FDA Approves Ayvakit as the First Treatment for Indolent Systemic Mastocytosis (ISM)

The US Food and Drug Administration (FDA) has approved Blueprint Medicines Corporation’s Ayvakit (avapritinib), the first and only treatment for adults with indolent systemic mastocytosis (ISM). 

• Systemic mastocytosis (SM) is a rare hematologic disorder. The Vast majority of SM cases are ISM, and Ayvakit has been designed to inhibit KIT D816V, the primary underlying cause of the disease. 
• 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.
• The NEJM Evidence published detailed results from the PIONEER trial of Ayvakit, showing that patients treated with the drug achieved rapid, durable, and statistically significant reductions on all pathological mast cell burden measures.
• 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