for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM-AHN and mast cell leukemia (MCL). Under the brand name AYVAKIT, the medicine is approved in the U.S. The EC decision follows the positive opinion by the Committee for Medicinal Products for Human Use (CHMP) and is based 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 all disease subtypes after at least one systemic therapy and a generally well-tolerated safety profile.ĪYVAKYT® (avapritinib) is a kinase inhibitor approved by the European Commission for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring the PDGFRA D842V mutation and 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. AYVAKYT will be available in 25 mg, 50 mg, 100mg and 200mg dose strengths, and the recommended starting dose in advanced SM is 200 mg once daily. In Europe, Blueprint Medicines plans to initiate its first commercial launch in Germany following the EC approval, and the timing of AYVAKYT commercial availability will vary for other countries based on local reimbursement and access pathways. "We believe that AYVAKYT has the potential to shift the treatment paradigm to a precision therapy approach in advanced forms this disease, and we look forward to working closely with national reimbursement bodies across Europe to bring AYVAKYT to patients." "Today we are incredibly proud to bring an innovative new treatment option to individuals who have been impacted by advanced systemic mastocytosis," said Georg Pirmin Meyer, M.D., Senior Vice President, International at Blueprint Medicines.
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