Blueprint Medicines Corporation’s newly approved Ayvakit (avapritinib) appears well-positioned to challenge its only competitor in advanced systemic mastocytosis (SM), Novartis AG’s Rydapt (midostaurin). Blueprint is planning an initial rollout to specialized cancer centers and an education push to facilitate moving the drug into community oncology settings, similar to its strategy for the drug in gastrointestinal stromal tumor (GIST).
Cambridge, MA-based Blueprint announced 16 June that the FDA had approved Ayvakit for advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). The company is also developing the drug for indolent SM, which encompasses about 10 times as many patients as advanced SM. The drug is targeted to KIT D816V, the central driver mutation of the disease. It previously received approval in January 2020 for GIST, a type of sarcoma, harboring PDGFRA exon 18 mutations
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