Benzinga | Jan 28, 2022 08:06AM EST

Blueprint Medicines' AYVAKYT Receives Positive CHMP Opinion For Treatment Of Adults With Advanced Systemic Mastocytosis

Blueprint Medicines Corporation (NASDAQ:BPMC) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion, recommending to expand the current indication for AYVAKYT(r) (avapritinib) to include monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL), after at least one systemic therapy.

"The positive opinion from the CHMP is a result of nearly a decade of collaboration with the systemic mastocytosis community and a dedication to bring a transformative therapy to treat and manage this rare, life-threatening disease," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "Today's announcement brings patients with advanced forms of SM in Europe one step closer to accessing AYVAKYT, the first specifically designed precision therapy to selectively target the primary driver of their disease. Blueprint Medicines remains committed to improving outcomes for patients living with systemic mastocytosis around the world."

"Patients across Europe are waiting for innovative treatment options for advanced systemic mastocytosis, which is associated with organ damage due to mast cell proliferation and poor survival outcomes," said Prof. Dr. Andreas Reiter, M.D., Center of Excellence for Myeloproliferative Neoplasms, University Medicine Mannheim. "A precision therapy associated with potent and selective targeting of the KIT D816V mutation brings the prospect of changing the course of disease and transforming treatment for patients to set a new standard of care in Europe."

The CHMP opinion will now be reviewed by the European Commission, which has the authority to grant and expand marketing authorizations for medicinal products in the European Union. A final decision on the application to expand the indication for AYVAKYT is anticipated by early April 2022.

The CHMP based its opinion on results from the Phase 1 EXPLORER trial and Phase 2 PATHFINDER trial, in which AYVAKYT showed durable clinical efficacy in advanced SM patients across disease subtypes and a generally well-tolerated safety profile. These two studies, which enrolled approximately 150 patients with up to four years of follow-up and comprise the largest ever reported dataset in advanced systemic mastocytosis, were recently published in Nature Medicine.