FDA Approves Ayvakit for the Treatment of Certain Patients with Gastrointestinal Stromal Tumor

The Food and Drug Administration (FDA) approved Ayvakit (avapritinib) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.

“Today's approval of Ayvakit brings forward a new standard of care for patients with PDGFRA exon 18 mutant GIST, a genomically defined population that previously had very limited treatment options. For the first time, we can offer these patients a highly effective treatment that targets the underlying genetic cause of their disease,” said Dr. Michael Heinrich, professor of medicine at Oregon Health & Science University and an investigator on the NAVIGATOR trial that led to the FDA approval, in a press release.

In the NAVIGATOR trial, Ayvakit demonstrated durable responses in patients with PDGFRA exon 18 with an overall response rate of 84%.

The most common side effects included edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, hair color changes, increased lacrimation, abdominal pain, constipation, rash and dizziness.

Blueprint Medicines, the manufacturer of the drug, plans to make Ayvakit available in the United States within a week of its approval.

Check back later on what you need to know about this approval.