Blueprint Medicines Corporation announced the submission of a supplemental new drug application to the U.S. Food and Drug Administration (FDA) for AYVAKIT® (avapritinib) for the treatment of adults with indolent systemic mastocytosis (SM). AYVAKIT was designed to potently 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.
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“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,” said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. “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 seeking 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 www.annualmeeting.aaaai.org on February 3, 2023.
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 AYVAKIT.com. 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 PDGFRA D842V mutation. Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT. AYVAKIT/AYVAKYT is not approved for the treatment of any other indication in the U.S. or Europe.
SOURCE: PR Newswire