This company’s treatment is addressing the root cause of a rare blood disorder

Indolent Systemic Mastocytosis (ISM) is a rare blood disorder that affects some 32,000 adults in the U.S., causing allergy-like reactions across the body and impacting the daily lives of patients, who often limit work or daily activities to isolate themselves and protect against unpredictable triggers.



Blueprint Medicines became the first company to offer a precision treatment for ISM in May 2023, when the FDA approved Ayvakit (avapritinib). First approved in 2021 to treat more aggressive mast cell disease variants (which can cause organ dysfunction), Ayvakit’s ISM approval changes the treatment paradigm for the condition from supportive care to targeting the primary underlying cause of the disease.



In clinical trials, Ayvakit demonstrated statistically significant reductions in ISM symptoms compared to a placebo at 24 weeks.



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