Syndax Pharmaceuticals has received FDA approval for Revuforj (revumenib), a first-in-class menin inhibitor, for the treatment of adult and pediatric patients (one year and older) with relapsed or refractory acute leukemia harboring KMT2A translocations. This approval marks a significant advancement for patients with this aggressive form of leukemia, who often face poor prognoses with standard treatments.
The FDA's decision was based on data from early-stage clinical trials involving 104 patients treated with Revuforj. The results showed a complete remission rate of just over 20%, with a median duration of approximately six months. Furthermore, 23% of the patients who achieved remission went on to receive a stem cell transplant, highlighting the potential for Revuforj to bridge patients to potentially curative therapies.
Differentiation Syndrome Warning
The Revuforj label includes a black box warning for differentiation syndrome, a potentially fatal complication associated with acute leukemia therapies. In clinical trials, 29% of patients treated with Revuforj experienced differentiation syndrome. This adverse event requires careful monitoring and management by experienced healthcare professionals.
Addressing an Unmet Need
KMT2A-rearranged acute leukemia is characterized by a high relapse rate and a median overall survival of less than one year. Syndax estimates that approximately 2,000 individuals in the U.S. have this specific subtype of acute leukemia. Revuforj offers a targeted approach to treating this disease, inhibiting the interaction between the menin protein and the KMT2A fusion protein, which drives leukemic cell growth.
Commercialization and Pricing
Syndax plans to launch Revuforj in two dose forms (110 mg and 160 mg) later in November, with a lower dose (25 mg) becoming available next year. The drug is priced at approximately $475,000 per year before rebates and discounts. Despite the high cost, analysts predict that most patients will have commercial insurance coverage due to the younger age of the affected population. Peak annual sales for Revuforj in the KMT2A indication are projected to reach $200 million.
