FDA grants an expanded Ayvakit label to Blueprint Medicines
The FDA on Monday approved Blueprint Medicines’ Ayvakit for a much broader, though still rare population of patients with indolent systemic mastocytosis, a disease in which too many mast cells — a type of white blood cell — build up in the body.
The drug is the first approved treatment for the condition, and the FDA granted Blueprint a broader label — it’s now approved for all patients with indolent SM — than the clinical trials, which only included patients with moderate-to-severe symptoms. The drug was approved based on data showing that it decreased symptoms of the disease significantly more than placebo over 24 weeks.
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