Quarterly Drug Pipeline: October 2025 - Prime Therapeutics
Quarterly Drug Pipeline: October 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.
With only a couple of months left in 2025, thus far the agency has approved 34 novel drugs, compared to about 38 novel approvals about the same time last year. For the remainder of the year, 25 notable drugs filed with the agency are profiled, each with an anticipated FDA decision in 2025.
Some of the noteworthy approvals in the third quarter of 2025 include the first SC version of Keytruda in the oncology setting, first-in-class agent for bronchiectasis (a chronic inflammatory lung condition), new options for hereditary angioedema and the first treatment for Barth Syndrome (a rare life-threatening pediatric disease).
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 (35%) continue to grow for agents with applications submitted to the FDA. Five therapies, four of which are 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. Among the products submitted to the FDA, two products are voucher recipients under the new Commissioner’s National Priority Voucher (CNPV) pilot as products with significant potential to address a major national priority.
Notable anticipated approvals for fourth quarter 2025 include:
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First oral GLP-1 for chronic weight management
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Two gene therapies for rare conditions
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An agent for obstructive hypertrophic cardiomyopathy
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Next generation option for severe eosinophilic asthma
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New prodrug for children with achondroplasia
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First-in-class therapy for a rare condition marked by severely high triglycerides
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A monoclonal antibody for an inflammatory kidney condition
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A targeted oral agent for advanced lung cancer
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
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
depemokimab SC
Proposed indications
- Add-on maintenance treatment of asthma with type 2 inflammation characterized by an eosinophilic phenotype in patients aged ≥ 12 years
- Add-on maintenance treatment of chronic rhinosinusitis with nasal polyps (CRSwNP) in adults
Clinical overview
Mechanism of actionDepemokimab is an ultra-long-acting monoclonal antibody (mAb) that targets IL-5, a key cytokine in type 2 inflammation.
The Phase 3a, placebo-controlled SWIFT-1 (NCT04719832; n=395) and SWIFT-2 (NCT04718103; n=397) replicate trials evaluated depemokimab in patients ≥ 12 years of age with severe asthma and an eosinophilic phenotype characterized by a high eosinophil count (≥ 300 cells per microliter in the previous 12 months or ≥ 150 cells/microliter). Patients were randomized 2:1 to depemokimab or placebo, in combination with their baseline maintenance asthma SOC treatment throughout the study. Pooled data revealed a 54% reduction in the primary endpoint of annualized exacerbation rate (AER) (0.51 versus 1.11, respectively; rate ratio [RR], 0.46; p<0.001). In addition, the secondary endpoint of clinically significant exacerbations requiring hospitalization or emergency department visit was reduced by 72% with depemokimab compared to placebo (AER, 0.02 versus 0.09, respectively; RR, 0.28; p=0.002). The type and frequency of adverse events were similar among the depemokimab and placebo groups.
The Phase 3, double-blind, placebo-controlled, parallel-group, ANCHOR-1 (NCT05274750; n=271) and ANCHOR-2 (NCT05281523; n=257) replicate trials evaluated depemokimab in adults with inadequately controlled CRSwNP, an endoscopic bilateral nasal polyps score ≥ 5, previous surgery for CRSwNP or previous treatment with or intolerance to systemic corticosteroids, and severe symptoms. Patients were randomized 1:1 to depemokimab or placebo. Pooled data revealed statistically significant improvements from baseline with depemokimab compared to placebo in the coprimary endpoints of total nasal polyps score at 52 weeks (treatment difference, -0.7; nominal p<0.001) and mean nasal obstruction verbal response scale (VRS) score over weeks 49–52 (treatment difference, -0.24; nominal p=0.003). The VRS scoring showed improvement for both rhinorrhea and sense of smell with depemokimab compared to placebo. The type and frequency of adverse events were similar among the depemokimab and placebo groups, with < 1% of patients discontinuing treatment due to adverse events.
Dosage and administration
In the above clinical trials, depemokimab was administered at a dose of 100 mg SC every six months (weeks 0 and 26) using a prefilled safety syringe. There is potential for depemokimab to be self-administered using an auto-injector.
Place in therapy
There are approximately 1.4 million emergency department visits annually in the United States that are attributable to asthma. Severe asthma has been defined as asthma that can only be controlled with high-dose inhaled glucocorticoids plus a second controller medication and/or systemic glucocorticoids, or that remains uncontrolled despite this therapy. Type 2 inflammation is present in most individuals with severe asthma. It is characterized by the presence of cytokines (e.g., IL-4, IL-5, IL-13) and elevation of eosinophils or fractional concentration of exhaled nitric oxide (FeNO). The addition of biologic therapy is recommended for patients with asthma exacerbations or poor symptom control despite high-dose ICS-LABA treatment in patients with severe asthma. Biologic agents approved for severe asthma include the anti-immunoglobulin E mAb omalizumab (Xolair, biosimilar Omlyclo), anti-IL-4 mAb dupilumab (Dupixent), anti-IL-5 mAbs benralizumab (Fasenra), mepolizumab (Nucala) and reslizumab (Cinqair), and anti-thymic stromal lymphopoietin mAb tezepelumab-ekko (Tezspire). While indications for the anti-IL-5 agents require eosinophilic phenotype confirmation, the indications for the remaining biologics do not. Xolair, Dupixent, Fasenra and Nucala are approved for use in patients ≥ 6 years of age, Tezspire is approved for those ≥ 12 years of age and Cinqair is approved for use in adults only. In Phase 3 clinical trials, depemokimab was evaluated in patients ≥ 12 years of age.
Chronic rhinosinusitis (CRS) is defined as inflammation of the paranasal sinuses and linings of the nasal passages lasting at least three months. The estimated prevalence of CRS is 2–16% of the population in the United States and CRSwNP accounts for about 18–30% of CRS cases. CRS is associated with nasal congestion, facial pain or pressure and a decreased sense of smell. Nasal polyps are growths inside the nose or sinuses that may block the airways which may lead to infections. Recommended initial treatment for CRSwNP includes saline nasal irrigation and an intranasal corticosteroid (INCS). Approved biologics for CRSwNP include Dupixent, Nucala, Tezspire and Xolair/Omlyclo. These agents may be preferred in patients with high disease burden, particularly if it continues after four weeks of INCS therapy. In a multicenter cohort study, functional endoscopic sinus surgery provided greater improvement in sino-nasal outcomes test (SNOT-22) compared to biologic agents and may be particularly beneficial in patients with CRS with obstructing nasal polyps; however, relapse is common, and surgery may need to be repeated.
If approved, depemokimab will compete with the current biologic agents for the treatment of severe asthma with an eosinophilic phenotype in patients ≥ 12 years of age and for the treatment of adults with CRSwNP. Depemokimab offers a lower treatment burden compared to existing biologic agents. In clinical trials, depemokimab was administered SC every six months. Cinqair is administered via IV infusion every four weeks, Nucala and Tezspire are administered SC every four weeks, Xolair/Omlyclo is administered SC every two or four weeks, Dupixent is administered SC every two weeks and Fasenra is administered SC every eight weeks. While Cinqair must be administered by a HCP, the other approved agents may be administered by the patient or caregiver. A Phase 1, single-dose study (NCT05602025) demonstrated that depemokimab administered via an autoinjector was bioequivalent to that administered via a safety syringe device in healthy adults, allowing for the potential of depemokimab self-administration via an autoinjector.
FDA approval timeline
Dec. 16, 2025
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $0 |
| 2026 | $187 |
| 2027 | $420 |
| 2028 | $650 |
| 2029 | $847 |
lerodalcibep SC
Manufacturer: LIB Therapeutics
Proposed indications
To reduce low-density lipoprotein cholesterol (LDL-C) for the treatment of patients with atherosclerotic cardiovascular disease (ASCVD), or very high or high risk of ASCVD, and primary hyperlipidemia, including heterozygous familial hypercholesterolemia (HeFH) and those ≥ 10 years of age with homozygous familial hypercholesterolemia (HoFH). Clinical overview
Mechanism of actionLerodalcibep is an adnectin-based, third-generation proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor.
Clinical trial(s)
The global LIBerate program includes Phase 3, double-blind, placebo-controlled trials evaluating the efficacy and safety of lerodalcibep. The LIBerate-CVD (NCT04797247; n=922), LIBerate-HR (NCT04806893; n=922) and LIBerate-HeFH (NCT04797104; n=478) trials enrolled patients with ASCVD or at very high or high risk for ASCVD, including HeFH, on stable maximally tolerated statin therapy with or without other oral agents who require additional LDL-C reduction. Pooled data from the trials revealed a placebo-adjusted reduction in the primary endpoint of LDL-C at trough (week 52) of 62.3% in the per-protocol analysis and 59% in the mITT analysis. The placebo-adjusted LDL-C reduction at peak (2 weeks post dose) in the mITT population was 73.7% at week 50 and the mean of weeks 50 and 52 was 66%. In addition, 90% of patients on lerodalcibep achieved European Society of Cardiology (ESC)/European Atherosclerosis Society (EAS) recommended targets of > 50% additional LDL-C reduction from baseline and LDL-C level < 55 mg/dL. Lerodalcibep was well tolerated. A total of 1,468 patients who completed the LIBerate-CVD and LIBerate-HR trials continued onto the 72-week open-label extension (OLE) trial (NCT04798430). More than a 60% reduction (p<0.0001) in LDL-C from baseline was maintained throughout the additional 72 weeks and over 90% of patients achieved ESC/EAS targets. Lerodalcibep was well tolerated throughout the OLE trial.
The Phase 3, open-label LIBerate-IV (NCT05004675) study was conducted outside the United States and compared lerodalcibep to inclisiran (284 mg on days 1 and 90) in 166 patients with or at very high risk for CVD on high intensity statin requiring additional LDL-C reduction. In the per-protocol population, the mean decrease in LDL-C from baseline at day 270 was 53% with lerodalcibep and 45.3% with inclisiran (p=0.0319). In addition, more patients achieved ESC/EAS targets with lerodalcibep compared to inclisiran (58% versus 42%). Both agents were well tolerated.
The Phase 3, open-label (efficacy parameters masked), crossover, non-inferiority LIBerate-HoFH (NCT04034485) study compared lerodalcibep and evolocumab. In the study, 66 patients ≥ 10 years of age with genetically confirmed HoFH and on oral lipid lowering therapy for at least four weeks were randomized to lerodalcibep or evolocumab (420 mg SC once monthly) for 24 weeks (period A) followed by an eight-week washout and then were crossed over to the alternate therapy for the next 24 weeks (period B). At week 24, the mean reduction in LDL-C in the ITT analysis was -4.9% with lerodalcibep and -10.3% with evolocumab (mean difference, 5.4%), which did not meet the non-inferiority margin of 6%. However, in general, the LDL-C response was similar in the same patients with both agents. Both agents were well tolerated.
The 12-week, open-label, Phase 3 LIBerate-H2H (NCT04790513) study compared lerodalcibep to evolocumab and alirocumab in 204 adults with ASCVD, or at high risk for ASCVD who were on stable high intensity statin therapy with or without ezetimibe (Zetia) and no PCSK9 mAb. Results of the completed study have not been announced.
Dosage and administration
In the LIBerate trials, lerodalcibep 300 mg was administered SC once monthly. Lerodalcibep may be self-administered.
Place in therapy
According to the CDC, about 86 million adults aged ≥ 20 years in the United States have total cholesterol levels > 200 mg/dL and nearly 25 million have total cholesterol levels > 240 mg/dL. Familial hypercholesterolemia (HeFH and HoFH) is a common genetic disease that impairs the breakdown of LDL-C leading to high LDL-C levels in the blood and increased risk of ASCVD. Oral statin therapy, as adjunct to diet and exercise, is the cornerstone of pharmacotherapy for LDL-C lowering, but use can be limited by resistance and intolerance to therapy. When target LDL-C is not achieved despite maximally tolerated statin therapy or the patient is unable to take statin therapy, oral ezetimibe (Zetia, generic), bempedoic acid (Nexletol) or an injectable PCSK9-inhibiting mAb (alirocumab [Praluent], evolocumab [Repatha]) may be added. In addition, inclisiran (Leqvio), a small interfering RNA (siRNA) directed to PCSK9 mRNA, is FDA approved as adjunct to diet and exercise in adults with hypercholesterolemia, including HeFH.
Lerodalcibep is a third-generation PCSK9 inhibitor. If approved, it could compete with Leqvio, Praluent and Repatha as add-on therapy to reduce LDL-C in select patients. Notably, a cross-over study did not demonstrate non-inferiority of lerodalcibep compared to Repatha in patients with HoFH and clinical data comparing lerodalcibep and Praluent have not been published. A clinical trial conducted outside the United States reported greater LDL-C reduction with lerodalcibep compared to Leqvio in patients with or at high risk of ASCVD. Lerodalcibep, Praluent and Repatha may be self-administered SC every four weeks, and the latter two agents also have every-two-week dosing options. Leqvio should be administered by an HCP with maintenance doses given SC every six months.
FDA approval timeline
Dec. 12, 2025Financial forecast (reported in millions)
The projected yearly U.S. sales for lerodalcibep is not available.navepegritide (TransCon CNP) SC
Manufacturer: Ascendis
Proposed indications
Achondroplasia in children
Clinical overview
Mechanism of action
Navepegritide is a prodrug of C-type natriuretic peptide (CNP). It provides continuous exposure of active CNP resulting in inhibition of the fibroblast growth factor receptor 3 (FGFR3) pathway, which is overactive in achondroplasia, and ultimately leads to stimulation of bone growth.
Clinical trials
The Phase 2/3, double-blind, placebo-controlled ApproaCH (NCT05598320) trial evaluated navepegritide in 84 children aged 2–11 years with achondroplasia. Patients were randomized 2:1 to navepegritide or placebo. At baseline, mean age was 5.7 years and mean height was 89 cm. Navepegritide demonstrated a statistically significant greater change in the primary endpoint of annualized growth velocity (AGV) with navepegritide compared to placebo at 52 weeks (AGV, 5.89 versus 4.41 cm/year, respectively; LSM difference, 1.49 cm/year; p<0.0001) in the overall study population. In addition, the change from baseline in achondroplasia (ACH)-specific height Z-score was significantly greater with navepegritide than placebo (LSM difference, 0.28; p<0.0001). Improvements in body proportionality were also seen. Navepegritide significantly reduced tibia-femoral angle (TFA) (LSM difference, -1.81 degrees; p=0.0094) and improved fibula-to-tibia ratio (LSM difference, -0.016; p=0.0001) compared to placebo. Navepegritide was generally well tolerated, with mild to moderate adverse events similar to placebo. Infrequent mild injection site reactions were reported. No cases of symptomatic hypotension or bone-related safety events, such as fractures or acceleration of bone age versus chronological age, were observed.
Dosage and administration
In the ApproaCH trial, navepegritide 100 µg/kg was administered SC by the caregiver once weekly.
Place in therapy
Achondroplasia is the most common type of skeletal dysplasia in humans and is reported in about 1 in 15,000 to 40,000 live births. It is characterized by dwarfism, with large head size (macrocephaly), disproportionate short stature (height below average with limbs shorter than torso) and normal intelligence. Most cases (80%) are not inherited but rather result from new mutations in the fibroblast growth factor receptor 3 (FGFR3) gene causing abnormal formation of cartilage and bone and ultimately shorter bones; however, if both parents have achondroplasia, there is a 50% chance that their children will be affected. Without treatment, adult height is typically below 4 ft 6 in. This abnormal growth leads to skeletal malformations (e.g., curvature of the spine, spinal stenosis, bowed legs) that contribute to challenges of daily activities.
Medical management of achondroplasia varies between individuals and includes spinal decompression, leg straightening, spinal fusion and ear tubes. Use of growth hormone is not recommended in this population as it has the potential to worsen the skeletal disproportion seen in these patients. In 2021, the CNP analog vosoritide (Voxzogo) by Biomarin was granted Accelerated Approval to increase linear growth in pediatric patients with achondroplasia with open epiphyses based on an improvement in AGV. The weight-based dose is administered once daily until closure of epiphyses occurs. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory studies. In the clinical trial in patients ≥ 5 years with achondroplasia, treatment with Voxzogo for 52 weeks resulted in a treatment difference in the change from baseline in AGV of 1.57 cm/year compared to placebo after 52 weeks.
If approved, navepegritide will be the second CNP treatment available in the United States to increase linear growth in children with achondroplasia. It will compete with Voxzogo and provide a once-weekly dosing option compared to Voxzogo which is administered once daily. In non-comparator clinical trials, both agents significantly increased AGV at 52 weeks; however, it is not known whether final adult height will be increased or if there are harms associated with long-term therapy. Like Voxzogo, there is potential for navepegritide to be administered by a caregiver, as demonstrated in clinical trials (e.g., ApproaCH, ACcomplisH).
FDA approval timeline
FDA designations: Orphan Drug, Priority Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $4 |
| 2026 | $45 |
| 2027 | $105 |
| 2028 | $168 |
| 2029 | $243 |
onasemnogene abeparvovec-xioi (Zolgensma, OAV101IT) intrathecal
Proposed indications
Spinal muscular atrophy (SMA)
Clinical overview
Mechanism of action
OAV101IT is an intrathecal (IT) formulation of onasemnogene abeparvovec-xioi (Zolgensma), a single-dose adeno-associated virus (AAV) vector-based gene therapy that delivers a functioning copy of the survival motor neuron 1 (SMN1) gene to the cell nucleus without modifying the existing DNA.
Clinical trial(s)
The Phase 3, double-blind STEER (NCT05089656) trial evaluated OAV101IT in treatment-naïve patients with type 2 SMA, who were 2 to < 18 years of age and were able to sit but did not achieve independent walking. Mean age at dosing was 5.88 years (range, 2.1–16.6). Patients were randomized 3:2 to OAV101IT or sham procedure. At 52 weeks, the OAV101IT group demonstrated a statistically significant improvement by 2.39 points on the primary endpoint of Hammersmith Functional Motor Scale Expanded (HFMSE) compared to 0.51 points in the sham control group (p=0.0074). The most common adverse events for both groups were upper respiratory tract infection and pyrexia, with similar incidence between the treatment groups. The most serious TEAEs with OAV101IT were pneumonia and vomiting, and for sham were pneumonia and lower respiratory tract infection. Increases in transaminases were uncommon and were low-grade and transient in nature.
The Phase 3b, open-label, single-arm STRENGTH (NCT05386680) safety study evaluated OAV101IT in 27 patients 2 to < 18 years of age with SMA who were able to sit but never walked independently. The mean age was 7.4 years (range, 2.4–17.7). Enrolled patients had discontinued treatment with nusinersen or risdiplam. Topline results reported a safety profile like that seen in the STEER trial. Secondary and exploratory efficacy endpoints demonstrated stabilization of motor function over 52 weeks of follow-up, including a 1.05-point increase in the HFMSE from baseline to week 52.
Dosage and administration
In the STEER and STRENGTH trials, OAV101IT was administered intrathecally as a one-time 1.2 x 10¹⁴ vector genomes dose.
Place in therapy
SMA is a rare, debilitating, hereditary disease characterized by progressive motor function decline and muscular atrophy that spares cognitive abilities. Approximately 25,000 people in the United States have SMA. Most cases are caused by mutations in the SMN1 and SMN2 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.
Onasemnogene abeparvovec-xioi (Zolgensma) is a gene replacement therapy FDA approved for one-time IV administration (1.1 x 10¹⁴ vector genomes/kg body weight [vg/kg]) for the treatment of pediatric patients < 2 years of age with SMA with bi-allelic mutations in the SMN1 gene. Zolgensma via IV administration crosses the blood-brain barrier (BBB) to provide a rapid onset and lasting therapeutic effect for treating SMA. Labeling for IV Zolgensma carries a boxed warning regarding cases of acute liver failure with fatal outcomes and the risk for acute serious liver injury and elevated aminotransferases associated with the product.
Children affected with milder forms of SMA (e.g., types 2 and 3) may not be diagnosed until after 2 years of age, beyond the window of approved IV Zolgensma treatment. At this older age, patients would require a higher dose of IV onasemnogene abeparvovec, based on the approved weight-based dosing, which could pose additional safety risks due to greater viral load. The intrathecal formulation of onasemnogene abeparvovec-xioi, OAV101IT, allows for a fixed-dose administration of onasemnogene abeparvovec and a greater neuronal transduction on a vg/kg basis. In preclinical studies, intrathecal injection reduced the amount of viral vector administered by a factor of nearly 10, with equal distribution and efficacy throughout the CNS and reduced viral vector loads in major peripheral organs (e.g., liver).
If approved, OAV101IT could provide an alternative to Zolgensma IV for the treatment of SMA, particularly in older patients who do not qualify for IV administration due to age restrictions. Notably, unlike IV Zolgensma, the STEER and STRENGTH clinical trials did not report liver injury with OAV101IT.
Other disease modifying pharmacological agents for SMA include nusinersen (Spinraza) administered via lumbar puncture and oral risdiplam (Evrysdi), both of which increase the production of the SMN. On Sept. 23, 2025, Scholar Rock received a Complete Response Letter (CRL) from the FDA for the selective latent myostatin inhibitor apitegromab for the treatment of type 2 or 3 SMA. The CRL was based on observations not specific to apitegromab that were identified during a routine general site inspection of a third-party fill-finish facility. No concerns regarding safety or efficacy of apitegromab were cited.
FDA approval timeline
October 2025 – March 2026
FDA designations: Breakthrough Therapy (type 1 SMA only), Fast Track, Orphan Drug
Financial forecast (reported in millions)
The projected yearly U.S. sales for intrathecally administered onasemnogene abeparvovec-xioi (OAV101IT) is not available.
plozasiran SC
Manufacturer: Arrowhead
Proposed indications
Familial chylomicronemia syndrome (FCS)
Clinical overview
Mechanism of action
Plozasiran is a first-in-class, small interfering RNA interference (siRNAi) therapeutic designed to reduce production of apolipoprotein C-III (APOC3), thereby reducing circulating triglycerides (TG).
Clinical trials
The Phase 3, double-blind, placebo-controlled PALISADE (NCT05089084) trial evaluated plozasiran in 75 adults with genetically confirmed or clinically diagnosed FCS. Patients were randomized to receive plozasiran 25 mg (n=26), plozasiran 50 mg (n=24) or placebo (n=25). Enrolled patients had a fasting TG of ≥ 880 mg/dL at screening that was refractory to standard lipid lowering therapy. Median baseline TG levels were 2,008 mg/dL in the plozasiran 25 mg group, 1,902 mg/dL in the plozasiran 50 mg group and 2,053 mg/dL in the placebo group. At 10 months, the study reported the median change from baseline in the fasting TG level (the primary endpoint) was -80% with plozasiran 25 mg, -78% with plozasiran 50 mg, and -17% with placebo (p<0.001 for both doses compared to placebo). Significant TG reductions were observed as early as one month after the first dose and were maintained at month 12. As a secondary endpoint, the percentage of patients who achieved fasting TG < 880 mg/dL at 10 months was 75% in the plozasiran 25 mg group, 55% in the plozasiran 50 mg group and 21% in the placebo group. Similarly, the percentages of patients who attained fasting TG < 500 mg/dL, considered the threshold for pancreatitis, was 50%, 46%, and 5% across the respective groups. A lower incidence of acute pancreatitis was reported among patients who received plozasiran (4%, pooled doses) compared to those who received placebo (20%) (odds ratio, 0.17; p=0.03). The most common TEAE (> 10%) reported with plozasiran that differed from placebo was COVID-19 infection. No reductions in platelet counts were reported.
Dosage and administration
In the PALISADE clinical trial, plozasiran 25 mg and 50 mg doses were administered SC every three months for 12 months. It is uncertain whether plozasiran can be self-administered.
Place in therapy
FCS is a rare, inherited condition characterized by severe hypertriglyceridemia (persistent TG ≥ 880 mg/dL) and is estimated to affect one to 10 individuals per 1,000,000. Most cases of FCS are caused by gene mutations resulting in a lack of a functional lipoprotein lipase (LPL) protein. LPL typically removes TG from chylomicrons circulating in the blood. If this process is not functioning properly, TG breakdown cannot occur and will accumulate in various organs in the body. The most serious complication of FCS is frequent bouts of acute pancreatitis, which can lead to pancreatic damage or diabetes, and can be life-threatening. Other signs and symptoms of FCS include abdominal pain, nausea, fatigue, diarrhea, hepatosplenomegaly, eruptive xanthomas, lipemia retinalis and failure to thrive.
Standard lipid-lowering medications (e.g., statins, fibrates, niacin) and plasmapheresis are generally not effective in patients with FCS. The SOC for FCS is a fat restricted diet (fat < 10% of calories or < 20 grams per day). Patients with FCS should also maintain a healthy weight, incorporate physical activity and avoid processed foods, alcohol and smoking. In 2024, the FDA approved the APOC3-directed antisense oligonucleotide olezarsen (Tryngolza) as an adjunct to diet in adults with FCS as a monthly SC injection (self- or HCP-administered). Notably, volanesorsen by Ionis is an siRNA for APOC3 that is approved for FCS outside the United States; however, while it lowered TG levels below 750 mg/dL in 77% of patients at 3 months, it did not gain FDA approval due to concerns of an increased risk for thrombocytopenia. Unlike volanesorsen, thrombocytopenia was not reported in clinical trials for plozasiran or Tryngolza.
If approved, plozasiran will be the first siRNAi agent that targets APOC3 to reduce TG levels in the blood and could compete with Tryngolza for the treatment of patients with FCS. Tryngolza is administered SC via autoinjector once a month by the patient or caregiver. In clinical trials, plozasiran was administered SC every three months. It remains to be seen if plozasiran will be self-administered.
FDA approval timeline
FDA designations: Breakthrough Therapy, Fast Track, Orphan Drug
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales reported for all lipid lowering proposed indications |
|---|---|
| 2025 | $4 |
| 2026 | $25 |
| 2027 | $87 |
| 2028 | $194 |
| 2029 | $443 |
setmelanotide (Imcivree) SC
Proposed indications
Acquired hypothalamic obesity
Currently indicated to reduce excess body weight and maintain weight reduction long term in patients ≥ 2 years of age with syndromic or monogenic obesity due to:
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Bardet-Biedl syndrome (BBS)
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Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic or of uncertain significance (VUS)
Clinical overview
Mechanism of actionSetmelanotide (Imcivree) is a melanocortin 4 (MC4) receptor agonist.
Clinical trial(s)
The Phase 3, double-blind, randomized, placebo-controlled TRANSCEND (NCT05774756) trial evaluated setmelanotide in 120 patients ≥ 4 years of age (range, 4–66 years) with acquired hypothalamic obesity. Top-line results revealed a statistically significant change from baseline in the primary endpoint of mean change from baseline in BMI in patients who received setmelanotide compared to those who received placebo (-16.5% versus +3.3%, respectively; p<0.0001) at 52 weeks. Change in BMI was similar between pediatric and adult patients. Setmelanotide was generally well tolerated. In addition, about 80% of patients in the setmelanotide group experienced ≥ 5% reduction in BMI compared to 10% in the placebo group. Based on a patient questionnaire using an 11-point numeric rating scale ranging from 0–10, setmelanotide led to a statistically significant reduction in hunger compared to placebo in patients ≥ 12 years of age (-2.7 versus -1.2 points, respectively; p<0.003). In addition, the study reported significant BMI reductions with setmelanotide in patients with prior use or concomitant use of GLP-1 receptor agonists (-24.7% and -27.1% placebo-adjusted differences in BMI reduction from baseline, respectively). The most common TEAEs (> 20%) were nausea, vomiting, diarrhea, injection site reaction, skin hyperpigmentation and headache.
Dosage and administration
In the TRANSCEND trial, setmelanotide was administered by the patient or caregiver via SC injection once daily. No dosage was reported in the published study information.
Place in therapy
The hypothalamus helps regulate both energy intake (food intake) and energy expenditure (calories burned). Acquired hypothalamic obesity occurs due to injury to the hypothalamus that disrupts energy balance. Injury can occur with certain brain tumors (e.g., craniopharyngioma) and related surgery and/or radiation therapy (newer methods may mitigate injury), traumatic brain injury , and infections, inflammation or bleeding in the brain. Depending on the cause, weight gain can begin shortly after the injury (traumatic brain injury or inflammation) or months to years after the injury (radiation). Individuals may exhibit significant weight gain despite reduced calorie intake, uncontrollable hunger, low energy expenditure and fatigue.
Acquired hypothalamic obesity may be diagnosed based on evidence of injury seen on brain imaging, timing of weight gain related to the injury and the presence of hypothalamic/pituitary hormone deficits. Notably, individuals with acquired hypothalamic obesity should be monitored for comorbid conditions such as MASH, T2DM, dyslipidemia, sleep-disordered breathing, pseudotumor cerebri syndrome and psychosocial disorders, which occur at increased rates compared to individuals with polygenic obesity (common obesity due to multiple gene variants and environmental influences) with similar BMI.
There is currently no FDA-approved medication to treat acquired hypothalamic obesity. Management strategies, such as pituitary hormone replacement optimization, diet (lower carbohydrate, portion control), physical activity, mental and behavioral health interventions, may provide modest benefit in some patients. Metabolic/bariatric surgery may provide moderate benefit for select individuals. There is also some evidence that off-label use of medications such as stimulants, metformin and GLP-1 receptor agonists may be of potential benefit in some patients.
If the FDA approves the new indication, setmelanotide (Imcivree) will be the only medication indicated to treat acquired hypothalamic obesity, a condition with considerable unmet need.
FDA approval timeline
Dec. 20, 2025
FDA designations: Breakthrough Therapy, Priority Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales for all obesity indications |
|---|---|
| 2025 | $128 |
| 2026 | $216 |
| 2027 | $387 |
| 2028 | $675 |
| 2029 | $974 |
tividenofusp alfa IV
Proposed indications
Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome
Clinical overview
Mechanism of action
Tividenofusp alfa is a next-generation enzyme replacement therapy (ERT) that contains the recombinant iduronate 2-sulfatase (I2S) enzyme, which breaks down heparan sulfate (HS) and dermatan sulfate in the lysosome to maintain cellular homeostasis. Tividenofusp alfa uses the proprietary TransportVehicle (TV) platform to deliver I2S enzyme across the blood-brain barrier (BBB) and treat neuropathic and systemic forms of MPS II.
Clinical trial(s)
An open-label, Phase 1/2 (NCT04251026) study evalutated tividenofusp alfa in 43 patients ≤ 18 years of age with MPS II. Patients were stratified into multiple cohorts depending on age, MPS II type (neuronopathic, non-neuronopathic) and other patient demographics. Patients receiving standard of care ERT at baseline switched to IV-administered tividenofusp alfa without a washout period. During the 24-week treatment period, various cohorts received doses ranging from 3–30 mg/kg. In the open-label extension, all patients received 15 mg/kg of tividenofusp alfa. Interim data demonstrate a 90.9% mean reduction in cerebral spinal fluid (CSF) HS level from baseline at week 24 with all participants having normal or near normal levels at week 24, which was sustained through week 153 (-89.3%). The mean urine HS was reduced by 84.6% from baseline to week 24 and sustained through week 129 (-80.1%). There was also a significant increase in the proportion of patients with normal total urine glycosaminoglycans (GAGs) from baseline (5–77%) at week 24, which was sustained through week 129. The study also demonstrated a significant reduction from baseline to week 61 (-41.1%) in serum neurofilament light chain (NfL) with continued reduction seen through week 129 (-81.5%, all within normal range), which suggests a decrease in neuronal injury. In addition, adaptive behavior and cognitive scores improved or stabilized. The most common TEAEs were infusion-related reactions, anemia, vomiting, pyrexia, upper respiratory tract infection and rash; the majority being mild to moderate in severity.
The double-blind, active-controlled, Phase 2/3 COMPASS (NCT05371613) study has been initiated to evaluate IV-administered tividenofusp alfa compared to idursulfase in patients 2 to < 6 years of age with neuronopathic MPS II (cohort A) or 6 to < 26 years of age with non-neuropathic MPS II (cohort B). Coprimary outcome measures are the percent change from baseline in CSF HS concentration and change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3) in cohort A. Study completion is anticipated in December 2025.
Dosage and administration
In extension trial, tividenofusp alfa 15 mg/kg with or without prior dose escalation was administered via weekly IV infusion.
Place in therapy
MPS II is a rare, genetic, metabolic condition that almost exclusively affects 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 based on neurologic impact and rate of progression. The severe phenotype, neuropathic MPS II, accounts for about 60% of cases. Signs and symptoms present around 2 to 4 years of age and include hydrocephalus, seizures, behavioral changes, cognitive decline, hearing loss and speech delay. Other organ systems affected include the airway, heart, liver, spleen, eyes, skin, bones and joints. Death due to upper airway disease or cardiovascular failure occurs between 10–20 years of age. Patients with non-neuropathic MPS II experience normal cognitive and intellectual development, although other organ systems are affected and retinal degeneration and neurological symptoms may appear in advanced disease. Diagnosis is often made after 10 years of age. 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, Elaprase led to significant increases in 6MWT and FVC, 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 long-term efficacy is uncertain.
If approved, tividenofusp alfa will be an alternative treatment to Elaprase. Unlike Elaprase, tividenofusp alfa crosses the BBB, but whether this results in improved CNS symptoms has not been studied in a clinical trial.
Nippon Shinyaku and Regenxbio are awaiting FDA approval of their gene therapy, clemidsogene lanparvovec (RGX-121) to treat MPS II. Clemidsogene lanparvovec is administered as a one-time intracisternal injection to deliver a functional copy of the I2S enzyme directly to the brain.
FDA approval timeline
Apr. 5, 2026
FDA designation: Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, Rare Pediatric Disease, seeking Accelerated Approval
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $0 |
| 2026 | $27 |
| 2027 | $86 |
| 2028 | $143 |
| 2029 | $177 |
troriluzole oral
Proposed indications
Spinocerebellar ataxia (SCA)
Clinical overview
Mechanism of action
Troriluzole, a prodrug conjugate of riluzole, reduces glutamate levels in synapses by moderating presynaptic release and facilitating clearance from the synapse.
Clinical trial(s)
The Phase 3, randomized, double-blind, placebo-controlled BHV4157-206 (NCT03701399) trial evaluated troriluzole in 218 adults with SCA (all genotypes). At screening, patients had the ability to ambulate 8 meters without human assistance, a Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) total score ≥ 3 and score of ≥ 1 on the gait subsection of the f-SARA. The f-SARA includes measures for gait, stance, sitting and speech disturbance, with individual items rated 0–4 with total score 0–16. Top-line results showed that the study did not meet the primary endpoint of f-SARA since both the troriluzole and placebo groups displayed minimal change in f-SARA at 48 weeks (5.1 and 5.2 points, respectively; p=0.76). However, post hoc analysis revealed a numerically greater change in f-SARA with troriluzole compared to placebo in patients with SCA type 3 (LSM change difference, -0.55; nominal p=0.053). In addition, the study reported approximately a 58% decrease in fall risk among patients who received troriluzole compared to those given placebo (10% versus 23%, respectively; nominal p=0.043). Troriluzole was well tolerated. TEAEs reported more often with troriluzole than placebo and ≥ 5% were headache, nausea, fatigue and COVID-19. Eligible patients could enroll in the open-label extension (OLE) phase (weeks 1 up to 192).
The BHV4157-206-RWE (NCT06529146) study compared effectiveness of troriluzole in the 3-year OLE Study BHV4157-206 (NCT03701399) to real-world-data (RWD) from external control subjects collected from the U.S. SCA Natural History cohort (Clinical Research Consortium for SCA; CRC-SCA) and European SCA Natural History Cohort (EUROSCA). The study reported troriluzole reduced SCA disease progression by approximately 50% compared to the CRC-SCA control and by approximately 70% compared to the EUROSCA control; troriluzole led to about a 60% decrease in disease progression against pooled external natural history data.
In the BHV4157-206 clinical trial, patients received a starting dose of troriluzole 140 mg orally once daily for the first four weeks, followed by 200 mg once daily for the remaining 44 weeks of the double-blind randomization phase. In the OLE phase, patients received troriluzole 200 mg once daily for up to 192 weeks.
Place in therapy
SCA is a rare, inherited, progressive, neurodegenerative disorder that primarily affects the cerebellum. It affects an estimated 15,000–20,000 people in the United States. SCA is characterized by poor balance and muscle coordination. Several genotypes have been identified, the most common being SCA1, SCA2, SCA3 and SCA6, which make up about half of all cases. Severity and onset of symptoms and rate of progression vary among SCA genotypes.
There is no cure for SCA. Treatment focuses on reducing seizures, tremors, ataxia, depression and ocular manifestations. Speech therapy, occupational therapy and physical therapy are commonly used as well as medications including antiepileptics, botulinum toxin, antidepressants and levodopa.
If approved, troriluzole will be the first and only medication indicated to treat SCA. Troriluzole is also being evaluated in a Phase 3 trial in patients with glioblastoma.
FDA approval timeline
October-December 2025
FDA designations: Fast Track, Orphan Drug, Priority Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2025 | $16 |
| 2026 | $86 |
| 2027 | $164 |
| 2028 | $247 |
| 2029 | $321 |
Keep on your radar
| Drug generic name | Therapeutic category | Oct 2025 pipeline - Total U.S. sales for 2029 (Dollars in millions) |
|---|---|---|
| aficamten | Cardiovascular | $965 |
| anitocabtagene autoleucel | Oncology – Gene therapy | $1,420 |
| atacicept | Immunology | $752 |
| cagrilintide / semaglutide | Endocrine | $9,295 |
| camizestrant | Oncology | $551 |
| cretostimogene grenadenorepvec | Oncology – Gene therapy | $819 |
| denecimig | Hematology | $569 |
| gedatolisib | Oncology | $778 |
| icotrokinra | Immunology | $526 |
| insulin icodec | Endocrine | $701 |
| marnetegragene autotemcel (Kresladi) | Metabolic – Gene therapy | $12 |
| orforglipron | Endocrine | $7,549 |
| veligrotug | Ophthalmology | $701 |
| ziftomenib | Oncology | $282 |
Drug list
Gene and cellular therapies
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| etuvetidigene autotemcel | Fondazione Telethon | Wiskott-Aldrich syndrome | IV | BLA – OD, RMAT, RPD | Oct-Dec 2025 |
| tabelecleucel | Pierre Fabre | Epstein-Barr virus-associated post-transplant lymphoproliferative disease | IV | BLA – BT, OD, PR | 01/10/2026 |
| clemidsogene lanparvovec (RGX-121) | Nippon Shinyaku, Regenxbio | Mucopolysaccharidosis II (Hunter syndrome) | Intrathecal | BLA – FT, OD, PR, RMAT, RPD, seeking AA | 02/08/2026 |
| marnetegragene autotemcel (Kresladi) | Rocket | Leukocyte adhesion deficiency type I | IV | BLA – FT, OD, RMAT, RPD | 03/28/2026 |
| allogeneic T-cell immunotherapy (Orca-T) | Orca | Hematological malignancies (e.g., ALL, AML, myelodysplastic syndromes) | IV | BLA – PR, RMAT | 04/06/2026 |
| vusolimogene oderparepvec (RP1) | Replimune | Melanoma (advanced) | Intratumoral | BLA – BT | 04/10/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| onasemnogene abeparvovec-xioi (Zolgensma) | Novartis | Spinal muscular atrophy | Intrathecal | sBLA – BT, FT, OD | Oct 2025-Mar 2026 |
| lisocabtagene maraleucel (Breyanzi) | Bristol Myers Squibb | Marginal zone lymphoma (R/R, ≥ 2 prior lines of systemic therapy) | IV | sBLA – PR | 12/05/2025 |
| omidubicel-onlv (Omisirge) | Gamida | Aplastic anemia (severe) | IV | sBLA – PR | 12/10/2025 |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| aglatimagene besadenovec (CAN-2409) | Candel | Prostate cancer | Transrectal injection | BLA – FT, RMAT | TBD |
| anitocabtagene autoleucel | Arcellx, Gilead | Multiple myeloma | IV | BLA – FT, OD, RMAT | TBD |
| anzutresgene autoleucel (IMA203) | Immatics | Melanoma | IV | BLA | TBD |
| avalotcagene ontaparvovec (DTX301) | Ultragenyx | Ornithine transcarbamylase deficiency | IV | BLA – FT, OD | TBD |
| baluretgene parvec (OCU400) | Ocugen | Retinitis pigmentosa | Subretinal | BLA – OD, RMAT | TBD |
| bidridistrogene xeboparvovec (SRP-9003) | Sarepta | Limb-girdle muscular dystrophy | IV | BLA – FT, OD, RPD, may seek AA | TBD |
| botaretigene sparoparvovec (AAV-RPGR) | Janssen | Retinitis pigmentosa | Subretinal | BLA – FT, OD | TBD |
| cretostimogene grenadenorepvec | CG Oncology | Bladder cancer | Intravesical | BLA – BT, FT | TBD |
| deramiocel | Nippon Shinyaku, Capricor | DMD-related cardiomyopathy | IV | BLA – OD, RMAT, RPD | TBD |
| giroctocogene fitelparvovec | Sangamo | Hemophilia A | IV | BLA – FT, OD, RMAT | TBD |
| laruparetigene zovaparvovec (AGTC-501) | Beacon | Retinitis pigmentosa | Subretinal | BLA – FT, OD, RMAT | TBD |
| pariglasgene brecaparvovec (DTX401) | Ultragenyx | Glycogen storage disease | IV | BLA – FT, OD | TBD |
| rexlemestrocel-L | Mesoblast | End-stage ischemic HFrEF implanted with an LVAD | IM | BLA – OD, RMAT, may seek AA | TBD |
| RGX-202 | Regenxbio | DMD | IV | BLA – FT, OD, RPD | TBD |
| surabgene lomparvovec (ABBV-RGX-314) | Abbvie | DME; Wet AMD | Subretinal | BLA – OD | TBD |
Biosimilars
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| aflibercept (biosimilar to Regeneron's Eylea) | Alvotech, Teva | Wet AMD; Macular edema following RVO; DME; Diabetic retinopathy | Intravitreal | BLA | Oct–Dec 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Amneal, mAbxience | Osteoporosis; Bone cancer | SC | BLA | Oct–Dec 2025 |
| denosumab (biosimilar to Amgen's Prolia, Xgeva) | Teva | 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 | Breast cancer (HER2+) | IV | BLA | December 2025 |
| insulin aspart (biosimilar to Novo Nordisk's Novolog) | Amphastar | T1DM; T2DM | SC | BLA | Jan–Mar 2026 |
| golimumab (biosimilar to Janssen's Simponi) | Bio-Thera | RA; AS; PsA; UC | SC | BLA | 05/16/2026 |
| omalizumab (biosimilar to Genentech's Xolair) | Amneal | Urticaria | SC | BLA | September 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 |
None
Specialty
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| ataluren (Translarna) | PTC | DMD (nonsense mutation) | Oral | NDA – FT, OD | Oct-Dec 2025 |
| ketamine (NRX-100) | Hope | Suicidal depression | IV | NDA – FT, PR, seeking AA | Oct-Dec 2025 |
| sevabertinib | Bayer | NSCLC (advanced, HER2 mutation, 2nd-line) | Oral | NDA – BT, PR | Oct-Dec 2025 |
| troriluzole | Biohaven | Spinocerebellar ataxia | Oral | 505(b)(2) NDA – FT, OD, PR | Oct-Dec 2025 |
| bitopertin | Disc Medicine | Porphyria | Oral | NDA – CNPV, OD, RPD, seeking AA | Nov–Dec 2025 |
| plozasiran | Arrowhead | Familial chylomicronemia syndrome | SC | NDA – BT, FT, OD | 11/18/2025 |
| navepegritide | Ascendis | Achondroplasia | SC | NDA – OD, PR | 11/28/2025 |
| sibeprenlimab | Otsuka | IgA nephropathy (Berger's disease) | IV, 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 with nasal polyps (add-on maintenance, adults) | SC | BLA | 12/16/2025 |
| aficamten | Cytokinetics | Obstructive hypertrophic cardiomyopathy | Oral | NDA – BT, OD | 12/26/2025 |
| narsoplimab | Omeros | HSCT-associated thrombotic microangiopathy | IV | BLA – BT, OD | 12/26/2025 |
| fibrinogen | Grifols | Fibrinogen deficiency (acquired and congenital) | IV | BLA | 12/27/ 2025 |
| tolebrutinib | Sanofi | MS (non-relapsing secondary progressive) | Oral | NDA – BT, PR | 12/28/2025 |
| relacorilant | Corcept | Cushing's syndrome | Oral | NDA – OD | 12/30/2025 |
| camizestrant | AstraZeneca | Breast cancer (advanced, HR+, HER2-, ESR1-mutated) | Oral | NDA | Jan–Jun 2026 |
| idebenone | Chiesi | Leber's hereditary optic neuropathy | Oral | NDA – OD, PR | 02/28/2026 |
| linerixibat | GlaxoSmithKline | Cholangitis pruritus | Oral | NDA – OD | 03/24/2026 |
| pegargiminase | Polaris | Mesothelioma (malignant pleural, non-epithelioid histology) | IM | BLA – FT, OD | Apr-Jun 2026 |
| sasanlimab | Pfizer | Bladder cancer (high-risk, non-muscle-invasive, in combination with BCG) | SC | BLA | Apr-Jun 2026 |
| tividenofusp alfa | Denali | Mucopolysaccharidosis II (Hunter syndrome) | IV | BLA – BT, FT, OD, PR, RPD, seeking AA | 04/05/2026 |
| nilotinib | Xspray | CML | Oral | 505(b)(2) NDA – OD | 06/18/2026 |
| vepdegestrant | Arvinas, Pfizer | Breast cancer (advanced or metastatic, HR+, HER2-, ESR1-mutated, prior ERT) | Oral | NDA – FT | 06/05/2026 |
| sirolimus/pegadricase | SOBI | Gout | IV | BLA – FT | 06/27/2026 |
| relacorilant | Corcept | Ovarian cancer (platinum-resistant) | Oral | NDA – OD | 07/11/2026 |
| icotrokinra | Janssen | PSO (ages ≥ 12 years) | Oral | NDA | 07/21/2026 |
| denecimig | Novo Nordisk | Hemophilia A | SC | NDA – OD | 09/29/2026 |
| pivekimab sunirine | Abbvie, Jazz | Blastic plasmacytoid dendritic cell neoplasm | IV | NDA – BT, OD | 09/30/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| aflibercept (Eylea HD) 8 mg | Regeneron | Macular edema from RVO (8 mg dose) | Intravitreal | sNDA – PR | Oct-Dec 2025 |
| durvalumab (Imfinza) | AstraZeneca | Gastric and gastroesophageal junction cancers (resectable, early-stage and locally advanced) | SC | sBLA – BT, PR | Oct-Dec 2025 |
| teplizumab-mzwv (Tzield) | Sanofi | T1DM (delay progression of stage 3) | IV | sBLA – BT, CNPV, OD, seeking AA | Nov-Dec 2025 |
| mitapivat (Pyrukynd) | Agios | Thalassemia (alpha or beta, non-transfusion-dependent and transfusion-dependent) | Oral | sNDA – OD | 12/07/2025 |
| berotralstat (Orladeyo) granule | Biocryst | HAE (ages 2–11 years) | Oral | sNDA – FT, OD, PR | 12/12/2025 |
| inebilizumab-cdon (Uplizna) | Amgen | Myasthenia gravis | IV | sBLA – OD | 12/14/2025 |
| setmelanotide (Imcivree) | Rhythm | Acquired hypothalamic obesity | SC | sNDA – BT, PR | 12/20/2025 |
| mosunetuzumab-axgb (Lunsumio) | Genentech, Biogen | Follicular lymphoma (≥ 3rd-line) | SC | sBLA – BT, OD | 12/22/2025 |
| nerandomilast (Jascayd) | Boehringer Ingelheim | Progressive pulmonary fibrosis | Oral | sNDA – BT, OD | Jan-Mar 2026 |
| imiglucerase (Cerezyme) | Sanofi | Gaucher's disease (types 1 and 3) | IV | sNDA – OD | 01/13/2026 |
| sparsentan (Filspari) | Travere, Bristol Myers Squibb | Focal segmental glomerulosclerosis | Oral | sNDA – OD | 01/13/2026 |
| fam-trastuzumab deruxtecan-nxki (Enhertu) | Daiichi Sankyo, AstraZeneca | Breast Cancer (unresectable/metastatic, HER2+, with pertuzumab, 1st-line) | IV | sBLA – BT, PR, RTOR | 01/23/2026 |
| leniolisib (Joenja) | Pharming, Novartis | Activated PI3K delta syndrome (ages 4–11 years) | Oral | sNDA – OD, PR | 01/31/2026 |
| dupilumab (Dupixent) | Sanofi | Urticaria (ages 2–11 years) | SC | sBLA | Feb-Apr 2026 |
| niraparib/abiraterone (Akeega) | Janssen | Prostate cancer (M1 metastatic, castration-sensitive) | Oral | sNDA | Feb-Apr 2026 |
| pembrolizumab (Keytruda) | Merck | Ovarian cancer (in combination with chemotherapy ± bevacizumab, platinum-resistant) | IV | sBLA – PR | 02/20/2026 |
| decitabine/cedazuridine (Inqovi) | Otsuka | AML (newly diagnosed, ineligible for intensive induction chemotherapy, in combination with venetoclax) | Oral | sNDA | 02/25/2026 |
| deucravacitinib (Sotyktu) | Bristol Myers Squibb | PsA | Oral | sNDA | 03/26/2026 |
| enfortumab vedotin-ejfv (Padcev) | Astellas | Bladder cancer (muscle invasive, neoadjuvant/adjuvant, with pembrolizumab, ineligible for cisplatin) | IV | sBLA – BT, PR | 04/07/2026 |
| ustekinumab (Stelara) | Janssen | CD (ages 2–17 years) | IV, SC | sBLA – OD | 04/17/2026 |
| fam-trastuzumab deruxtecan-nxki (Enhertu) | Daiichi Sankyo, AstraZeneca | Breast cancer (neoadjuvant, HER2+, stage 2–3, followed by paclitaxel, trastuzumab & pertuzumab) | IV | sBLA – BT, PR, RTOR | 05/18/2026 |
| acalabrutinib (Calquence) | AstraZeneca | CLL (treatment-naïve, in combination with venetoclax) | Oral | sNDA – OD | 05/29/2026 |
| guselkumab (Tremfya) | Janssen | PsA (inhibition of structural damage) | IV, SC | sBLA | 05/29/2026 |
| venetoclax (Venclexta) | Abbvie | CLL (treatment-naïve, in combination with acalabrutinib) | Oral | sNDA – BT, OD | 05/29/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designation | FDA decision |
|---|---|---|---|---|---|
| abelacimab | Novartis | Stroke prevention in atrial fibrillation | IV, SC | BLA – FT | 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) | 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, OD, RPD | TBD |
| brepocitinib | Priovant, Pfizer | Dermatomyositis | Oral | NDA – OD | TBD |
| buntanetap | Annovis | Alzheimer's disease; Parkinson's disease | Oral | NDA | TBD |
| carbetocin | Acadia | Prader-Willi syndrome | Intranasal | NDA – FT, OD | TBD |
| condoliase | Ferring | Sciatica | Intervertebral disc injection | BLA | TBD |
| delpacibart braxlosiran | Avidity | Facioscapulohumeral muscular dystrophy | IV | NDA – FT, OD, may seek AA | TBD |
| delpacibart etedesiran | Avidity | Myotonic muscular dystrophy | IV | BLA – BT, FT, OD | TBD |
| depemokimab | GlaxoSmithKline | Eosinophilic granulomatosis with polyangiitis | SC | BLA | TBD |
| deucrictibant | Pharvaris | HAE | Oral | NDA – OD | TBD |
| divarasib | Genentech | NSCLC | Oral | NDA – BT | 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 |
| glepaglutide | Zealand | Short bowel syndrome | SC | NDA – OD | TBD |
| glutamate decarboxylase | Diamyd | T1DM | Intralymphatic, SC | NDA – FT, OD, may seek AA | TBD |
| gold nanocrystal | Clene | ALS | Oral | NDA – OD | TBD |
| govorestat | Applied | Sorbitol dehydrogenase (SORD) deficiency | Oral | NDA – OD | TBD |
| hydromethylthionine mesylate | Taurx | Alzheimer's disease | Oral | NDA | TBD |
| hydroxypropyl-beta-cyclodextrin | Rafael | Niemann-Pick disease (type C) | IV | NDA – FT, OD, RPD | TBD |
| ianalumab | Novartis | ITP; Sjogren's syndrome | SC | BLA – FT, OD | TBD |
| imlifidase | Sarepta | Kidney transplant rejection | IV | BLA – FT, OD, may seek AA | TBD |
| immune globulin/hyaluronidase | Takeda | Primary immunodeficiencies | SC infusion | BLA | TBD |
| immune-modulating cancer vaccine | IO Biotech | Melanoma | SC | BLA – BT | TBD |
| imsidolimab | Vanda | Generalized pustular psoriasis | 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, Biocon | GVHD treatment | IV | BLA – FT | TBD |
| ivonescimab | Akeso | NSCLC | IV | BLA – FT | TBD |
| lanifibranor | Inventiva | MASH | Oral | NDA – BT, FT, may seek AA | TBD |
| latozinemab | Alector, GlaxoSmithKline | Frontotemporal dementia | IV | BLA – BT, FT, OD | TBD |
| lonvoguran ziclumeran | Intellia | HAE | IV | BLA – OD, RMAT | TBD |
| molgramostim | Savara | Acute pulmonary alveolar proteinosis | Inhaled | BLA – BT, FT, OD | TBD |
| obefazimod | Abivax | UC | Oral | NDA | TBD |
| OCU410ST | Ocugen | Stargardt disease | SC infusion | BLA – OD, RPD | 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 (EGFR-mutated) | IV | BLA – BT | TBD |
| pelacarsen | Novartis | Dyslipidemia | SC | NDA – FT | TBD |
| povetacicept | Vertex | IgA nephropathy (Berger's disease) | SC | NDA – BT | TBD |
| povorcitinib | Incyte | Hidradenitis suppurativa | Oral | NDA | TBD |
| REGN-5713-5715 | Regeneron | Birch allergy | SC | BLA | TBD |
| remibrutinib | Novartis | MS | Oral | NDA | TBD |
| riliprubart | Sanofi | Chronic inflammatory demyelinating polyneuropathy | IV, SC | BLA – OD | TBD |
| rocatinlimab | Amgen | Atopic dermatitis | SC | BLA | TBD |
| rusfertide | Takeda | Polycythemia vera | SC | NDA – FT, OD | TBD |
| saroglitazar | Zydus | Primary biliary cholangitis | Oral | NDA – FT, OD | TBD |
| sefaxersen | Genentech | IgA nephropathy (Berger's disease) | SC | NDA | TBD |
| serplulimab | Henlius | SCLC | IV | BLA – OD | TBD |
| suramin | Kuvatris | Human African sleeping sickness | IV | NDA – OD | TBD |
| tinlarebant | Belite | Stargardt disease | Oral | NDA – BT, FT, OD, RPD | TBD |
| vamikibart | Genentech | Uveitic macular edema | Intravitreal | BLA – OD | TBD |
| veligrotug | Viridian | Thyroid eye disease | IV | BLA – BT | TBD |
| venglustat | Sanofi | Fabry disease; Gaucher 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 | SC | BLA | TBD |
| zaltenibart | Omeros | PNH | IV | BLA – OD | TBD |
| zasocitinib | Takeda | PSO | Oral | NDA | TBD |
| zelenectide pevedotin | Bicycle | Bladder cancer | IV | NDA – FT | TBD |
| zilganersen | Ionis | Alexander disease | Intrathecal | NDA – FT, OD | TBD |
| ziltivekimab | Corvidia | CVD | SC | BLA | TBD |
| zimislecel | Vertex, Novartis | T1DM | Hepatic portal vein infusion | BLA – FT, RMAT | 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 | Bladder cancer (high risk, muscle-invasive) | IV | sBLA | TBD |
| brolucizumab-dbll (Beovu) | Novartis | Diabetic retinopathy | Intravitreal | sBLA | TBD |
| cemiplimab-rwlc (Libtayo) | Regeneron | Melanoma | IV | sBLA – FT | TBD |
| crovalimab-akkz (Piasky) | Genentech | Hemolytic uremic syndrome | IV, SC | sBLA | TBD |
| eplontersen (Wainua) | Ionis | Transthyretin amyloid cardiomyopathy | 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, Biogen | DLBCL (2nd-line) | 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; SLE | IV | sBLA – OD | TBD |
| olezarsen (Tryngolza) | Akcea | Severe hypertriglyceridemia | SC | sNDA | TBD |
| pitolisant (Wakix) | Harmony | Excessive sleepiness associated with medical conditions | Oral | sNDA – OD | TBD |
| ropeginterferon alfa-2b-njft (Besremi) | Pharmaessentia | Essential thrombocythemia | SC | sBLA – OD | TBD |
| satralizumab-mwge (Enspryng) | Genentech | Myelin oligodendrocyte glycoprotein antibody-associated disease; Thyroid eye disease | SC | sBLA – OD | TBD |
| secukinumab (Cosentyx) | Novartis | Giant cell arteritis; Polymyalgia rheumatica | SC | sBLA | TBD |
| treprostinil sodium (Tyvaso) | United Therapeutics | 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 |
|---|---|---|---|---|---|
| carbidopa/levodopa | Mitsubishi Tanabe | Parkinson's disease motor fluctuations | SC | 505(b)(2) NDA | Oct-Dec 2025 |
| semaglutide (Wegovy) 25 mg oral | Novo Nordisk | Obesity | Oral | NDA | Oct-Dec 2025 |
| elinzanetant | Bayer | Menopause-related vasomotor symptoms | Oral | NDA | 10/30/2025 |
| lerodalcibep | LIB | LDL-C reduction (patients with ASCVD, or very high or high risk of ASCVD); HeFH; HoFH (ages ≥ 10 years) | 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 | Topical | NDA | 12/16/2025 |
| tradipitant | Vanda, Eli Lilly | Motion sickness | Oral | NDA | 12/30/2025 |
| brimonidine/carbachol | Visus | Presbyopia | Topical | 505(b)(2) NDA | 01/28/2026 |
| epinephrine film | Aquestive | Type 1 allergic reaction | SL | 505(b)(2) NDA – FT | 01/31/2026 |
| milsaperidone | Vanda | Bipolar I disorder; Schizophrenia | Oral | NDA | 02/21/2026 |
| desmopressin oral solution | Eton | Central diabetes insipidus | Oral | 505(b)(2) NDA | 02/25/2025 |
| insulin icodec | Novo Nordisk | T2DM | SC | BLA | March 2026 |
| nimodipine | Grace | Subarachnoid hemorrhage | IV | 505(b)(2) NDA – FT, OD | 04/23/2025 |
| doravirine/islatravir | Merck | HIV-1 infection (adults) | Oral | NDA | 04/28/2025 |
| dexmethylphenidate IR/ER formulation | Cingulate | ADHD | Oral | 505(b)(2) NDA | 05/31/2025 |
| ensitrelvir | Shionogi | COVID-19 post-exposure prophylaxis | Oral | NDA – FT | 06/16/2026 |
| cytisinicline | Achieve Life | Smoking cessation (nicotine dependence) | Oral | NDA – BT | 06/20/2026 |
| tavapadon | Abbvie | Parkinson's disease | Oral | NDA | 09/25/2026 |
| apixaban oral dissolving film | Taho | Thromboembolic disorders | Oral | 505(b)(2) NDA | 10/01/2026 |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designations | FDA decision |
|---|---|---|---|---|---|
| semaglutide (Ozempic) inj. | Novo Nordisk | PAD (with T2DM) | SC | sNDA | Oct-Dec 2025 |
| semaglutide (Wegovy) inj. | Novo Nordisk | Chronic HFpEF (with obesity) | SC | sNDA | Oct-Dec 2025 |
| flibanserin (Addyi) | Sprout | Female sexual dysfunction | Oral | sNDA – PR | Nov-Dec 2025 |
| nirmatrelvir/ritonavir (Paxlovid) | Pfizer | COVID-19 treatment (high-risk children) | Oral | sNDA – FT | Nov-Dec 2025 |
| celecoxib (Elyxyb) ready-to-use oral solution | Scilex | Acute pain | Oral | sNDA | 11/21/2025 |
| gepotidacin (Blujepa) | GlaxoSmithKline | Urogenital gonorrhea (uncomplicated, ages ≥ 12 years) | Oral | sNDA – QIDP, PR | 12/11/2025 |
| ferric maltol (Accrufer) | Shield | Anemia (ages ≥ 10 years) | Oral | sNDA – PR | Jan-Mar 2026 |
| RSV vaccine, adjuvanted (Arexvy) | GlaxoSmithKline | RSV immunization (ages 18–49 years, increased risk) | IM | sBLA – FT | Jan-Jun 2026 |
| tirzepatide (Mounjaro) | Eli Lilly | T2DM (pediatric) | SC | sNDA | Feb-Apr 2026 |
| lumateperone (Caplyta) | Johnson & Johnson | Schizophrenia (relapse prevention) | Oral | sNDA | 05/08/2026 |
| human insulin (Afrezza) | MannKind | T1DM (ages 4–17 years); T2DM (ages 4–17 years) | Inhaled | sNDA | 05/29/2026 |
| 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 | Focal-onset seizures; MDD | Oral | NDA | TBD |
| brilaroxazine | Reviva | Schizophrenia | 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 | Postsurgical pain | Oral | NDA | TBD |
| centanafadine | Otsuka | ADHD | Oral | NDA | TBD |
| cytomegalovirus vaccine (mRNA-1647) | Moderna | Cytomegalovirus infection prevention | IM | BLA | TBD |
| dipalmitoyl hydroxyproline (QRX003) | Quoin | Netherton syndrome | Topical | NDA – RPD | TBD |
| doxycycline | Polypid | Surgical site infection prevention (abdominal colorectal surgery-related) | Surgical implant | NDA – BT, FT, QIDP | TBD |
| influenza/COVID-19 vaccine (mRNA-1083) | Moderna | COVID-19/influenza immunization | IM | BLA – FT | TBD |
| insulin efsitora alfa | Eli Lilly | T2DM | SC | BLA | TBD |
| lorundrostat | Mineralys | Hypertension | Oral | NDA | 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 prevention | IM | NDA | TBD |
| obicetrapib | New Amsterdam | Dyslipidemia | Oral | NDA | TBD |
| orforglipron | Eli Lilly | Obesity; OSA (with obesity); T2DM | Oral | NDA | TBD |
| oveporexton | Takeda | Narcolepsy | Oral | NDA – BT | TBD |
| PL-9643 | Palatin | DED | Topical | NDA | TBD |
| pritelivir | Aicuris | HSV treatment | Oral | NDA – BT, FT | TBD |
| quadrivalent influenza mRNA vaccine (mRNA-1010) | Moderna | Seasonal influenza vaccination | IM | BLA | TBD |
| rabies vaccine (vero cell-derived) | Sanofi | Rabies prevention (pre- and post-exposure) | IM | BLA | TBD |
| ralinepag | United Therapeutics | PAH | Oral | NDA – OD | TBD |
| reboxetine | Axsome, Pfizer | Narcolepsy | Oral | NDA – BT, OD | TBD |
| relutrigine | Praxis | Seizure disorders | Oral | NDA – BT, OD, RPD | TBD |
| retatrutide | Eli Lilly | Obesity; CVD (with obesity); Osteoarthritis of knee (with obesity); T2DM; Diabetic retinopathy | SC | NDA | TBD |
| RSV live attenuated vaccine (SP0125) | Sanofi | RSV immunization | Intranasal | BLA | TBD |
| semaglutide / insulin icodec | Novo Nordisk | T2DM | SC | BLA | TBD |
| survodutide | Zealand, Boehringer Ingelheim | MASH; Obesity; CVD (with obesity) | SC | NDA – BT | TBD |
| tebipenem pivoxil | GlaxoSmithKline | UTI (complicated) | Oral | NDA – FT, QIDP | TBD |
| tegoprazan | Sebela | Erosive esophagitis; Non-erosive reflux disease | Oral | NDA | TBD |
| tiratricol | Egetis | Monocarboxylate transporter 8 (MCT8) deficiency | Oral | NDA – BT, FT, OD, RPD | TBD |
| ulixacaltamide | Praxis | Essential tremor | Oral | NDA | TBD |
| valiltramiprosate | Alzheon | Alzheimer's disease | Oral | NDA – FT | TBD |
| yellow fever vaccine (SP0218) | Sanofi | Yellow fever immunization | SC | BLA | TBD |
| Name | Manufacturer | Clinical use | Dosage form | Development status - FDA designation | FDA decision |
|---|---|---|---|---|---|
| semaglutide (Ozempic) | Novo Nordisk | Diabetic retinopathy | SC | sNDA | TBD |
| semaglutide (Rybelsus) | Novo Nordisk | Alzheimer's disease | Oral | sNDA | TBD |
| semaglutide (Wegovy) | Novo Nordisk | Osteoarthritis of knee (with obesity) | SC | sNDA | TBD |
| tirzepatide (Mounjaro) | Eli Lilly | T2DM and CV outcomes | SC | sNDA | TBD |
| tirzepatide (Zepbound) | Eli Lilly | CKD (with obesity/overweight); HFpEF (with obesity) | SC | sNDA | TBD |
| Name | Manufacturer | Clinical use | Dosage form | Development status |
|---|---|---|---|---|
| apitegromab | Scholar Rock | Spinal muscular atrophy | IV | CRL |
| bevacizumab-vikg | Outlook | Wet AMD | Intravitreal | CRL |
| brexpiprazole (Rexulti) | Otsuka | PTSD (in combination with sertraline) | Oral | CRL |
| copper histidinate | Zydus | Menkes disease | SC | CRL |
| dasatinib (amorphous formulation) | Handa | CML | Oral | CRL |
| dasiglucagon (Zegalogue) | Zealand | Congenital Hyperinsulinism (dosing up to 3 weeks) | SC | CRL |
| nusinersen (Spinraza) high dose | Ultragenyx | Spinal muscular atrophy | Intrathecal | CRL |
| ranibizumab (biosimilar to Genentech's Lucentis) | Stada, Xbrane | DME; Diabetic retinopathy; Macular edema from RVO; Myopic choroidal neovascularization; Wet AMD | Intravitreal | CRL |
| sodium dichloroacetate (SL-1009) | Saol | Pyruvate dehydrogenase complex deficiency | Oral | CRL |
| tradipitant | Vanda, Eli Lilly | Gastroparesis | Oral | CRL |
| vatiquinone | PTC | Friedreich's ataxia | Oral | CRL |