Blueprint Medicines’ Supplemental NDA for Ayvakit approved by FDA

Blueprint Medicines’ supplemental new drug application for Ayvakit for the treatment of adults with indolent systemic mastocytosis has been accepted by the FDA. The FDA granted priority review with an action date of May 22. 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.