Blueprint Medicines Corporation announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental new drug application for AYVAKIT (avapritinib) for the treatment of adults with indolent systemic mastocytosis (SM). The FDA granted priority review with an action date of May 22, 2023 under the Prescription Drug User Fee Act (PDUFA).
This regulatory application is based on results from the global PIONEER trial, the largest randomized, placebo-controlled study ever conducted in indolent SM. The FDA previously granted breakthrough therapy designation to AYVAKIT for the treatment of moderate to severe indolent SM. AYVAKIT was designed to potently and selectively inhibit D816V mutant KIT, the primary underlying cause of SM.
“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 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. 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.”