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News
The FDA has accepted a new drug application (NDA) for bezuclastinib to treat advanced systemic mastocytosis, with a target action date of June 29, 2027. The decision is backed by phase 2 APEX trial data showing a 65% overall response rate per mIWG-MRT-ECNM criteria and 81% per PPR criteria. This marks another regulatory milestone for Cogent Biosciences as it pursues approvals across multiple KIT-driven diseases.
The FDA has accepted Cogent Biosciences' new drug application (NDA) for bezuclastinib, a targeted therapy for patients with advanced systemic mastocytosis (AdvSM) — a rare and aggressive mast cell disorder. The agency set a Prescription Drug User Fee Act (PDUFA) target action date of June 29, 2027.
The NDA is supported by data from the phase 2 APEX trial, which enrolled patients with aggressive systemic mastocytosis, systemic mastocytosis with associated hematologic neoplasms, or mast cell leukemia. Bezuclastinib was administered at 150 mg once daily, and the trial demonstrated meaningful response rates with a manageable safety profile — no treatment-related deaths were observed, and most patients remained on their starting dose throughout the study.
By the Numbers:
Why it matters: Advanced systemic mastocytosis is a rare, life-threatening disease with limited treatment options. Bezuclastinib's NDA acceptance — alongside pending applications for non-advanced systemic mastocytosis and GIST — positions it as a potentially broad-reaching therapy for KIT-driven diseases, offering new hope to patients with few alternatives.
