FDA Rejects Blueprint Medicines NDA for Avapritinib

FDA has issued Blueprint Medicines a complete response letter for its NDA for avapritinib, indicated for treating adults with unresectable or metastatic fourth-line gastrointestinal stromal tumor (GIST). The company did not disclose FDA’s reasons for the letter or whether is requested any additional data or clinical trials. Avapritinib is a kinase inhibitor approved by FDA under the trade name Ayvakit for treating adults with unresectable or metastatic GIST who harbor a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. The company says it is continuing to advance development of avapritinib for treating systemic mastocytosis. Based on top-line results reported in April for Blueprint’s Phase 3 VOYAGER trial, the company previously announced plans to discontinue further development of avapritinib in GIST indications other than PDGFRA exon 18 mutant GIST.