FDA gives Syndax the green light for innovative leukemia drug

A significant advancement in oncology: The U.S. Food and Drug Administration (FDA) has approved the novel drug Revuforj by Syndax Pharmaceuticals for the treatment of a specific form of blood cancer. This involves acute leukemia with a KMT2A translocation, a genetic alteration found in about 10% of patients with acute leukemia. This has led to a 3% increase in Syndax shares in after-hours trading. Revuforj belongs to the class of drugs known as menin inhibitors. These prevent the binding of the menin protein to the MLL protein, thereby stopping the activation of proteins that can promote cancer cell growth. The approval is based on a study with 104 participants, which showed a complete remission rate of 21% and a partial recovery of certain blood cells. Annually, more than 60,000 people in the U.S. are diagnosed with leukemia, according to the National Institutes of Health. This is already the second FDA approval for Syndax this year; previously, the company, together with its partner Incyte, received approval for Niktimvo, a drug for chronic graft-versus-host disease. Revuforj is expected to be available in tablet form at 110 mg and 160 mg in the U.S. later this month. This progress demonstrates how innovations in biotechnology can offer new treatment approaches for previously challenging disease profiles.