Blueprint reports 84% ORR for avapritinib in PDGFRα D842V-driven GIST

Blueprint Medicines Corp. (NASDAQ:BPMC) reported updated data from the Phase I NAVIGATOR trial showing that oral avapritinib led to an objective response rate (ORR) of 84% in 56 evaluable patients with PDGFRα D842V-driven advanced gastrointestinal tumors (GIST). Median progression-free survival (PFS) was not reached. In 109 evaluable heavily treated patients with fourth-line or later GIST, avapritinib led to an ORR of 20%. Median PFS as assessed by central review was 3.7 months. Data from the open-label, international trial were presented at the Connective Tissue Oncology Society meeting in Rome.

The company reiterated its plan to submit an NDA to FDA in 1H19 for avapritinib to treat PDGFRα exon 18 mutant GIST, which primarily includes patients with the PDGFRα D842V mutation, and GIST in the fourth-line setting. Avapritinib has breakthrough therapy designation from FDA to treat PDGFRα D842V-driven GIST...