Research & Development
Blueprint Medicines gets FDA approval for AYVAKIT for treating unresectable or metastatic GIST in adults
10 January 2020 -

Precision therapy company Blueprint Medicines Corporation (NASDAQ:BPMC) revealed on Thursday the receipt of approval from the US Food and Drug Administration (FDA) for AYVAKIT (avapritinib) for treating unresectable or metastatic gastrointestinal stromal tumor (GIST) in adults.

GIST is a rare, genomically driven sarcoma of the gastrointestinal (GI) tract. Approximately 6% of patients with newly diagnosed GIST have PDGFRA exon 18 mutations.

AYVAKIT is is a kinase inhibitor and the first precision therapy approved to treat a genomically defined adult population of patients with GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations. AYVAKIT is a selective and potent inhibitor of KIT and PDGFRA mutant kinases.

The FDA's full approval of AYVAKIT is based on efficacy results from the company's Phase 1 NAVIGATOR clinical trial, as well as combined safety results from multiple clinical trials for avapritinib. In patients with PDGFRA exon 18 mutant GIST, AYVAKIT had an overall response rate (ORR) of 84% and a median duration of response (DOR) was not reached.

Blueprint Medicines also said it will launch YourBlueprint, a patient support programme that offers access and affordability solutions for individuals receiving AYVAKIT.



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