Cogent Biosciences announced on October 20, 2025 that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to its lead candidate, bezuclastinib, for the treatment of Non-Advanced Systemic Mastocytosis (NonAdvSM) patients who have previously been treated with avapritinib, as well as for patients with Smoldering Systemic Mastocytosis, a population with no approved standard of care.
The designation is based on positive results from the registration‑directed SUMMIT trial, in which bezuclastinib achieved statistically significant improvements across all primary and key secondary endpoints in NonAdvSM patients. The FDA’s recognition is intended to expedite the review process, potentially allowing for a faster priority review and earlier market access for the drug.
This regulatory milestone is a material development for Cogent, as the Breakthrough Therapy Designation can accelerate the drug’s approval pathway and enhance the company’s competitive positioning in the rare disease oncology space.
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