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FDA Alerts
Hematology

FDA approves avapritinib for indolent systemic mastocytosis

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The U.S. Food and Drug Administration (FDA) has approved Ayvakit (avapritinib) for the treatment of adults with indolent systemic mastocytosis (ISM), according to a press release. This is the first and only approved medicine to treat ISM.

Approval was based on data from the PIONEER trial, a double-blind and placebo-controlled study in which patients received either avapritinib or placebo along with best supportive care. Avapritinib showed significant improvements compared to placebo in primary and secondary endpoints, including overall symptoms and measures of mast cell burden.

Avapritinib was well-tolerated and had a favorable safety profile when compared to placebo. Most adverse reactions were mild to moderate in severity. The most common adverse reactions associated with avapritinib were eye edema, dizziness, peripheral edema, and flushing. Serious adverse reactions and discontinuations due to adverse reactions were rare, occurring in less than 1 percent of patients.

“After decades of caring for people with indolent systemic mastocytosis, I have seen firsthand its profound impact on patients’ underlying mast cell burden, symptoms, physical and mental health, and ability to work and participate in daily activities,” said Cem Akin, MD, PhD, Professor of Medicine at the University of Michigan, and an investigator on the PIONEER trial in the press release. “Despite the use of multiple supportive care treatments, a considerable number of patients with indolent systemic mastocytosis continue to experience a substantial disease burden. 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. Ayvakit delivered statistically significant and consistent clinical improvements in the PIONEER trial, and based on these practice-changing data, I feel a tremendous sense of hope for the future for all those affected by the disease.”

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