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First drug to target mutation causing gastrointestinal tumors approved by FDA

The FDA has Blueprint Medicine's drug, Ayvakit, designed to treat adults with unresectable or metastatic gastrointestinal stromal tumors with a certain type of mutation. 

乐动体育Gastrointestinal stromal tumors, or GISTs, most commonly occur in the stomach or small intestine. GISTs with a certain type of mutation don't respond to typical GIST therapies, and Ayvakit is the first drug approved to target the specific mutation, which occurs in about 10 percent of GISTs. 

乐动体育Ayvakit is a kinase inhibitor, meaning it blocks a type of enzyme called a kinase that helps keep cancer cells from growing. 

In clinical trials, almost 85 percent of patients saw tumor shrinkage with Ayvakit. 

乐动体育The drug was granted breakthrough therapy designation, fast track designation and orphan drug designation. 

乐动体育Read the full news release . 

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