Indolent Systemic Mastocytosis (ISM) & its First FDA Approved Treatment
March 31, 2025 • ISM Content Hub
Avapritinib, branded as Ayvakit, became the first FDA-approved treatment for indolent systemic mastocytosis (ISM) in 2023, marking a major advancement for patients. ISM, driven by the KIT D816V mutation, causes debilitating symptoms across multiple systems, including skin, GI, neurocognitive, and allergic responses. Previously, symptom management relied on multiple over-the-counter therapies with limited relief. Now, patients like Suki—who experienced severe anaphylaxis and hypersensitivities—have a daily oral option that addresses both symptoms and the disease itself, offering meaningful improvements in quality of life.
