FDA Accepts Cogent Biosciences’ NDA For Bezuclastinib In Non-Advanced Systemic Mastocytosis With PDUFA Date Set For December 2026

Cogent Biosciences, Inc., a biotechnology company focused on developing precision therapies for genetically defined diseases, announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for bezuclastinib in patients with Non-Advanced Systemic Mastocytosis (NonAdvSM). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of December 30, 2026. At this time, the FDA indicated that there are no plans to hold an advisory committee meeting and has not identified any potential review issues. The NDA submission is supported by data from the pivotal SUMMIT trial, which evaluated bezuclastinib in patients with NonAdvSM. The trial demonstrated clinically meaningful and highly statistically significant improvements across the primary and all key secondary endpoints. Data through 48 weeks showed continued and deepening symptomatic improvement over time, suggesting the potential for sustained clinical benefit with longer therapy. The trial also highlighted benefits in patient populations with high unmet medical need, positive effects on bone mineral density, and evidence of disease modification. Bezuclastinib was generally well tolerated, with a favorable safety profile supporting its potential use as a chronic therapy for NonAdvSM patients. Cogent Biosciences also confirmed that the NDA submission for bezuclastinib in Gastrointestinal Stromal Tumors (GIST) for patients previously treated with imatinib remains on track for April 2026. This submission is being conducted under the FDA’s Real-Time Oncology Review (RTOR) program, and bezuclastinib has received Breakthrough Therapy Designation for this indication. Additionally, the NDA submission for bezuclastinib in Advanced Systemic Mastocytosis (AdvSM) is on schedule for the first half of 2026. With these regulatory milestones, Cogent Biosciences is advancing bezuclastinib across multiple indications, aiming to provide meaningful clinical benefits for patients with systemic mastocytosis and GIST while continuing to demonstrate the therapy’s potential for long-term safety and disease modification.