FDA Decisions Expected: November 2026

Drug pipeline for November 2026

At Prime Therapeutics (Prime), we have positioned ourselves to best prepare our clients to manage new drugs. Our clinical and trade relations teams keep a keen eye on drugs awaiting approval by the Food and Drug Administration (FDA).

November 2026: asundexian

Bayer's asundexian is under FDA Priority Review for the prevention of secondary ischemic stroke following noncardioembolic ischemic stroke or high-risk transient ischemic attack (TIA). The Factor XIa inhibitor received Fast Track designation and was evaluated in the Phase 3 OCEANIC-STROKE trial.¹ Adults (n=12,327) were randomized to oral asundexian 50 mg once daily or placebo within 72 hours after the onset of a noncardioembolic ischemic stroke or high-risk TIA. At a median follow-up of nearly 19 months, asundexian as an add-on to antiplatelet therapy resulted in a 26% reduction in recurrent ischemic stroke without a statistically significant increase in major bleeding. If approved, asundexian would be the first Factor XIa inhibitor available in the United States and could represent a new adjunctive option to antiplatelet therapy for secondary stroke prevention.

November 2026: cemdisiran

Regeneron's cemdisiran is under FDA Priority Review for the treatment of adults with anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG). The Orphan Drug is a small interfering RNA (siRNA) therapeutic that lowers circulating levels of complement component 5 (C5), a key driver of neuromuscular junction damage and muscle weakness associated with gMG. In the Phase 3, double-blind NIMBLE trial in adults with AChR antibody-positive gMG and a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score ≥6, cemdisiran 600 mg administered subcutaneously (SC) every 12 weeks achieved a placebo-adjusted least-squares mean (LSM) difference of -2.3 points from baseline in the MG-ADL score (P=0.0005) at Week 24.² If approved, cemdisiran could offer an alternative to existing agents for patients with AChR gMG, including complement inhibitors (eculizumab [Soliris], ravulizumab-cwvz [Ultomiris], zilucoplan [Zilbrysq]), neonatal Fc receptor blockers (nipocalimab-aahu [Imaavy], rozanolixizumab-noli [Rystiggo]) and the CD19-directed cytolytic antibody inebilizumab-cdon [Uplizna].

Nov. 14, 2026: ivonescimab

Akeso and Summit Therapeutics are awaiting an FDA decision for ivonescimab for use in combination with chemotherapy for the treatment of locally advanced or metastatic nonsquamous non-small cell lung cancer (nsq-NSCLC) with epidermal growth factor receptor (EGFR) mutation after tyrosine kinase inhibitor (TKI) therapy. Ivonescimab is a first-in-class bispecific antibody that targets both programmed death-1 (PD-1) and vascular endothelial growth factor (VEGF) with a single molecule. In the Phase 3 international HARMONi trial, ivonescimab - in combination with platinum-doublet chemotherapy - demonstrated a statistically significant improvement in progression-free survival (PFS) compared with chemotherapy alone (median PFS, 6.8 months versus 4.4 months, respectively; hazard ratio [HR], 0.52; P<0.00001) but not overall survival (OS; median OS, 16.8 months versus 14 months, respectively; HR, 0.79; P=0.057). However, the separate China-based HARMONi-A trial, which included a longer OS follow-up period compared with the HARMONi trial, did report a significant improvement in the median OS.³ Notably, subsequent analyses using a June 2026 data cutoff for Western patients showed improved OS trending, with an HR of 0.76 (nominal P=0.0151) in the HARMONi overall global population.⁴ Ivonescimab is administered via intravenous (IV) infusion on Day 1 of each 3-week cycle, and it received Fast Track designation from the FDA. If approved, ivonescimab would compete with existing options for subsequent therapy for patients with locally advanced or metastatic nsq-NSCLC with EGFR mutations that have progressed after TKI therapy.

For more information, see the ivonescimab deep dive in the July 2026 edition of Prime's Quarterly Drug Pipeline.

Nov. 17, 2026: adrabetadex

The FDA Priority Review for Mando Health's adrabetadex for the treatment of infantile-onset Niemann-Pick disease type C1 (NPC1) has been extended by three months. NPC1 is an ultra-rare genetic disorder characterized by impaired intracellular cholesterol trafficking that leads to progressive neurologic impairment and premature death. Adrabetadex is a mixture of 2-hydroxypropyl-β-cyclodextrin isomers designed to increase intracellular cholesterol trafficking in NPC1. It is administered intrathecally every two weeks. Adrabetadex was granted Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease (RPD) FDA designations. Although initial analysis of the open-label Phase 2b/3 trial in patients 4-21 years of age with NPC1 did not demonstrate a statistically significant improvement in clinical disease activity with adrabetadex, additional data from longitudinal, pooled and supportive analyses showed a significant reduction in the annual rate of neurologic disease progression, including improvement in key NPC1 biomarkers consistent with increased cholesterol trafficking and reduced neuronal injury.^5,6 In an externally controlled analysis, adrabetadex improved survival among children with infantile-onset NPC1 compared with matched external controls.⁷ If approved, adrabetadex may offer the first FDA-approved treatment option that directly targets impaired intracellular cholesterol trafficking in NPC1.

For more information, see the adrabetadex deep dive in the April 2026 edition of Prime's Quarterly Drug Pipeline.

Nov. 22, 2026: deramiocel

The FDA extended its review for deramiocel, Nippon Shinyaku's and Capricor Therapeutics' off-the-shelf cellular therapy for the treatment of Duchenne muscular dystrophy (DMD), in answer to the companies' request for review of a refined proposed indication focused on upper limb function. DMD is a rare, X-linked neuromuscular disorder associated with progressive muscle degeneration, pulmonary dysfunction and cardiomyopathy. Deramiocel consists of allogeneic, cardiosphere-derived cells and was granted Orphan Drug, RPD and Regenerative Medicine Advanced Therapy (RMAT) FDA designations. In July 2025, the FDA issued a Complete Response Letter (CRL) for deramiocel, requiring additional clinical data; however, review of the Biologic License Application (BLA) was resumed after submission of additional data from the Phase 3 HOPE-3 trial.⁸ Top-line data revealed that deramiocel 1.5×10⁸ cells administered by IV infusion every three months resulted in a 54% slowing in decline in total Performance of Upper Limb (PUL2.0) over 12 months. If approved, deramiocel will be the first cellular therapy to treat DMD.

For more information, see the deramiocel deep dive in the April 2025 edition of Prime's Quarterly Drug Pipeline.

Nov. 22, 2026: molgramostim

The FDA has extended its Priority Review period by three months for Savara's BLA for molgramostim. Molgramostim is being evaluated for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP), a rare, chronic lung disease characterized by the abnormal accumulation of surfactant within the alveoli, impairing gas exchange. Molgramostim is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) designed to activate alveolar macrophages and restore their ability to clear excess surfactant from the lungs. The therapy has received FDA Breakthrough Therapy, Fast Track and Orphan Drug designations for aPAP. In the Phase 3, double-blind IMPALA-2 trial, once-daily nebulized molgramostim significantly improved pulmonary gas transfer, as demonstrated by the primary endpoint of change from baseline to Week 24 in hemoglobin-adjusted diffusing capacity for carbon monoxide (DL_CO%), with an LSM difference versus placebo of 6 percentage points (P<0.001).⁹ If approved, molgramostim would become the first FDA-approved medication specifically indicated for the treatment of aPAP.

For more information, see the molgramostim deep dive in the April 2026 edition of Prime's Quarterly Drug Pipeline.

Nov. 25, 2026: venglustat

Venglustat is an oral glucosylceramide synthase inhibitor (GCSi) by Sanofi. It is undergoing FDA Priority Review for the treatment of Gaucher disease type 3 (GD3), a rare lysosomal storage disorder. GD3 is characterized by accumulation of sugar-and-fat molecules (glycosphingolipids) in the spleen, liver, bone marrow, lung and central nervous system, where it can manifest as neurological complications, including cognitive deficits and difficulty with coordination and balance (ataxia). Venglustat received Breakthrough Therapy, Fast Track and Orphan Drug FDA designations. Venglustat, which crosses the blood-brain barrier, was evaluated in the active-controlled Phase 3 LEAP2MONO study in adults and pediatric patients with neurological manifestations of GD3 stabilized on enzyme replacement therapy (ERT). Patients randomized to venglustat switched from their background ERT to venglustat. At Week 52, once-daily venglustat showed statistically significant superiority to every-two-week IV imiglucerase for neurologic outcomes, based on the global O'Brien test of modified Scale for Assessment and Rating of Ataxia (mSARA) and Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) scores (open-label study statistic, 2.59; one-sided P=0.007).¹⁰ Cognitive scores improved with venglustat and declined with imiglucerase (LSM difference, 8.32; nominal one-sided P=0.016), and systemic disease control was maintained. If approved, venglustat would be the first medicine to address the progressing neurological manifestations in patients with GD3.

Nov. 27, 2026: neladalkib

Nuvalent's neladalkib is under FDA Priority Review for the treatment of advanced anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) in adults who had prior treatment with a TKI. Neladalkib is a next-generation, selective, brain-penetrant ALK inhibitor designed to overcome ALK resistance observed in existing NSCLC treatments, including agents with single or compound ALK mutations. Neladalkib was granted Breakthrough Therapy and Orphan Drug FDA designations. The NDA is supported by the single-arm, Phase 1/2 ALKOVE-1 trial. Based on interim data, once-daily oral neladalkib demonstrated a 31% objective response rate (ORR) in previously treated TKI-exposed patients (n=253), with durable responses for at least 12 and 18 months in 64% and 53% of responders, respectively.¹ Among patients with intracranial involvement (n=92), the ORR was 32%, with 71% of responses estimated to persist for at least 12 months. If approved, neladalkib may provide a targeted treatment option for patients with advanced ALK-positive NSCLC whose disease has progressed after prior ALK-inhibitor therapy - including the third-generation ALK-inhibitor lorlatinib (Lorbrena) - addressing an unmet need in the post-TKI setting.

Nov. 27, 2026: ribitol

BridgeBio's NDA for ribitol is under FDA Priority Review for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), a rare genetic disorder characterized by progressive atrophy and weakness of the muscles of the hip and shoulder (limb-girdle area). Individuals affected by LGMD2I/R9 may eventually lose the ability to walk or stand without assistance. Ribitol is an oral glycosylation substrate therapy designed to stabilize or improve muscle function in patients with LGMD2I/R9. The FDA has granted ribitol Fast Track, Orphan Drug and RPD designations. The Phase 3 FORTIFY trial is evaluating ribitol in patients 12-60 years of age with genetically confirmed LGMD2I/R9.¹² Interim analysis reported that ribitol resulted in a significant 1.8-fold increase compared with placebo in the primary endpoint of glycosylated αDG from baseline to Month 3 (P<0.0001), and the response was sustained through Month 12. Secondary endpoints revealed ribitol led to a statistically significant improvement compared with placebo in the 100-meter timed test (100MTT) velocity (difference, 0.27 meters/second; P<0.0001) and forced vital capacity (FVC; difference, 5%; predicted volume, P=0.0071) at Month 12. In the FORTIFY trial, ribitol was supplied as granules for oral solution that were administered twice daily after reconstitution. There is no cure for LGMD2I/R9. Current management includes supportive approaches with goals to maintain mobility and functional independence. If approved, ribitol will be the first disease-modifying therapy for patients with the condition.

For more information, see the ribitol deep dive in the July 2026 edition of Prime's Quarterly Drug Pipeline.

Nov. 27, 2026: saroglitazar

Zydus Therapeutics' oral peroxisome proliferator-activated receptor (PPAR) α/γ agonist, saroglitazar, is under FDA Priority Review for primary biliary cholangitis (PBC), for use in combination with ursodeoxycholic acid (UDCA) in adults who have had an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA. PBC is a rare, progressive autoimmune disease that destroys the bile ducts, resulting in an accumulation of bile in the liver that can ultimately lead to fibrosis, cirrhosis, the need for liver transplantation or death. Saroglitazar targets both bile-acid toxicity and liver inflammation and has received Fast Track and Orphan Drug FDA designations. Top-line data from the Phase 3, double-blind EPICS-III trial reported that once-daily oral saroglitazar met its primary endpoint, resulting in a statistically significant improvement in biochemical response compared with placebo (56.7% versus 9.8%; P<0.001) at Week 52 in adults with PBC who had an inadequate response to or intolerance of UDCA.¹³ In the study, composite biochemical response was defined as alkaline phosphatase (ALP) levels <1.67 times the upper limit of normal (ULN), a ≥15% decrease in ALP from baseline, and total bilirubin ≤ULN or direct bilirubin ≤ULN in patients with known Gilbert's syndrome. If approved, saroglitazar may compete as second-line therapy with the oral PPAR agonists elafibranor (Iqirvo) and seladelpar lysine (Livdelzi).

Nov. 30, 2026: bezuclastinib

Bezuclastinib, developed by Cogent Biosciences, is under FDA Priority Review and part of the Real-Time Oncology Review (RTOR) program for use in combination with sunitinib (Sutent) for the treatment of patients with gastrointestinal stromal tumors (GIST) previously treated with imatinib (Gleevec). Bezuclastinib is a selective TKI designed to target the KIT mutations that drive GIST tumor growth. Sunitinib is the preferred second-line therapy for most patients with advanced KIT-mutant GIST. The Phase 3 PEAK trial demonstrated a 16.5-month median PFS and 45.6% ORR with bezuclastinib plus sunitinib, compared with a 9.2-month median PFS and 25.8% ORR with sunitinib alone, in patients with GIST with KIT exon 9 mutation previously treated with imatinib.¹⁴ Both agents were administered once daily. Bezuclastinib has received Breakthrough Therapy and Orphan Drug FDA designations for GIST. If approved, the combination of sunitinib with bezuclastinib could establish a new second-line standard of care in this patient population. Separately, Cogent has submitted NDAs for bezuclastinib for the treatment of nonadvanced systemic and advanced systemic mastocytosis, with FDA decisions anticipated by Dec. 30, 2026, and June 29, 2027, respectively.

Nov. 30, 2026: giredestrant

Genentech's oral selective estrogen receptor degrader (SERD), giredestrant, is under FDA Priority Review for adjuvant treatment of adults with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative stage I, II and III breast cancer. Giredestrant received Fast Track FDA designation. The NDA is supported by the interim analysis of the open-label, Phase III lidERA Breast Cancer trial conducted in patients with ER-positive, HER2-negative early breast cancer.¹⁵ The study showed giredestrant reduced the risk of invasive disease recurrence or death at three years by 30% compared with standard-of-care endocrine therapy (SOC ET; HR, 0.7; P=0.0014). At three years, 92.4% of giredestrant-treated patients were alive and free of invasive disease compared with 89.6% who received SOC ET. If approved, giredestrant would become the first oral SERD indicated in the adjuvant setting for ER-positive/HER2-negative early breast cancer. Genentech is also awaiting an FDA decision by Dec. 18, 2026, for giredestrant, for use in combination with everolimus for treatment of ER-positive/HER2-negative, ESR1-mutated, locally advanced or metastatic breast cancer.

Nov. 30, 2026: povetacicept

Vertex Pharmaceuticals is seeking FDA Accelerated Approval for povetacicept for the treatment of immunoglobulin A nephropathy (IgAN), also known as Berger's disease. Povetacicept is an Fc fusion protein and dual inhibitor of the BAFF and A proliferation inducing ligand (APRIL) cytokines that are implicated in IgAN. The FDA has granted povetacicept Breakthrough Therapy designation and a Priority Review. The BLA is supported by top-line data from the double-blind, Phase 3 RAINIER trial, which demonstrated monthly SC injections of povetacicept resulted in a placebo-adjusted 49.8% reduction in the 24-hour urine protein-to-creatinine ratio (UPCR) from baseline to 36 weeks (P<0.0001).¹⁶ Povetacicept can be self-administered. If approved, it will be the second dual BAFF/APRIL inhibitor for IgAN, following the approval of atacicept (Trutakna) in July 2026.

For more information, see the atacicept and povetacicept deep dive in the April 2026 edition of Prime's Quarterly Drug Pipeline.

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