The U.S. Food and Drug Administration (FDA) on Friday approved a new drug from Syndax Pharmaceuticals for treating adult and pediatric patients with a specific type of blood cancer, causing the company’s shares to rise by 3% in after-hours trading.
The drug, revumenib, marketed under the brand name Revuforj, is designed to treat acute leukemia with KMT2A rearrangement, a chromosome abnormality involving the KMT2A gene that occurs in about 10% of acute leukemia patients.
Revuforj belongs to a new class of drugs called menin inhibitors, which work by preventing a protein called menin from binding to another protein called MLL. This action halts the activation of proteins that promote cancer cell growth.
The approval was based on early to mid-stage clinical trials involving 104 patients. The drug demonstrated a 21% complete remission rate, with some patients showing partial recovery in specific blood cell levels.
According to the National Institutes of Health, leukemia affects more than 60,000 people annually in the United States.
This marks the second FDA approval for Syndax this year. In August, the FDA approved Niktimvo, a drug developed in collaboration with Incyte, for patients with chronic graft-versus-host disease, a potentially life-threatening immune reaction that can occur after stem cell transplants.
Syndax expects that Revuforj tablets (110 mg and 160 mg) will be available for order in the U.S. later this month.