The FDA approved Blueprint Medicines’s Ayvakit (avapritinib) to treat adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation.
The approval was based on the results of a phase 1 clinical trial and combined safety results from other clinical trials. Patients treated with Ayvakit had an overall response rate of 84 percent.
The approval makes Ayvakit the first targeted therapy for the condition. It was previously granted breakthrough therapy, fast track and orphan drug designations.