Blueprint scores first FDA OK for a precision GIST cancer drug, just ahead of a rival treatment. And the price is a big surprise
Hitting the regulatory finish line just ahead of a possible precision rival, Blueprint Medicines has scored its first FDA OK. The green light came through for avapritinib, to be dubbed Ayvakit on the market.
Blueprint’s $BPMC crew, headed by CEO Jeff Albers, got an accelerated OK ahead of the priority review PDUFA date of February 14, in keeping with a slew of cancer drugs that have warranted spotlight treatment at the agency. It’s precisely targeted against a niche: gastrointestinal stromal tumor — GIST — with a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.
That patient group scored an overall response rate of 84%.
But there are some flies in the ointment.
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 77,000+ biopharma pros reading Endpoints daily — and it's free.