First drug to target mutation causing gastrointestinal tumors approved by FDA

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

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. 

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. 

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

Read the full news release here

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