Blueprint Medicines, a global commercial-stage precision medicine drug developer, has received FDA approval for AYVAKIT as a treatment for adults with indolent systemic mastocytosis (ISM). ISM is a rare hematologic disorder that causes debilitating symptoms across multiple organ systems. The majority of systemic mastocytosis (SM) cases are classified as ISM.
AYVAKIT is the first FDA-approved treatment for ISM, and it inhibits the primary underlying driver of the disease. It is a precision therapy that targets the KIT D816V mutation, which is responsible for driving SM. It has been approved by the FDA for the treatment of advanced SM since June 2021. The medicine has shown significant clinical improvements in terms of symptoms and mast cell burden reduction in a large clinical trial.
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