Silver Spring, Maryland: The U.S. Food and Drug Administration (FDA) granted approval on Friday to Syndax Pharmaceuticals’ new drug, Revuforj, for the treatment of both adult and pediatric patients suffering from a specific type of blood cancer. The approval led to a 3% rise in the company’s stock during after-hours trading.
Revuforj, also known as revumenib, is designed to target acute leukemia with KMT2A translocations, chromosomal rearrangements involving the KMT2A gene. These genetic abnormalities are present in approximately 10% of acute leukemia cases.
This drug belongs to a new class called menin inhibitors. It works by preventing the binding of menin—a protein involved in the activation of cancer-causing proteins—to MLL (mixed-lineage leukemia), ultimately halting the growth of cancer cells.
The FDA’s approval was supported by results from a phase 1/2 clinical trial, involving 104 patients. In this trial, Revuforj demonstrated a 21% complete remission rate, with partial recovery observed in specific blood cell levels.
Leukemia is a significant health challenge, affecting more than 60,000 people in the United States each year, according to the National Institutes of Health.
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This approval marks the second FDA breakthrough for Syndax in 2024. In August, the FDA also approved a treatment in partnership with Incyte for chronic graft-versus-host disease, a life-threatening complication following stem cell transplants.
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Syndax announced that Revuforj, available in 110 mg and 160 mg tablets, is expected to be available for order in the U.S. later this month.
