News
Ayvakyt receives positive CHMP opinion for the treatment of adults with advanced systemic mastocytosis- Blueprint Medicines.
Blueprint Medicines Corporation has 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 (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 CHMP based its opinion on results from the Phase 1 EXPLORER trial and Phase II 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. ( previously cited).
Condition: Systemic Mastocytosis
Type: drug
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