Blueprint Medicines Wins First FDA Approval

Blueprint Medicines, a Cambridge biotech focused on genomically defined cancers, rare diseases and cancer immunotherapy, has announced FDA approval for Ayvakit (avapritinib), its kinase inhibitor for the treatment of a genomically defined population of patients with gastrointestinal stromal tumor (GIST), a rare, sarcoma of the GI tract.

“The full approval of Ayvakit…is an incredibly exciting milestone for our company and, more importantly, for GIST patients with a PDGFRA exon 18 mutation who have been waiting for a new treatment option,” said Jeff Albers, Chief Executive Officer at Blueprint Medicines. “Ayvakit is the first of what we hope will be many approved medicines enabled by our research platform…”

Blueprint Medicines is pursuing a broad clinical development program for avapritinib across multiple lines of GIST treatment, as well as for advanced, smoldering and indolent systemic mastocytosis (SM). The FDA has granted Breakthrough Therapy Designation to avapritinib for two indications: one for the treatment of unresectable or metastatic GIST harboring the PDGFRA D842V mutation and one for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia. (Source: Blueprint Medicines Website, 09 January 9, 2020)