Research & Development
Blueprint Medicines reports EMA validation of avapritinib MAA for treatment of GIST
19 July 2019 -

Precision therapy company Blueprint Medicines Corporation (NASDAQ:BPMC) on Thursday reported the European Medicines Agency's (EMA) validation of its Marketing Authorization Application (MAA) for avapritinib for the treatment of adult patients with PDGFRα D842V mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST.

GIST is a sarcoma, or tumor of bone or connective tissue, of the gastrointestinal (GI) tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine.

The EMA validation of the MAA confirms that the avapritinib application is sufficiently complete to begin the formal review process.

Avapritinib is an investigational, potent and highly selective KIT and PDGFRA inhibitor for patients with advanced GIST. The European Commission has granted orphan medicinal product designation to avapritinib for the treatment of GIST.

Additionally, the company has submitted a New Drug Application to the US Food and Drug Administration (FDA) for avapritinib for the treatment of adult patients with PDGFRA Exon 18 mutant GIST, regardless of prior therapy and fourth-line GIST.