratory papillomatosis | SC | BLA – BT, OD, PR, seeking AA | 08/27/2025 |
| onasemnogene abeparvovec-xioi (Zolgensma) | Novartis | Spinal muscular atrophy | Intrathecal | BLA – BT, FT, OD | Oct 2025-Mar 2026 |
| etuvetidigene autotemcel (OTL-103) | Fondazione Telethon | Wiskott-Aldrich syndrome (WAS) | IV | BLA – OD, RMAT, RPD | Nov 2025-Mar 2026 |
| clemidsogene lanparvovec (RGX-121) | Nippon Shinyaku, Regenxbio | Mucopolysaccharidosis II (MPSII; Hunter syndrome) | Intracisternal | BLA – FT, OD, PR, RMAT, RPD, seeking AA | 11/09/2025 |
| tabelecleucel | Pierre Fabre, Atara | Epstein-Barr virus-associated post-transplant lymphoproliferative disease (ages ≥ 2 years) | IV | BLA – BT, OD | 01/10/2026 |
None
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| AAV8-ranibizumab (RGX-314) | Abbvie | Wet AMD | Subretinal | BLA – OD | TBD |
| aglatimagene besadenovec (CAN-2409) | Candel | Prostate cancer | Trans-rectal injection | BLA – FT, RMAT | TBD |
| anitocabtagene autoleucel | Arcellx, Gilead | Multiple myeloma | IV | BLA – FT, OD, RMAT | TBD |
| avalotcagene ontaparvovec (DTX301) | Ultragenyx | Ornithine transcarbamylase deficiency | IV | BLA – FT, OD | TBD |
| baluretgene parvec (OCU400) | Ocugen | Retinitis pigmentosa | Subretinal | BLA – OD, RMAT | TBD |
| botaretigene sparoparvovec | Janssen, MeiraGTx | Retinitis pigmentosa | Subretinal | BLA – FT, OD | TBD |
| cretostimogene grenadenorepvec (CG0070) | CG Oncology | Bladder cancer | Intravesical | BLA – BT, FT | TBD |
| giroctocogene fitelparvovec | Sangamo | Hemophilia A | IV | BLA – FT, OD, RMAT | TBD |
| intismeran autogene | Moderna | Melanoma | IM | BLA – BT | TBD |
| ixoberogene soroparvovec | Adverum | Wet AMD | Intravitreal | BLA – FT, RMAT | TBD |
| laruparetigene zovaparvovec | Beacon | Retinitis pigmentosa | Subretinal | BLA – FT, OD, RMAT | TBD |
| marnetegragene autotemcel (Kresladi) | Rocket | Leukocyte adhesion deficiency type I (LAD-I) | IV | BLA – FT, OD, RMAT, RPD | TBD |
| orca-T (allogeneic T-cell immunotherapy) | Orca | ALL; AML; Myelodysplastic syndrome | IV | BLA – RMAT | TBD |
| pariglasgene brecaparvovec (DTX401) | Ultragenyx | Glycogen storage disease (type 1a) | IV | BLA – FT, OD | TBD |
| rexlemestrocel-L (Revascor) | Mesoblast | End-stage ischemic HFrEF implanted with LVAD | IM | BLA – OD, RMAT, may seek AA | TBD |
| RGX-202 | Regenxbio | DMD | IV | BLA – FT, OD, RPD | TBD |
| rivunatpagene miziparvovec (UX701) | Ultragenyx | Wilson's disease | IV | BLA – FT, OD | TBD |
| zimislecel | Vertex, Novartis | T1DM | Hepatic portal vein infusion | BLA – FT, RMAT | TBD |
Biosimilars
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Organon, Henlius | Osteoporosis; Bone cancer | SC | BLA | Aug-Sept 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Teva | Osteoporosis; Bone cancer | SC | BLA | Aug-Sept 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Biocon | Osteoporosis; Bone cancer | SC | BLA | Aug-Nov 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Gedeon Richter | Osteoporosis; Bone cancer | SC | BLA | Oct-Nov 2025 |
| aflibercept (biosimilar to Regeneron's Eylea) | Alvotech, Teva | DME; Diabetic retinopathy; Macular edema from RVO; Wet AMD | Intravitreal | BLA | Oct-Dec 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Amneal, Mabxience | Osteoporosis; Bone cancer | SC | BLA | Oct-Dec 2025 |
| ranibizumab (biosimilar to Genentech's Lucentis) | Stada, Xbrane | Macular edema from RVO; Myopic choroidal neovascularization (mCNV); Wet AMD | Intravitreal | BLA | 10/21/2025 |
| pertuzumab (biosimilar to Genentech's Perjeta) | Organon, Henlius | Breast cancer | IV | BLA | December 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Dr. Reddy's, Alvotech | Osteoporosis; Bone cancer | SC | BLA | 12/31/2025 |
| insulin aspart (biosimilar to Novo Nordisk's Novolog) | Amphastar | T1DM; T2DM | SC | BLA | Jan-Mar 2026 |
| golimumab (biosimilar to Janssen's Simponi) | Alvotech, Teva | RA; AS; PsA; UC | SC | BLA | 05/16/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designation | FDA decision |
|---|---|---|---|---|---|
| bevacizumab (biosimilar to Genentech’s Avastin) | Essex | Wet AMD | IV | BLA | TBD |
| omalizumab (biosimilar to Genentech's Xolair) | Amneal | Urticaria | SC | BLA | TBD |
None
Specialty
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| ataluren (Translarna) | PTC | DMD (nonsense mutation) | Oral | NDA – FT, OD | 2025 |
| gemcitabine-releasing intravesical system (TAR-200) | Taris | Bladder cancer (BCG-unresponsive, high-risk non-muscle-invasive, with CIS) | Intravesical | NDA – BT, FT, PR, RTOR | 2025 |
| sodium pyruvate (N115) | Emphy | Idiopathic pulmonary fibrosis (IPF) | Intranasal | NDA | Jul-Sept 2025 |
| zongertinib | Boehringer Ingelheim | NSCLC (unresectable or metastatic, HER2+, prior systemic therapy) | Oral | NDA – BT, FT, PR | Jul-Sept 2025 |
| sodium chlorite (NP001) | Neuvivo | ALS | IV | NDA – FT, OD, seeking AA | Jul-Oct 2025 |
| doxecitine / doxribtimine | UCB | Thymidine kinase 2 (TK2) deficiency | Oral | NDA – BT, OD, PR | August 2025 |
| brensocatib | Insmed, AstraZeneca | Bronchiectasis | Oral | NDA – BT, PR | 08/12/2025 |
| dordaviprone (ONC201) | Chimerix | Glioma (diffuse, recurrent H3 K27M-mutant) | Oral | NDA – FT, OD, PR, RPD, seeking AA | 08/18/2025 |
| vatiquinone | PTC | Friedreich's ataxia | Oral | NDA – FT, OD, PR | 08/19/2025 |
| donidalorsen | Ionis | HAE (prophylaxis to prevent attack, ages ≥ 12 years) | SC | BLA – OD | 08/21/2025 |
| bevacizumab-vikg | Outlook | Wet AMD | Intravitreal | BLA | 08/27/2025 |
| sodium dichloroacetate (SL-1009) | Saol | Pyruvate dehydrogenase complex deficiency (PDCD) | Oral | NDA – FT, OD, PR, RPD | 08/27/2025 |
| rilzabrutinib | Sanofi | Immune thrombocytopenic purpura (ITP) | Oral | NDA – FT, OD | 08/29/2025 |
| lecanemab-irmb (Leqembi) SC | Eisai | Alzheimer's disease (mild, weekly maintenance dosing) | SC | BLA | 08/31/2025 |
| apitegromab | Scholar Rock | Spinal muscular atrophy | IV | BLA – FT, OD, PR, RPD | 09/22/2025 |
| pembrolizumab / hyaluronidase | Merck | All previously approved solid tumor indications for pembrolizumab (Keytruda) | SC | BLA | 09/23/2025 |
| narsoplimab | Omeros | Transplant-associated thrombotic microangiopathy (TA-TMA) | IV, SC | BLA – BT, OD | 09/25/2025 |
| paltusotine | Crinetics | Acromegaly | Oral | NDA – OD | 09/25/2025 |
| tolebrutinib | Sanofi | MS (non-relapsing secondary progressive) | Oral | NDA – BT, PR | 09/28/2025 |
| copper histidinate | Zydus | Menkes disease | SC | NDA – BT, FT, OD, PR, RPD | 09/30/2025 |
| imlunestrant | Eli Lilly | Breast cancer (HR+, HER2-) | Oral | NDA | Oct-Dec 2025 |
| nerandomilast | Boehringer Ingelheim | Idiopathic pulmonary fibrosis (IPF); Progressive pulmonary fibrosis (PPF) | Oral | NDA – BT, OD, PR | Oct-Dec 2025 |
| troriluzole | Biohaven | Spinocerebellar ataxia | Oral | 505(b)(2) NDA – FT, OD, PR | Oct-Dec 2025 |
| dasatinib (amorphous formulation) | Xspray | CML | Oral | 505(b)(2) NDA – OD | 10/07/2025 |
| remibrutinib | Novartis | Chronic spontaneous urticaria (CSU) | Oral | NDA – PR | November 2025 |
| sevabertinib | Bayer | NSCLC (advanced, ERBB2 mutations, prior systemic therapy) | Oral | NDA – BT, PR | Nov-Dec 2025 |
| plozasiran | Arrowhead | Familial chylomicronemia syndrome (FCS) | SC | NDA – BT, FT, OD | 11/18/2025 |
| navepegritide | Ascendis | Achondroplasia (children) | SC | NDA – OD, PR | 11/28/2025 |
| sibeprenlimab | Otsuka | IgA nephropathy (Berger's Disease) | SC | BLA – BT, PR | 11/28/2025 |
| ziftomenib | Kura | AML (R/R, NPM 1 mutation) | Oral | NDA – BT, OD, PR | 11/28/2025 |
| depemokimab | GlaxoSmithKline | Asthma (ages ≥ 12 years, type 2 inflammation); Chronic rhinosinusitis | SC | BLA | 12/16/2025 |
| aficamten | Cytokinetics | Obstructive hypertrophic cardiomyopathy | Oral | NDA – BT, OD | 12/26/2025 |
| fibrinogen | Grifols | Fibrinogen deficiency (acquired and congenital) | IV | BLA | 12/27/2025 |
| relacorilant | Corcept | Cushing's syndrome | Oral | NDA – OD | 12/30/2025 |
| tividenofusp alfa | Denali | Mucopolysaccharidosis II (MPS II; Hunter Syndrome) | IV | BLA – BT, FT, OD, PR, RPD, seeking AA | 01/05/2026 |
| vepdegestrant | Arvinas, Pfizer | Breast cancer (advanced or metastatic, HR+, HER2-, ESR1-mutated, prior ERT) | Oral | NDA – FT | Feb-Jun 2026 |
| linerixibat | GlaxoSmithKline | Cholestatic pruritus | Oral | NDA – OD | 03/24/2026 |
| relacorilant | Corcept | Ovarian cancer (platinum-resistant, in combination with nab-paclitaxel) | Oral | NDA | 07/14/2026 |
| icotrokinra | Janssen | PSO | Oral | NDA | 07/21/2026 |
| elamipretide | Stealth, AstraZeneca | Barth syndrome | SC | NDA – FT, OD, PR, RPD | PDUFA date passed |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| selumetinib (Koselugo) | AstraZeneca | Neurofibromatosis type 1 | Oral | sNDA – BT, OD | Jul-Dec 2025 |
| tafasitamab-cxix (Monjuvi) | Incyte | Marginal zone lymphoma | IV | sBLA – OD | Jul-Dec 2025 |
| sparsentan (Filspari) | Travere | IgA nephropathy (Berger's disease) | Oral | sNDA – OD, PR | 08/25/2025 |
| mitapivat (Pyrukynd) | Agios | Thalassemia (alpha or beta, non-transfusion-dependent and transfusion-dependent) | Oral | sNDA | 09/07/2025 |
| berotralstat (Orladeyo) | Biocryst | HAE (aged 2–11 years) | Oral | sNDA – FT, OD, PR | 09/12/2025 |
| guselkumab (Tremfya) | Janssen | UC (SC induction regimen) | SC | sBLA | 09/22/2025 |
| daratumumab / hyaluronidase-fihj (Darzalex Faspro) | Janssen | Multiple myeloma (high-risk, smoldering) | SC | sBLA | 09/30/2025 |
| obinutuzumab (Gazyva) | Genentech | Lupus nephritis | IV | sBLA – BT | October 2025 |
| guselkumab (Tremfya) | Janssen | PSO (ages 6–17 years); PsA (ages 5–17 years) | SC | sBLA | 10/02/2025 |
| lurbinectedin (Zepzelca) | Jazz | SCLC (extensive-stage, 1st-line mainenance) | IV | sNDA – OD, PR | 10/07/2025 |
| golimumab (Simponi) SC | Janssen, Bristol Myers Squibb | UC (ages ≥ 2 years) | SC | sBLA – OD | 10/16/2025 |
| tezepelumab-ekko (Tezspire) | Amgen | Chronic rhinosinusitis; Nasal polyposis | SC | sBLA | 10/19/2025 |
| riboflavin 5’-phosphate (Epioxa) | Glaukos | Keratoconus | Ophthalmic | sNDA – OD | 10/20/2025 |
| belantamab mafodotin (Blenrep) | GlaxoSmithKline | Multiple myeloma (R/R, ≥ 1 prior lines of therapy) | IV | sBLA – OD | 10/23/2025 |
| revumenib (Revuforj) | Syndax | AML (R/R, NPM 1 mutation) | Oral | sNDA – BT, FT, OD, PR, RTOR | 10/25/2025 |
| cemiplimab-rwlc (Libtayo) | Regeneron | Cutaneous squamous cell carcinoma (high-risk, post surgery) | IV | sBLA – BT | Nov 2025–Jan 2026 |
| nusinersen (Spinraza) | Biogen | Spinal muscular atrophy (high dose regimen) | Intrathecal | sNDA – FT, OD | 11/21/2025 |
| inebilizumab-cdon (Uplizna) | Amgen | Myasthenia gravis | IV | sBLA – OD | 12/14/2025 |
| sparsentan (Filspari) | Travere, Bristol Myers Squibb | Focal segmental glomerulosclerosis | Oral | sNDA – OD, PR | 01/13/2026 |
| decitabine / cedazuridine (Inqovi) | Taiho | AML (newly diagnosed, intensive induction chemotherapy ineligible; in combination with venetoclax, adults) | Oral | sNDA | 02/25/2026 |
| deucravacitinib (Sotyktu) | Bristol Myers Squibb | PsA | Oral | sNDA | 03/06/2026 |
| ustekinumab (Stelara) | Janssen | CD (ages 2–17 years) | IV, SC | sBLA – OD | 04/17/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designation | FDA decision |
|---|---|---|---|---|---|
| afimkibart | Roche | UC | SC | BLA | TBD |
| amlitelimab | Sanofi | Atopic dermatitis | SC | BLA | TBD |
| amniotic suspension allograft (ReNu) | Organogenesis | Osteoarthritis symptoms (knee) | Intra-articular | BLA – RMAT | TBD |
| apraglutide | Ironwood | Short bowel syndrome (parenteral support-dependent) | SC | NDA – OD | TBD |
| astegolimab | Genentech, Amgen | COPD | IV | BLA | TBD |
| atacicept | Vera, Bristol Myers Squibb | IgA nephropathy (Berger's disease); Lupus nephritis | SC | BLA – BT, OD | TBD |
| bemarituzumab | Amgen | Gastric cancer | IV | BLA – BT, OD | TBD |
| bepirovirsen | GlaxoSmithKline | HBV treatment | SC | NDA – FT | TBD |
| bis-choline-tetrathiomolybdate | Monopar | Wilson's disease | Oral | NDA – FT, OD | TBD |
| blarcamesine | Anavex | Alzheimer's disease; Rett syndrome | Oral | NDA – FT, RPD, may seek AA | TBD |
| buntanetap | Annovis | Alzheimer's disease | Oral | NDA | TBD |
| camizestrant | AstraZeneca | Breast cancer (HR+/HER2-) | Oral | NDA | TBD |
| carbetocin | Acadia | Prader-Willi syndrome | Intranasal | NDA – FT, OD | TBD |
| cetuximab sarotalocan | Rakuten | SCCHN | IV | BLA – FT | TBD |
| cobolimab | GlaxoSmithKline | NSCLC (2nd-line) | IV | BLA | TBD |
| condoliase | Ferring | Radicular leg pain associated with lumbar disc herniation | Intervertebral disc injection | BLA | TBD |
| delpacibart braxlosiran | Avidity | Facioscapulohumeral muscular dystrophy | IV | NDA – FT, OD, may seek AA | TBD |
| denecimig | Novo Nordisk | Hemophilia A | SC | BLA – OD | TBD |
| deucrictibant | Pharvaris | HAE | Oral | NDA – OD | TBD |
| divarasib | Genentech | NSCLC | Oral | NDA – BT | TBD |
| ensitrelvir | Shionogi | COVID-19 post-exposure prophylaxis | Oral | BLA – FT | TBD |
| ersodetug | Rezolute | Congenital hyperinsulinism | IV | BLA – BT, OD, RPD | TBD |
| fenebrutinib | Genentech | MS | Oral | NDA | TBD |
| fianlimab | Regeneron | Melanoma | IV | BLA – FT | TBD |
| frexalimab | Sanofi | MS | IV, SC | BLA | TBD |
| garetosmab | Regeneron | Fibrodysplasia ossificans progressiva | IV | BLA – FT, OD | TBD |
| gedatolisib | Celcuity, Pfizer | Breast cancer (HR+/HER2-) | IV | NDA – BT, FT | TBD |
| giredestrant | Genentech | Breast cancer (HR+/HER2-) | Oral | NDA – FT | TBD |
| glutamate decarboxylase | Diamyd | T1DM (newly diagnosed, ages 12–28 years, HLA DR3-DQ2 haplotype) | Intralymphatic | BLA – FT, OD, may seek AA | TBD |
| gold nanocrystal | Clene | ALS | Oral | NDA – OD | TBD |
| govorestat | Applied | Sorbitol dehydrogenase (SORD) deficiency | Oral | NDA – FT, OD, RPD | TBD |
| GYM329 / RG6237 | Genentech | Spinal muscular atrophy | SC | BLA – OD | TBD |
| hydromethylthionine mesylate | Taurx | Alzheimer's disease (mild-moderate) | Oral | NDA | TBD |
| hydroxypropyl beta cyclodextrin | Cyclo | Niemann-Pick disease (type C) | IV | NDA – FT, OD, RPD | TBD |
| ianalumab | Novartis | Autoimmune hepatitis; ITP; Sjogren's syndrome | SC | BLA – FT | TBD |
| immune globulin / hyaluronidase | Takeda | Primary immunodeficiency diseases | SC infusion | BLA | TBD |
| imsidolimab | Anaptysbio | Generalized pustular psoriasis (GPP) | IV, SC | BLA – OD | TBD |
| inaxaplin | Vertex | Focal segmental glomerulosclerosis | Oral | NDA – BT, may seek AA | TBD |
| itepekimab | Regeneron, Sanofi | COPD | SC | BLA – FT | TBD |
| itolizumab | Equillium | GVHD treatment | IV | BLA – FT, OD | TBD |
| ivonescimab | Summit | NSCLC | IV | BLA – FT | TBD |
| lanifibranor | Inventiva | MASH | Oral | NDA – BT, FT | TBD |
| latozinemab | Alector, GlaxoSmithKline | Frontotemporal dementia | IV | BLA – BT, FT, OD | TBD |
| lonvoguran ziclumeran | Intellia | HAE | IV | BLA – OD, RMAT | TBD |
| masitinib | AB Science | Alzheimer's disease | Oral | NDA | TBD |
| molgramostim | Savara | Acute pulmonary alveolar proteinosis | Inhaled | BLA – BT, FT, OD | TBD |
| obefazimod | Abivax | UC | Oral | NDA | TBD |
| pabinafusp alfa | JCR | Mucopolysaccharidosis II (Hunter syndrome) | IV | BLA – BT, FT, OD | TBD |
| palazestrant | Olema | Breast cancer (HR+/HER2-) | Oral | NDA – FT | TBD |
| patritumab deruxtecan | Merck | NSCLC | IV | BLA – BT | TBD |
| pegadricase | Swedish Orphan Biovitrum | Gout | IV | BLA – FT | TBD |
| pegargiminase | Polaris | Mesothelioma | IM | BLA – FT, OD | TBD |
| pelabresib | Novartis | Myelofibrosis | Oral | NDA – FT, OD | TBD |
| pelacarsen | Novartis | Dyslipidemia | SC | NDA – FT | TBD |
| potravitug | Memo | BK polyomavirus (BKV) infection | IV | BLA – FT | TBD |
| pridopidine | Prilenia | Huntington's disease | Oral | NDA – FT, OD | TBD |
| remibrutinib | Novartis | MS; Spontaneous urticaria | Oral | NDA | TBD |
| resiniferatoxin | Grunenthal | Osteoarthritis pain (knee) | Intra-articular | NDA – BT | TBD |
| riliprubart | Sanofi | Chronic inflammatory demyelinating polyneuropathy (CIDP) | IV, SC | BLA – OD | TBD |
| rusfertide | Takeda | Polycythemia vera | SC | NDA – FT, OD | TBD |
| savolitinib | AstraZeneca | NSCLC (MET+, 2nd-line); RCC | Oral | NDA – FT | TBD |
| sefaxersen | Genentech | IgA nephropathy (Berger's disease) | SC | NDA | TBD |
| serplulimab | Henlius | SCLC | IV | BLA – OD | TBD |
| suramin | PaxMedica | Human African sleeping sickness | IV | NDA – OD | TBD |
| tebipenem pivoxil | GlaxoSmithKline | UTI (complicated) | Oral | NDA – FT, QIDP | TBD |
| tiragolumab | Genentech | HCC; NSCLC (unresectable, Stage 3, 1st-line) | IV | BLA – BT, FT | TBD |
| tiratricol | Egetis | Monocarboxylate transporter 8 (MCT8) deficiency | Oral | NDA – FT, OD, RPD | TBD |
| veligrotug | Viridian | Thyroid eye disease (TED) | IV | BLA – BT | TBD |
| venglustat | Sanofi | Fabry's disease; Gaucher's disease | Oral | NDA – FT, OD | TBD |
| viaskin peanut | DBV | Peanut allergy | Transdermal | BLA – BT, FT | TBD |
| vidofludimus | Immunic | MS | Oral | NDA | TBD |
| VRDN-003 | Viridian | Thyroid eye disease (TED) | SC | BLA | TBD |
| zasocitinib | Takeda | Psoriasis | Oral | NDA | TBD |
| zelenectide pevedotin | Bicycle | Bladder cancer | IV | NDA – FT | TBD |
| zilganersen | Ionis | Alexander disease | Intrathecal | NDA – FT, OD | TBD |
| zipalertinib | Taiho | NSCLC (EGFR exon 20 insertion mutations) | Oral | NDA – BT | TBD |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designation | 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); Esophageal cancer; Prostate cancer (metastatic, castration-resistant) | IV | sBLA – OD | TBD |
| brolucizumab-dbll (Beovu) | Novartis | Diabetic retinopathy | Intravitreal | sBLA | TBD |
| cemiplimab-rwlc (Libtayo) | Regeneron, Sanofi | Melanoma | IV | sBLA – FT | 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 – FT, OD | TBD |
| iptacopan (Fabhalta) | Novartis | Hemolytic uremic syndrome | Oral | sNDA | TBD |
| mitapivat (Pyrukynd) | Agios | SCD | Oral | sNDA – OD | TBD |
| mosunetuzumab-axgb (Lunsumio) | Genentech | DLBCL (2nd-line, in combination with polatuzumab vedotin-piiq) | SC | sBLA | TBD |
| nipocalimab-aahu (Imaavy) | Janssen | Autoimmune hemolytic anemia | IV | sBLA – FT, OD | TBD |
| nogapendekin alfa inbakicept (Anktiva) | Immunitybio | NSCLC | IV, SC | sBLA | TBD |
| obinutuzumab (Gazyva) | Genentech | Membranous nephropathy; Nephrotic syndrome (pediatrics); SLE | IV | sBLA – OD | TBD |
| olezarsen (Tryngolza) | Akcea | Dyslipidemia | SC | sNDA | TBD |
| pitolisant (Wakix) | Harmony | Excessive sleepiness associated with medical conditions | Oral | sNDA | TBD |
| ropeginterferon alfa-2b-njft (Besremi) | PharmaEssentia | Essential thrombocythemia | SC | sBLA – OD | TBD |
| satralizumab-mwge (Enspryng) | Genentech | Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD); Thyroid eye disease (TED) | SC | sBLA – OD | TBD |
| secukinumab (Cosentyx) | Novartis | Giant cell arteritis; Lupus nephritis | SC | sBLA | TBD |
| treprostinil sodium (Tyvaso) | Ferrer, GlaxoSmithKline | Idiopathic pulmonary fibrosis | Inhaled | sNDA – OD | TBD |
| zanidatamab-hrii (Ziihera) | Jazz | Gastroesophageal adenocarcinoma (HER2+) | IV | sBLA – FT, OD | TBD |
Traditional
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| aceclidine | Lenz | Presbyopia | Ophthalmic | NDA | 08/08/2025 |
| cyclobenzaprine | Tonix | Fibromyalgia | SL | 505(b)(2) NDA – FT | 08/15/2025 |
| bumetanide | Corstasis | Edema (associated with CHF, liver disease, & kidney disease) | Inhaled | 505(b)(2) NDA | 09/14/2025 |
| lidocaine patch (Ionic Liquid Transdermal System) | Medrx | Post-herpetic neuralgia (PHN) | Transdermal | 505(b)(2) NDA | 09/24/2025 |
| carbidopa / levodopa | Mitsubishi Tanabe | Parkinson's disease motor fluctuations | SC | 505(b)(2) NDA | Oct-Dec 2025 |
| semaglutide (Wegovy) 25 mg tablet | Novo Nordisk | Obesity or overweight with ≥ 1 weight-related comorbidity; Reduce the risk of MACE in adults with obesity/overweight and established CVD | Oral | NDA | Oct-Dec 2025 |
| elinzanetant | Bayer | Vasomotor symptoms associated with menopause | Oral | NDA | 10/01/2025 |
| valacyclovir oral suspension | Hyloris | HSV infection | Oral | 505(b)(2) NDA | 10/12/2025 |
| atropine | Sydnexis | Myopia (pediatric) | Ophthalmic | 505(b)(2) NDA | 10/23/2025 |
| lerodalcibep | LIB | HeFH; HoFH; LDL-C reduction (patients with ASCVD, or very high or high risk of ASCVD) | SC | BLA | 12/12/2025 |
| etripamil (Cardamyst) | Milestone | Paroxysmal supraventricular tachycardia | Intranasal | NDA | 12/13/2025 |
| zoliflodacin | Innoviva | Gonorrhea (uncomplicated, ages ≥ 12 years) | Oral | NDA – FT, PR, QIDP | 12/15/2025 |
| reproxalap | Aldeyra, Abbvie | DED | Ophthalmic | NDA | 12/16/2025 |
| tradipitant | Vanda, Eli Lilly | Motion sickness | Oral | NDA | 12/30/2025 |
| brimonidine / carbachol | Visus, Tenpoint | Presbyopia | Ophthalmic | 505(b)(2) NDA | 01/28/2026 |
| epinephrine | Aquestive | Anaphylaxis | SL | 505(b)(2) NDA – FT | 01/31/2026 |
| milsaperidone | Vanda | Bipolar I disorder; Schizophrenia | Oral | NDA | 02/21/2025 |
| desmopressin oral solution | Eton | Central diabetes insipidus (CDI) | Intranasal | 505(b)(2) NDA | 02/25/2026 |
| doravirine / islatravir | Merck | HIV-1 infection treatment | Oral | NDA | 04/28/2026 |
| nimodipine | Grace | Subarachnoid hemorrhage | IV | 505(b)(2) NDA – FT, OD | 06/25/2026 |
| cytisinicline | Achieve | Smoking cessation (nicotine dependence) | Oral | NDA – BT | 06/26/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| brexpiprazole (Rexulti) | Otsuka | PTSD (in combination with sertraline) | Oral | sNDA | Jul–Dec 2025 |
| semaglutide (Wegovy) | Novo Nordisk | Chronic HFpEF | SC | sNDA | Jul–Dec 2025 |
| semaglutide (Wegovy) | Novo Nordisk | MASH | SC | sNDA - BT, PR | 08/18/2025 |
| baloxavir marboxil (Xofluza) | Genentech | Influenza (transmission reduction) | Oral | sNDA | September 2025 |
| risperidone ER (Uzedy) | Teva | Bipolar I disorder (maintenance, adults) | SC | sNDA | Sept–Oct 2025 |
| ruxolitinib (Opzelura) | Incyte | Atopic dermatitis (ages 2–11 years) | Topical | sNDA | 09/19/2025 |
| semaglutide (Rybelsus) 14 mg | Novo Nordisk | MACE risk reduction (in adults with T2DM & CVD and/or CKD) | Oral | sNDA | October 2025 |
| semaglutide (Ozempic) | Novo Nordisk | PAD (in patients with T2DM) | SC | sNDA | Oct–Dec 2025 |
| lumateperone (Caplyta) | Intra-Cellular Therapies | MDD (adjunct to antidepressants, adults) | Oral | sNDA | 10/03/2025 |
| roflumilast (Zoryve) 0.05% cream | Arcutis, AstraZeneca | Atopic dermatitis (ages 2–5 years) | Topical | sNDA | 10/13/2025 |
| RSV vaccine (Arexvy) | GlaxoSmithKline | RSV immunization (ages 18–49 years at increased risk for RSV disease) | IM | sBLA – FT | Jan–Jun 2026 |
| celecoxib (Elyxyb) ready-to-use oral solution | Scilex | Acute pain | Oral | sNDA | 01/21/2026 |
| fremanezumab-vfrm (Ajovy) | Teva | Migraine prevention (ages 6–17 years) | SC | sBLA – FT | 02/07/2026 |
| lumateperone (Caplyta) | Intra-Cellular Therapies | Schizophrenia (relapse prevention) | Oral, SC | sNDA – FT | 05/08/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designation | FDA decision |
|---|---|---|---|---|---|
| allantoin | Paradigm | Epidermolysis bullosa | Topical | NDA – BT, OD, RPD | TBD |
| aroxybutynin / atomoxetine | Apnimed | OSA | Oral | NDA – FT | TBD |
| azetukalner | Xenon | MDD; Seizure disorder | Oral | NDA | TBD |
| brilaroxazine | Reviva | Schizophrenia | Oral | NDA | TBD |
| buntanetap | Annovis | Parkinson's disease | Oral | NDA | TBD |
| cadisegliatin | vTv, Novo Nordisk | T1DM | Oral | NDA – BT | 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 |
| CMV vaccine (mRNA-1647) | Moderna | CMV infection prevention | IV | BLA | TBD |
| dipalmitoyl hydroxyproline (QRX003) | Quoin | Netherton syndrome | Topical | NDA | TBD |
| enobosarm | Veru | Obesity | Oral | NDA | TBD |
| influenza & COVID-19 vaccine (mRNA-1083) | Moderna | Seasonal influenza & COVID-19 immunization (ages ≥ 50 years) | IM | BLA – FT | TBD |
| insulin efsitora alfa | Eli Lilly | T1DM | SC | BLA | TBD |
| Lyme disease vaccine | Valneva, Pfizer | Lyme disease immunization | IM | BLA – FT | TBD |
| navacaprant | Neumora | MDD | Oral | NDA | TBD |
| norovirus vaccine (mRNA-1403) | Moderna | Norovirus immunization | IM | BLA | 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 – BT | 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 vaccination | IM | BLA | TBD |
| ralinepag | United Therapeutics | PAH | Oral | NDA – OD | TBD |
| reboxetine | Axsome, Pfizer | Narcolepsy | Oral | NDA – BT, OD | TBD |
| retatrutide | Eli Lilly | T2DM; Diabetic nephropathy; Obesity; OSA (in patients with obesity/overweight); Osteoarthritis of knee (in patients with obesity/overweight) | 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 – BT | TBD |
| tavapadon | Abbvie | Parkinson's disease | Oral | NDA | TBD |
| tebipenem pivoxil | GlaxoSmithKline | UTI (complicated) | Oral | NDA – FT, QIDP | TBD |
| tegoprazan | Sebela | Erosive esophagitis; Non-erosive reflux disease | Oral | NDA | TBD |
| ulixacaltamide | Praxis | Essential tremor | Oral | NDA | TBD |
| valiltramiprosate | Alzheon | Alzheimer's disease | Oral | NDA – FT | TBD |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designation | FDA decision |
|---|---|---|---|---|---|
| gepotidacin (Blujepa) | GlaxoSmithKline | Urogenital gonorrhea | Oral | sNDA – QIDP | TBD |
| semaglutide (Ozempic) | Novo Nordisk | Alzheimer's disease; Diabetic retinopathy | SC | sNDA | TBD |
| semaglutide (Rybelsus) | Novo Nordisk | Alzheimer's disease | Oral | sNDA | TBD |
| semaglutide (Wegovy) | Novo Nordisk | Osteoarthritis of knee (in patients with obesity/overweight) | SC | sNDA | TBD |
| tirzepatide (Zepbound) | Eli Lilly | HFpEF (in patients with obesity); CKD (in patients with obesity/overweight) | SC | sNDA | TBD |
| treprostinil sodium (Tyvaso) | Ferrer, GlaxoSmithKline | Idiopathic pulmonary fibrosis | Inhaled | sNDA – OD | TBD |
| Name | Manufacturer | Clinical use | Dosage form | Development status |
|---|---|---|---|---|
| aflibercept (Eylea HD) | Regeneron | 24-hour (8 mg) dosing for all approved indications | Intravitreal | CRL |
| deramiocel | Nippon Shinyaku | DMD cardiomyopathy | IV | CRL |
| glofitamab-gxbm (Columvi) | Genentech | DLBCL (R/R, in combination with gemcitabine and oxaliplatin, prior therapy, ineligible for autologous SCT) | IV | CRL |
| oxylanthanum carbonate (Renazorb) | Unicycive | Hyperphosphatemia (in patients with CKD on dialysis) | Oral | CRL |
| rebisufligene etisparvovec (UX111) | Ultragenyx | Mucopolysaccharidosis IIIA (Sanfilippo syndrome type A) | IV | CRL |
| vusolimogene oderparepvec (RP-1) | Replimune | Melanoma (in combination with nivolumab, prior anti-PD1 regimen) | Intratumoral | CRL |
6MWD 6 Minute Walking Distance
6MWT 6 Minute Walking Test
AA Accelerated Approval
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
BT Breakthrough Therapy
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 Differentiation 3
CD19 Cluster of Differentiation 19
CD20 Cluster of Differentiation 20
CD34 cluster of Differentiation 34
CD38 Cluster of Differentiation 38
CD52 cluster of Dfferentiation 52
CD79b Cluster of Differentiation 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
CMV cytomegalovirus
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 Deficient 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
ERBB2 Erythroblastic Oncogene B-2
ECOG Eastern Cooperative Oncology Group
eGFR estimated Glomerular Filtration Rate
EGFR Epidermal Growth Factor Receptor
ER Extended-Release
ERA Endothelin Receptor Agonist
ERK Extracellular signal-Regulated Kinase
ERT Endocrine Receptor Therapy
ESA Erythropoietin Stimulating Agent
ESR1 Estrogen Receptor 1
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
FT Fast Track
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-1RA 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
HLA Human Leukocyte Antigen
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
LVAD Left Ventricular Assist Device
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-Hodgkin Lymphoma
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
OD Orphan Drug
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
PR Priority Review
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
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