Cogent Biosciences Bezuclastinib NDA Accepted by FDA for Advanced Systemic Mastocytosis; June 29, 2027 PDUFA Action Date and No Planned Advisory Committee
Cogent Biosciences (NASDAQ: COGT) announced Tuesday September 15, 2026 that the FDA accepted the New Drug Application (NDA) for bezuclastinib (a selective oral tyrosine kinase inhibitor targeting KIT D816V) in patients with Advanced Systemic Mastocytosis (AdvSM), an aggressive form of the rare hematologic disorder driven by activating mutations in the KIT receptor. FDA assigned a PDUFA target action date of June 29, 2027. The agency communicated that no advisory committee is planned and no potential review issues have been identified. Application support: the APEX Phase 2 registration trial primary endpoint reported a 65% overall response rate per modified International Working Group Myeloproliferative Neoplasms Research and Treatment / European Competence Network on Mastocytosis (mIWG-MRT-ECNM) criteria as of the March 31, 2026 data cutoff, including 57% of patients achieving complete response (CR), CR with partial hematologic recovery (CRh), or partial response (PR) as best response. Cogent had earlier received FDA acceptance for the bezuclastinib NonAdvSM (non-advanced systemic mastocytosis) NDA in March 2026 supported by the SUMMIT Phase 3 trial. Bezuclastinib would compete against Blueprint Medicines' Ayvakit (avapritinib) in the AdvSM segment; both target KIT D816V but Cogent has emphasized bezuclastinib's cleaner off-target profile and better CNS penetration profile.