Blueprint Medicines recently announced the approval of AYVAKIT® (avapritinib) for the expanded indication of adults with Indolent Systemic Mastocytosis (ISM). AYVAKIT was previously approved for the treatment of adults with Advanced Systemic Mastocytosis (SM), including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL), as well as for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. PANTHERx Rare Pharmacy dispenses AYVAKIT for all previously approved indications and will now dispense AYVAKIT for the ISM patient population.
SM is a rare disease that is driven by the KIT D816V mutation in about 95% of cases. This mutation causes the uncontrolled growth and activation of mast cells, which in turn results in chronic, often unpredictable, and potentially severe symptoms across many different organ systems. The vast majority of people living with SM are diagnosed with ISM. People living with ISM may experience a broad range of symptoms. Common symptoms include anaphylaxis, rash, itching, diarrhea, brain fog, fatigue, and bone pain, and these symptoms often persist despite treatment with multiple therapies aimed at improving symptoms. These symptoms can have profound effects on people’s lives and can interfere with their ability to work and complete their normal daily activities, and may even lead to patients isolating themselves to protect against possible unexpected triggers.
AYVAKIT is a kinase inhibitor designed to target the KIT D816V mutation, the primary driver of the disease. In the registrational clinical trial for ISM, treatment with AYVAKIT significantly reduced mast cell burden and improved patient symptoms. The most common adverse reactions for AYVAKIT (≥10 percent) were eye edema, dizziness, peripheral edema and flushing.
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