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FDA approves first targeted therapy to treat a rare mutation in patients with gastrointestinal stromal tumors |
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FDA approves first targeted therapy to treat a rare mutation in patients with gastrointestinal stromal tumors

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The U.S. Food and Drug Administration has approved Ayvakit (avapritinib) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) – a type of tumor that occurs in the gastrointestinal tract, most commonly in the stomach or small intestine – harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. This approval includes GIST that harbors a PDGFRA D842V mutation, which is the most common exon 18 mutation. Ayvakit is a kinase inhibitor, meaning it blocks a type of enzyme called a kinase and helps keep the cancer cells from growing… (FDA, January 9, 2020)

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