Additional information is available at or. To learn about ongoing or planned clinical trials, contact Blueprint Medicines at 1-888-BLU-PRNT (1-88). AYVAKIT/AYVAKYT is not approved for the treatment of any other indication in the U.S. or Europe. Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT. This medicine is approved in Europe (AYVAKYT ®) 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. We look forward to collaborating with the FDA during its review process, with the goal of bringing the first approved medicine to patients with indolent SM and redefining the treatment landscape beyond symptom-directed therapies."ĪYVAKIT (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. "AYVAKIT achieved the primary and all key secondary endpoints in the PIONEER trial, with highly meaningful reductions in patient-reported symptoms and all measures of mast cell burden studied, and a well-tolerated safety profile supporting chronic treatment. "People with indolent systemic mastocytosis experience debilitating symptoms and poor quality of life, and we have the potential to transform clinical outcomes for these patients by targeting the genetic driver of disease with AYVAKIT," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. AYVAKIT was designed to potently and selectively inhibit D816V mutant KIT, the primary underlying cause of SM. The FDA previously granted breakthrough therapy designation to AYVAKIT for the treatment of moderate to severe indolent SM. This regulatory application is based on results from the global PIONEER trial, the largest randomized, placebo-controlled study ever conducted in indolent SM.
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