FDA Approves Ripretinib for Advanced GISTs

Nick Mulcahy

May 18, 2020

The US Food and Drug Administration (FDA) approved ripretinib (Qinlock, Deciphera Pharmaceuticals) tablets as the first-ever drug specifically approved as a fourth-line treatment for advanced gastrointestinal stromal tumors (GISTs).

A new kinase inhibitor, ripretinib is indicated for patients previously treated with three or more other kinase inhibitor therapies, including imatinib.

"Despite the progress that has been made over the past 20 years in developing treatments for GIST...some patients don't respond to treatment, and their tumors continue to progress. Today's approval provides a new treatment option for patients who have exhausted all FDA-approved therapies for GIST," said Richard Pazdur, MD, acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research.

He explained that the FDA has previously approved four targeted therapies ― imatinib in 2002, sunitinib in 2006, regorafenib in 2013, and avapritinib in 2020.

The new approval is based on the results of a multicenter, randomized, double-blind, placebo-controlled clinical trial that enrolled 129 patients with advanced GISTs whose disease progressed despite prior treatment with the other targeted therapies.

In the trial, patients received ripretinib or placebo once a day in 28-day cycles until disease progression or intolerable toxicity occurred.

Median progression-free survival was 6.3 months in the ripretinib arm, vs 1 month in the placebo arm.

The most common side effects with ripretinib were alopecia, fatigue, nausea, abdominal pain, constipation, myalgia, diarrhea, decreased appetite, palmar-plantar erythrodysesthesia syndrome, and vomiting.

Ripretinib can also cause skin cancer, hypertension, and cardiac dysfunction, manifested as a decrease in ejection fraction.

The FDA collaborated with the Australian Therapeutic Goods Administration and Health Canada on the review of this application as part of Project Orbis.

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