Several US Food and Drug Administration (FDA) regulatory decisions are expected in November, including potential new therapies for lung and breast cancers, sickle cell disease, rare neuromuscular disorders, and kidney disease.

Hematology

Mitapivat

Agios announced a November 1, 2026, PDUFA goal date for its supplemental new drug application for mitapivat (Pyrukynd), an oral pyruvate kinase activator, to treat sickle cell disease. The FDA accepted the application and granted Priority Review under the accelerated approval pathway. In the phase 3 RISE UP trial, In the phase 3 RISE UP trial, two primary endpoints were evaluated: hemoglobin response and annualized rate of sickle cell pain crises. The ongoing phase 3 REIGNITE trial is evaluating whether mitapivat reduces transfusion burden in patients with sickle cell disease.

Source: Agios

Oncology

Ivonescimab

Summit Therapeutics announced a November 14, 2026, PDUFA goal date for the biologics license application for ivonescimab, an investigational PD-1/VEGFbispecific antibody, in combination with chemotherapy for patients with EGFR-mutated locally advanced or metastatic nonsquamous non-small cell lung cancer following tyrosine kinase inhibitor treatment. The FDA accepted the application for review. The submission is supported by the global phase 3 HARMONi trial, which evaluated ivonescimab plus chemotherapy against placebo plus chemotherapy in patients previously treated with a third-generation EGFR inhibitor.

Source: Summit Therapeutics

Neladalkib

Nuvalent announced a November 27, 2026, PDUFA target action date for the New Drug Application for neladalkib, an investigational ALK-selective inhibitor, to treat patients with tyrosine kinase inhibitor (TKI)- pretreated advanced ALK-positive non-small cell lung cancer. The FDA accepted the application for filing and granted Priority Review. The submission is supported by data from TKI-pretreated patients enrolled in the global, registration-directed Phase 1/2 ALKOVE-1 clinical trial. The FDA previously granted neladalkib Breakthrough Therapy designation for patients with locally advanced or metastatic ALK-positive non-small cell lung cancer previously treated with two or more ALK TKIs, as well as Orphan Drug designation.

Source: Nuvalent

Giredestrant

Roche announced a November 30, 2026,  PDUFA goal date for its new drug application for giredestrant, an investigational oral selective estrogen receptor degrader, as adjuvant treatment for adults with estrogen receptor-positive, HER2-negative stage I to III breast cancer. The FDA accepted the application and granted Priority Review. In the phase 3 lidERA Breast Cancer trial, giredestrant reduced the risk of invasive disease recurrence or death by 30% compared with standard endocrine therapy. At 3 years, invasive disease-free survival was 92.4% with giredestrant and 89.6% with standard endocrine therapy.

Source: Roche

Bezuclastinib

Cogent Biosciences announced a November 30, 2026, PDUFA goal date for its new drug application for bezuclastinib in combination with sunitinib to treat gastrointestinal stromal tumors following prior imatinib treatment. The FDA accepted the application and granted Priority Review. In the phase 3 PEAK trial, median progression-free survival was 16.5 months with bezuclastinib plus sunitinib compared with 9.2 months with sunitinib alone. Objective response rates were 46% and 26%, respectively. The FDA previously granted the combination Breakthrough Therapy designation.

Source: Cogent Biosciences

Cardiology

Aficamten

Cytokinetics announced a November 14, 2026, PDUFA goal date for a supplemental new drug application (sNDA) for aficamten (Myqorzo), a cardiac myosin inhibitor approved for adults with symptomatic obstructive hypertrophic cardiomyopathy. The FDA accepted the supplemental application for review based on data from the phase 3 MAPLE-HCM trial, which evaluated aficamten monotherapy compared directly against metoprolol. If approved, this supplemental filing would expand the prescribing label for aficamten by incorporating pivotal head-to-head evidence against standard-of-care monotherapy.

Source: Cytokinetics

Neurology

Venglustat

Sanofi announced a November 25, 2026, PDUFA target action date for its New Drug Application for venglustat, an investigational oral glucosylceramide synthase inhibitor, to treat type 3 Gaucher disease (GD3). The FDA accepted the application with Priority Review. If approved,venglustat could become the first US treatment specifically addressing progressive neurological manifestations of GD3. The application is supported by the phase 3 LEAP2MONO study, an active-comparator trial evaluating venglustat against enzyme replacement therapy in patients aged 12 years and older. The study met both primary endpoints measuring changes in ataxia (SARA) and neurocognitive performance (RBANS) at week 52.Venglustat previously earned FDA Breakthrough Therapy, Fast Track, and Orphan designations.

Source: Sanofi

Deramiocel

Capricor Therapeutics announced a November 22, 2026,PDUFA target action date for its Biologics License Application (BLA) for deramiocel, an investigational cell therapy for Duchenne muscular dystrophy. The FDA extended its review period following a major amendment submission containing additional phase 3 HOPE-3 trial data to support a refined indication for upper limb function. The submission features 24-month open-label extension findings evaluated by the Performance of the Upper Limb score.Capricor reported that patients transitioning from 12 months of placebo to deramiocel experienced a notable slowing in disease progression, demonstrating lower rates of upper limb function decline compared to natural history projections

Source: Capricor Therapeutics

BBP-418

BridgeBio Pharma announced a November 27, 2026,  PDUFA target action date for its new drug application for BBP-418, an investigational oral treatment for limb-girdle muscular dystrophy type 2I/R9. The FDA accepted the application and granted Priority Review. The submission is supported by the phase 3 FORTIFY trial, which met all prespecified primary and secondary end points in a 12-month interim analysis. BridgeBio reported improvements across measures of muscle function and disease biomarkers among treated patients, while placebo recipients experienced deterioration. No treatments are currently FDA-approved specifically for this condition.

Source: BridgeBio Pharma

Pulmonology

Molgramostim

Savara announced a November 22, 2026, PDUFA target action date for its Biologics License Application (BLA) for molgramostim inhalation solution, an investigational recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) therapy for autoimmune pulmonary alveolar proteinosis (autoimmune PAP). The FDA granted Priority Review and extended the review period by three months after determining that responses to information requests constituted a major amendment. The FDA cited no safety, efficacy, or manufacturing concerns in connection with the extension.Molgramostim holds FDA Breakthrough Therapy, Fast Track, and Orphan Drug designations for autoimmune PAP, a rare lung disorder driven by autoantibodies against GM-CSF that impair surfactant clearance in the alveoli.

Source: Savara

Nephrology

Povetacicept

Vertex Pharmaceuticals announced a November 30, 2026 PDUFA target action date for its Biologics License Application (BLA) seeking accelerated approval of povetacicept, an investigational dual BAFF/APRIL antagonist, for adults with immunoglobulin A nephropathy (IgAN). The FDA accepted the BLA for review under the accelerated approval pathway. The submission is supported by a prespecified 36-week interim analysis of the Phase 3 RAINIER trial, where once-monthly subcutaneous povetacicept achieved a 52.0% reduction in urine protein-to-creatinine ratio (UPCR) from baseline, corresponding to a 49.8% placebo-adjusted reduction. The BLA targets accelerated approval based on proteinuria reduction as a surrogate endpoint, while the study continues toward its final 104-week analysis evaluating eGFR slope to confirm long-term kidney function preservation.

Source: Vertex Pharmaceuticals

Surgery

D-PLEX100

PolyPid announced a November 28, 2026 PDUFA target action date for its New Drug Application (NDA) for D-PLEX100, an investigational locally administered, extended-release doxycycline formulation designed to prevent surgical site infections (SSIs) in patients undergoing abdominal colorectal surgery. The FDA accepted the application and granted Priority Review. The submission is supported by the pivotal Phase 3 SHIELD II trial, which met its primary and key secondary endpoints, demonstrating a 60% relative risk reduction in surgical site infections compared to standard of care. D-PLEX100 previously received FDA Breakthrough Therapy Designation for preventing SSIs in elective colorectal surgery.

Source: PolyPid