FDA accepts Cogent’s bezuclastinib NDA in nonadvanced SM
CURRENT BRIEF VERSION: Cogent Biosciences said the US FDA has accepted its new drug application for bezuclastinib in adults with nonadvanced systemic mastocytosis, or NonAdvSM, and assigned a PDUFA target action date of December 30, 2026. The company said the agency isn't currently planning an advisory committee meeting and hasn't identified review issues at this stage. The filing is backed by results from the registration-directed SUMMIT trial, where bezuclastinib met the primary endpoint and all key secondary endpoints, with reported improvements in symptoms and objective markers of mast cell burden. (stocktitan.net)
Why it matters: While this is a human drug regulatory milestone, it matters to veterinary professionals because it reflects continued FDA momentum around highly selective KIT-targeted therapies, a pathway with relevance across mast cell disease biology. For clinicians and industry watchers, the notable points are the FDA’s acceptance of the dossier, the absence of early review flags, and the company’s push toward a possible US launch in the second half of 2026 if approved. Cogent has also framed bezuclastinib as a chronic-use therapy, supported by 48-week data and an expanded access program already underway in the US. More broadly, the company is trying to build a wider bezuclastinib franchise: it has also submitted an NDA under the FDA’s RTOR program for previously treated GIST based on the phase 3 PEAK trial of bezuclastinib plus sunitinib versus sunitinib alone, reported a 50% improvement in median progression-free survival (16.5 vs 9.2 months) and a higher objective response rate (46% vs 26%), and said FDA-aligned expanded access programs are available for eligible US patients with GIST or systemic mastocytosis receiving bezuclastinib-based therapies. (hcplive.com) (pharmashots.com)
What to watch: The next milestone is the FDA’s review ahead of the December 30, 2026, action date, alongside Cogent’s planned 2026 filings for GIST and advanced systemic mastocytosis and its planned phase 2 move in first-line GIST with exon 9 mutations. (stocktitan.net) (pharmashots.com)