Fact checked byMindy Valcarcel, MS

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May 23, 2023
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FDA approves Ayvakit for indolent systemic mastocytosis

Fact checked byMindy Valcarcel, MS
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The FDA approved avapritinib for treatment of adults with indolent systemic mastocytosis, according to a press release from the agent’s manufacturer.

Avapritinib (Ayvakit, Blueprint Medicines) selectively and potently inhibits KIT D816V, the primary underlying driver of systemic mastocytosis.

Generic FDA News infographic

The agent previously received FDA approval for treatment of aggressive systemic mastocytosis, systemic mastocytosis with an associated hematologic neoplasm, or mast cell leukemia. It also has been approved for adults with unresectable or metastatic gastrointestinal stromal tumor harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, according to the press release.

"Ayvakit is the first and only medicine approved by the FDA to treat indolent systemic mastocytosis, marking a shift in the treatment paradigm from supportive care to disease-modifying therapy,” Becker Hewes, MD, chief medical officer for Blueprint Medicines, said in the release.

Systemic mastocytosis, a rare hematologic disorder, can result in a variety of debilitating systems throughout organ systems, significantly impacting quality of life. Most cases are the indolent form of the disorder.

FDA based the approval for indolent systemic mastocytosis on results of the randomized, double-blind PIONEER trial, which showed significant improvements in disease symptoms and measures of mast cell burden with 25 mg once-daily avapritinib vs. placebo, in addition to best supportive care. Avapritinib also exhibited a favorable safety profile compared with placebo, with mostly mild to moderate adverse reactions including eye edema, dizziness, peripheral edema and flushing. Fewer than 1% of patients experienced serious adverse reactions and discontinuations due to adverse reactions, according to the manufacturer.

“Despite the use of multiple supportive care treatments, a considerable number of patients with indolent systemic mastocytosis continue to experience a substantial disease burden,” Cem Akin, MD, PhD, professor of medicine at University of Michigan and investigator on the PIONEER trial, said in the press release. “Ayvakit advances the treatment of indolent systemic mastocytosis by targeting KIT D816V, the primary underlying cause of the disease, and establishes a new standard of care for a broad population of patients with this disorder.”