Blueprint Medicines Submits Supplemental New Drug Application to FDA for AYVAKIT® (avapritinib) for the Treatment of Indolent Systemic Mastocytosis – QNT Press Release

— Company to present registrational PIONEER trial results at 2023 AAAAI Annual Meeting —

CAMBRIDGE, Mass., Nov. 22, 2022 /PRNewswire/ — Blueprint Medicines Corporation (NASDAQ: BPMC) today announced the submission of a supplemental new drug application to the US Food and Drug Administration (FDA) for AYVAKIT® (avapritinib) for the treatment of adults with indolent systemic mastocytosis (SM ). AYVAKIT was designed to potentially and selectively inhibit D816V mutant KIT, the underlying cause of SM in about 95 percent of cases.

The submission includes data from the registrational PIONEER trial, the largest randomized, placebo-controlled clinical study ever conducted in indolent SM. The FDA has granted breakthrough therapy designation to AYVAKIT for the treatment of moderate to severe indolent SM.

“This regulatory submission for AYVAKIT marks an important milestone to address the medical needs of a broad population of patients with systemic mastocytosis, characterized by debilitating symptoms and lifelong disease burden across multiple organs,” Chidi Office said Blue Becker Hewes, MD, print “By targeting the primary driver of SM with AYVAKIT, we are seeking to transform treatment beyond symptom-directed therapy, modify the course of disease and ultimately, make a meaningful difference in patients’ lives. Our FDA filing marks the first regulatory application seek approval for the treatment of indolent SM, and we look forward to working closely with the agency toward our goal of bringing AYVAKIT to indolent SM patients as quickly as possible.”

In addition, Blueprint Medicines today announced the acceptance of multiple abstracts highlighting registrational PIONEER trial data for oral and poster presentation at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting, February 24-27, 2023. AAAAI plans to post accepted abstracts at on February 32023.

About AYVAKIT (avapritinib)

AYVAKIT (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. For more information, visit 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 …

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