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Medicines Announces FDA Acceptance of Supplemental New Drug Application for AYVAKIT® (avapritinib)

Tuesday, January 24, 2023

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 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.

AYVAKIT (avapritinib) is a kinase inhibitor applicable for treating patients with Advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), adults consisting of unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

Systemic mastocytosis (SM), rare disease caused by the KIT D816V mutation in about 95 percent of cases. Individuals with indolent systemic mastocytosis consists of debilitating symptoms and result in poor quality of life. Hence, indolent systemic mastocytosis disease can be targeted with AYVAKIT.

AYVAKIT® (avapritinib) is approved in Europe (AYVAKYT®) to treat adults with ASM, SM-AHN or MCL, applicable after at least one systemic therapy, and for adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.

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