Blueprint Medicines Corporation (NASDAQ: BPMC), a US-based global precision therapy company, announced on Monday that the US Food and Drug Administration (FDA) has accepted the firm's supplemental new drug application for AYVAKIT (avapritinib) intended to treat adults with indolent systemic mastocytosis (SM), a rare disease driven by the KIT D816V mutation in about 95% of cases.

The company has received priority review with an action date of 22 May 2023 under the Prescription Drug User Fee Act (PDUFA) from the FDA.

AYVAKITis a kinase inhibitor approved by the FDA to treat adults with Advanced SM that includes aggressive SM (ASM), SM with an associated haematological neoplasm (SM-AHN) and mast cell leukaemia (MCL), and adults with unresectable or metastatic gastrointestinal stromal tumour (GIST) harbouring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

This regulatory application is based on results from the global PIONEER trial, the largest randomised, 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.