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.

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