Targeted Treatment for Gastrointestinal Stromal Tumor Mutation Gains FDA Approval

The FDA has approved Blueprint Medicines' oral kinase inhibitor Ayvakit (avapritinib) for rare unresectable or metastatic gastrointestinal stromal tumors (GIST) in adults with the platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation-making it the first precision therapy approved for GIST and the only one for this indication-as well as the subset of patients who harbor the PDGFRA D842V mutation. This medication is the first precision therapy approved to treat a genomically defined population of patients with GIST, according to Blueprint. Approximately 6% of patients with newly diagnosed GIST have PDGFRA exon 18 mutations.

The most common PDGFRA exon 18 mutation is the D842V mutation, which is resistant to all other approved therapies.

With Ayvakit, Blueprint is looking to target about 6% of these patients whose cancers are driven by PDGFRA exon 18 mutations. Patients received avapritinib 300 mg or 400 mg orally once daily until disease progression or unacceptable toxicity.

GISTs arise from specialized nerve cells in the walls of the gastrointestinal tract that aid in the movement of food through the intestines and control various digestive processes. While the median duration of response was not reached, 61 percent of the responding patients with exon 18 mutations had a response lasting six months or longer (31 percent of patients with an ongoing response were followed for less than six months).

In patients with the mutation, the cancer tends to progress in a median of about three to four months after being treated with now available therapies.

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.

Pregnant or breastfeeding patients should not receive avapritinib.

In its approval notice, FDA called for dose reductions or treatment cessation if intracranial hemorrhage occurs and for dose interruptions or cessation if severe central nervous system effects develop (i.e., cognitive impairment, dizziness, hallucinations, or sleep, mood, or speech disorders).

Investigators determined that 300 mg once daily was the recommended dose. Blueprint hopes to make Ayvakit available in the United States in less than a week.

U.S. biotech Blueprint Medicines has its first approved drug, after the FDA backed its oncology drug Ayvakit for a certain kind of stomach cancer.

Blueprint Medicines is dedicated to helping patients with PDGFRA exon 18 mutant GIST access treatment with AYVAKIT and providing robust support throughout their treatment journey.

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