EMA's Advisory Committee Recommends Approval Of Blueprint Medicines' Avapritinib In Blood Disorder

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended expanding the use of Blueprint Medicines Corporation's BPMC Ayvakyt (avapritinib).

• The positive opinion covers Ayvakyt as monotherapy for aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL) after at least one systemic therapy.
• A final decision on the application to expand the indication for Ayvakyt is anticipated by early April 2022.
• Related: Blueprint Medicines Expands Precision Therapy With Lengo Therapeutics Acquisition.
• The CHMP based its opinion on results from the Phase 1 EXPLORER trial and Phase 2 PATHFINDER trial. 
• Ayvakyt showed durable clinical efficacy in advanced SM patients across disease subtypes and a generally well-tolerated safety profile. 
• These two studies enrolled approximately 150 patients with up to four years of follow-up.
• Price Action: BPMC shares are down 1.36% at $69.16 during the market session on the last check Friday.