FDA Accepts NDA for Bezuclastinib in the Treatment of Advanced SM
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The FDA accepted Cogent Biosciences' NDA for bezuclastinib for advanced systemic mastocytosis, scheduling a PDUFA action date of June 29, 2027.
The US Food and Drug Administration (FDA) approved a New Drug Application (NDA) for bezuclastinib, a highly selective tyrosine kinase inhibitor (TKI) designed to inhibit KIT D816V mutations and other KIT exon 17 mutations, in the treatment of advanced systemic mastocytosis (AdvSM), according to a news release from the treatment’s manufacturer, Cogent Biosciences.
The NDA submission was supported by phase 2 results from the APEX trial (NCT04996875), which showed a 65% overall response rate, including complete response (CR), partial response (PR), complete remission with partial hematological recovery (CRh), and clinical improvement (CI). Limiting to just CR, CRh, and PR accounted for 57% of patients, and 81% experienced a response based on the pure pathological response (PPR) criteria as of the March 31, 2026 data cutoff date.
“With the FDA’s acceptance of our NDA in Advanced Systemic Mastocytosis, we have reached another important regulatory milestone for bezuclastinib and are one step closer to potentially bringing this therapy to patients with AdvSM,” said Andrew Robbins, president and chief executive officer of Cogent Biosciences.
Patients also experienced durable response to treatment, with 87% of patients experiencing 12-month overall survival (OS) and 79% achieving 12-month progression-free survival (PFS). By the end of the follow-up period, the median PFS and OS time had not been reached.
The FDA assigned a Prescription Drug User Fee Act (PDUFA) date of June 29, 2027. Cogent Biosciences has previously announced an NDA for bezuclastinib in nonadvanced SM (NonAdvSM) with a PDUFA date of December 30, 2026.
“This milestone further reinforces the potential of bezuclastinib across multiple patient populations with KIT-driven diseases. With an excellent commercial and medical team in place, we are excited and ready for the potential launches later this year,” Robbins continued.
