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FDA Decisions Expected: November 2025

Your monthly synopsis of new drugs expected to hit the market 

October 13, 2025

Drug pipeline for November 2025

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 United States (U.S.) Food and Drug Administration (FDA).
Nov. 18, 2025: plozasiran
Plozasiran, by Arrowhead, is a first-in-class RNA interference (RNAi) therapeutic designed to reduce production of apolipoprotein C-III (APOC3), thereby reducing circulating triglycerides (TG). It was submitted to the FDA for the treatment of familial chylomicronemia syndrome (FCS), a rare, inherited condition characterized by severe hypertriglyceridemia. The Phase 3 double-blind PALISADE trial evaluated plozasiran administered subcutaneously (SC) every three months in adults with genetically confirmed or clinically diagnosed FCS.¹ At 10 months, the study reported the median change from baseline in the fasting TG level (the primary end point) was -80% with plozasiran 25 mg, -78% with plozasiran 50 mg and -17% with placebo (p<0.001 for both doses). The FDA granted plozasiran Breakthrough Therapy, Fast Track and Orphan Drug designations. If approved, plozasiran will be the first RNAi agent that targets APOC3 to reduce TG levels in the blood and could compete with the antisense oligonucleotide olezarsen (Tryngolza) for the treatment of patients with FCS.

For more information, see the plozasiran Deep Dive in the October 2025 edition of Prime’s Quarterly Drug Pipeline.
Nov. 28, 2025: navepegritide (TransCon CNP)
Ascendis is awaiting the FDA decision for navepegritide for the treatment of achondroplasia. Navepegritide is a prodrug of C-type natriuretic peptide (CNP). It provides continuous exposure of active CNP to stimulate bone growth. The Phase 2/3, double-blind, placebo-controlled ApproaCH trial evaluated navepegritide in children ages 2–11 years with achondroplasia.² 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/yr, respectively; LSM difference, 1.49 cm/yr; p<0.0001) in the overall study population. In the clinical trial, a weight-based dose of navepegritide was administered SC once weekly. Navepegritide was granted Orphan Drug designation and a Priority Review by the FDA. If approved, navepegritide will be the second CNP available in the U.S. to increase linear growth in children with achondroplasia and will compete with the CNP analog vosoritide (Voxzogo), which is administered once daily by the patients or caregiver until closure of epiphyses occurs.

For more information, see the navepegritide Deep Dive in the October 2025 edition of Prime’s Quarterly Drug Pipeline.
Nov. 28, 2025: sibeprenlimab
Sibeprenlimab is a monoclonal antibody by Otsuka that selectively inhibits the activity of A PRoliferation-Inducing Ligand (APRIL). The FDA granted a Breakthrough Therapy designation and Priority Review for the treatment of Immunoglobulin A (IgA) nephropathy (IgAN), also known as Berger’s disease. The ongoing, randomized, double-blind, placebo-controlled Phase 3 VISIONARY (NCT05248646) trial evaluated sibeprenlimab, administered SC every 4 weeks, for the treatment of adults with IgAN who are receiving maximally tolerated standard of care treatment.³ The primary endpoint was reduction of proteinuria as measured by the change in 24-hour urine protein-to-creatinine ratio (UPCR) at 9 months. Interim analysis revealed that patients treated with sibeprenlimab achieved a 51.2% (p<0.0001) reduction in proteinuria at 9 months when compared to placebo. If approved, sibeprenlimab will be a first-in-class APRIL inhibitor providing a new mechanism of action for treatment of IgAN when used in addition to supportive care. Sibeprenlimab has the potential to be self-administered.

For more information, see the sibeprenlimab Deep Dive in the July 2025 edition of Prime’s Quarterly Drug Pipeline.
Nov. 28, 2025: ziftomenib
The FDA granted a Priority Review for Kura Oncology’s menin inhibitor ziftomenib for the treatment of adults with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation. The FDA also granted Breakthrough Therapy, Fast Track and Orphan Drug designations. Kura is seeking full approval for ziftomenin based on the Phase 1/2, open-label KOMET-001 study.⁴ Full published results reveal that the study achieved its primary endpoint with a complete remission with full or partial hematologic recovery (CR/CRh) at a rate of 22% (p=0.0058) with ziftomenin 600 mg orally once daily monotherapy in patients with R/R NPM1-mutated AML. The CR/CRh response rate is 12% with historical standard-of-care. The median overall survival was 6.6 months with ziftomenin. If approved, ziftomenib will be the first therapy to specifically target NPM1-mutated AML.
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