The FDA approved avapritinib (Ayvakit) as the first and only therapy for indolent systemic mastocytosis in adults, a rare hematologic disorder that affects multiple organ systems and significantly impacts quality of life. This approval extends avapritinib’s use, as it was previously sanctioned for advanced systemic mastocytosis in June 2021. Avapritinib works by selectively inhibiting KIT D816V, a primary driver of the disease. The approval is based off of results from the PIONEER trial, a double-blind, placebo-controlled study, where avapritinib showed significant improvement over placebo in primary and key secondary endpoints, including overall symptoms and measures of mast cell burden.
The PIONEER trial data demonstrated consistent clinical benefits through 48 weeks of avapritinib treatment, emphasizing the oral therapy’s impact on reducing disease burden and symptoms. The approval of avapritinib represents a milestone for the systemic mastocytosis community, offering new hope to patients and families affected by this disorder.
Reference: The ASCO Post Staff. FDA Approves Avapritinib for Indolent Systemic Mastocytosis. The ASCO Post. Published May 23, 2023. Accessed April 5, 2024. https://ascopost.com/news/may-2023/fda-approves-avapritinib-for-indolent-systemic-mastocytosis/
Link: https://ascopost.com/news/may-2023/fda-approves-avapritinib-for-indolent-systemic-mastocytosis/
