FDA Approves Ayvakit™ for Gastrointestinal Cancer

Henrietta Strickland
January 13, 2020

The U.S. Food and Drug Administration approved Blueprint Medicines Corp's oral therapy to treat a rare form of cancer that affects the stomach and small intestine, the agency said on Thursday.

The US regulator has cleared Ayvakit (avapritinib) - a KIT and PDGFRA inhibitor - for adults with GIST that can't be treated with surgery or has spread around the body and expresses a mutation on exon 18 known as PDGFRA D842V. "However, today's approval provides patients with the first drug specifically approved for GIST harbouring this mutation", said Dr Richard Pazdur, director of the FDA's Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research.

Avapritib was approved based on results from the phase I NAVIGATOR trial (NCT02508532) which showed durable responses in 43 patients with PDGFRA exon 18 mutations. In the subgroup, the ORR was 89% (95% CI, 75%-97%), with 8% CRs and 82% PRs.

Median duration of response was not reached, but 61% of the patients had responses that lasted 6 months or beyond - 31% had ongoing responses but were followed for less than 6 months.

Common adverse events in the study included abdominal pain, changes in hair color, constipation, cognitive difficulties and dizziness, decreased appetite, diarrhea, edema, fatigue, increased lacrimation, nausea and vomiting, and rash.

Avapritinib may also cause intracranial hemorrhage, central nervous system effects, including cognitive impairment, dizziness, sleep disorders, mood disorders, speech disorders, and hallucinations, and can cause harm to a developing fetus or newborn baby. In contrast, patients who received imatinib had an ORR of 0%. Effective contraception should be used during treatment and for up to 6 weeks after the final dose.

The approval "ushers in a new era of precision medicine in this disease", according to NAVIGATOR investigator Michael Heinrich of Oregon Health & Science University in the US. The trial determined the recommended dose was 300mg taken once daily and measured the overall response rate of patients. It could cost an average $32,000 per month, but that depends on treatment duration and insurance coverage.

"Blueprint Medicines plans to make its avapritinib product available in the United States within a week".

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.

Blueprint is also pursuing FDA approval for Ayvakit in patients with GIST who have exhausted all other options of care - a much larger population of patients.

Other reports by Click Lancashire

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