FDA Approves a New Treatment for Gastrointestinal Stromal Tumors

According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved avapritinib (marketed as AYVAKIT) as a treatment for gastrointestinal stromal tumors in cases when the PDGFRA exon 18 mutation is present. The drug was developed by the precision therapy company Blueprint Medicines Corporation, which is focused on developing treatments for genomically linked cancers and rare diseases. The approval makes AYVAKIT the first precision therapy for this group of genomically defined patients.

About Gastrointestinal Stromal Tumors

Gastrointestinal stromal tumors (GISTs) is a form of tumor that can appear in the digestive tract. These tumors appear in the interstitial cells of Cajal, which control smooth muscle activity. These tumors are connected to mutations that affect the KIT gene or the PDGFRA gene in the vast majority of cases. In most cases, these mutations appear sporadically, and are only rarely inherited. Typically they occur as part of another genetic disorder when they are inherited. They can occur in a number of locations along the digestive tract, most often the stomach, small intestine, or esophagus. Symptoms may include abdominal pain, difficulty swallowing, liver metastases, and gastrointestinal bleeding. Treatment usually involves surgery or medications when surgery is not possible. Gastrointestinal stromal tumors generally do not respond to radiation or chemotherapy. To learn more about gastrointestinal stromal tumors, click here.

A New Opportunity for Precision Treatment

The approval of this new treatment follows promising clinical trial findings in which AYVAKIT was able to produce an overall response rate of 84 percent in patients with gastrointestinal stromal tumors that carried the PDGFRA exon 18 mutation. The drug is expected to be effective for around six percent of gastrointestinal stromal tumor patients. This mutation displayed considerable resistance to all other approved treatments. The approval of this new treatment will give these patients access to an effective therapy for the first time.

The approval of this treatment will also highlight the need for genetic testing for newly diagnosed patients, a process which is increasing in importance for successful cancer treatment as more precision therapies become available. AYVAKIT is currently not approved by the FDA for any other uses.


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