Blueprint Lays Out Path To Early Approval In Small GIST Population

Blueprint Medicines will pursue an accelerated path to approval for its newly designated breakthrough therapy BLU-285 in the treatment of gastrointestinal stromal tumors in a small subset of patients.

Phase I data for Blueprint Medicines Corp.'s kinase inhibitor BLU-285 prompted the US FDA to grant a breakthrough therapy designation for the treatment of a small, genetically defined group of patients with gastrointestinal stromal tumors (GIST), which could result in approval based on positive results from the expansion cohort of the early-stage clinical trial.

BLU-285 selectively inhibits the PDGFRα and KIT kinases, but the breakthrough therapy designation only covers the drug's use as a treatment for GIST patients with a PDGFRα D842V mutation

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