Blueprint Medicines Corporation announced data showcasing its commitment to advance the scientific understanding and treatment of systemic mastocytosis at the 65th American Society of Hematology Annual Meeting and Exposition being held December 9-12 in San Diego. Data that will be presented include results from the HARBOR Part 1 trial of elenestinib in indolent systemic mastocytosis and analyses of real-world data highlighting the burden of and urgency to treat ISM. In November 2023, the Committee for Medicinal Products for Human Use of the European Medicines Agency issued a positive opinion recommending the approval of AYVAKIT for the treatment of adults with ASM, SM-AHN or MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.
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