FDA OKs Blueprint Medicines' Blood Disorder Drug As First Treatment For SM Patients

The U.S. Food and Drug Administration (FDA) approved Blueprint Medicines Corporation's BPMC Ayvakit (avapritinib) for the treatment of adults with indolent systemic mastocytosis (ISM). 

• Systemic mastocytosis (SM) is a rare hematologic disorder. ISM represents the vast majority of SM cases, and Ayvakit is now available for adults with ISM at the recommended dose of 25 mg once daily. 
• Ayvakit was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease. 
• Ayvakit has been FDA-approved for advanced SM since June 2021.
• The approval of Ayvakit in ISM is based on data from the PIONEER trial. 
• Ayvakit demonstrated significant improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden.
• Concurrently, NEJM Evidence, a journal from The New England Journal of Medicine Group, published detailed results from the PIONEER trial of Ayvakit.
• Patients treated with Ayvakit achieved rapid, durable, and statistically significant reductions on all pathological mast cell burden measures, including serum tryptase, KIT D816V variant allele fraction, and bone marrow mast cells.
• Ayvakit showed clinically meaningful improvements in multiple exploratory endpoints of quality of life.
• In the first quarter of 2023, Blueprint Medicines reported Ayvakit sales of $39.1 million, up from $23.8 million a year ago.

Price Action: BPMC shares are up 3.99% at $58.50 on the last check Tuesday, according to Benzinga Pro.

Next: FDA Removes Partial Clinical Hold From Blueprint Medicines' Cancer Study