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  • FDA approves Ayvakit to treat advanced systemic ma...

FDA approves Ayvakit to treat advanced systemic mastocytosis.-Blueprint Medicines Corpn.

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Published:17th Jun 2021
Blueprint Medicines Corporation announced that the FDA has approved Ayvakit (avapritinib) for the treatment of adult patients with advanced systemic mastocytosis (Advanced SM), including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). For the first time, advanced SM patients can now receive a targeted therapy designed to potently and selectively inhibit D816V mutant KIT, the central driver of the disease.

Systemic Mastocytosis is a rare hematologic disorder caused by the KIT D816V mutation in nearly all cases. Across advanced SM subtypes, the median overall survival is approximately 3.5 years in ASM, approximately two years in SM-AHN and less than six months in MC.

The FDA granted full approval to Ayvakit for adults with advanced SM based on data from the Phase 1 EXPLORER trial and Phase II PATHFINDER trial. Treatment response was evaluated using modified IWG-MRT-ECNM criteria, with assessments based on at least 12 weeks of response duration, resolution of at least one finding of non-hematologic and hematologic organ damage, and 50 percent or greater reductions in biomarker response, mast cell burden and serum tryptase. The overall response rate (ORR) in the U.S. prescribing information is defined as complete remission with full or partial hematologic recovery (CR/CRh), or partial remission (PR).

Condition: Systemic Mastocytosis
Type: drug

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