WASHINGTON, 10-31-25 – The U.S. Food and Drug Administration has expanded the approval of the menin inhibitor revumenib (Revuforj®) to treat more people with acute myeloid leukemia (AML).
AML is one of the most common blood cancers and also one of the most aggressive. Years of research have shown that AML is not a single disease. There are more than 10 main types of AML and other less common subtypes, each connected to different genetic mutations.
“Because each type of AML is different, there is no one-size-fits-all treatment,” said Lore Gruenbaum, Ph.D., Blood Cancer United® Chief Scientific Officer. “Revumenib is now available for patients with relapsed or refractory NPM1-mutant AML, in addition to AML with KMT2A translocation, for which revumenib had been approved in November 2024. This is great news for patients, but we need to keep working to find treatments for all types of AML.”
New approval will benefit a substantial portion of AML patients
Nearly 1 in 3 AML cases carry the NPM1 gene mutation. Almost half of these patients relapse or are resistant to standard treatment within a year. After relapse, they generally have poor outcomes with less than 10% of patients achieving complete responses with additional therapy.
In the clinical trial that supported this additional approval for revumenib, more than 1 in 5 patients (23.1%) had either a complete remission or a partial remission with undetectable cancer cells, but blood counts not completely returned to normal. Out of 46 patients who required blood or platelet transfusions before treatment, 17% did not after treatment.
Among the 30 patients (of the 64 in the trial) whose disease responded to treatment, five went on to have a stem cell transplant, which can cure AML in some cases.
Blood Cancer United is a leader in AML and menin treatment research
Blood Cancer United devotes a significant portion of its research budget to finding new treatments for AML.
We advance AML research through grant funding to scientists working in academic labs, investments in biotech partnerships as part of our strategic venture philanthropy initiative, the Therapy Acceleration Program (TAP)® and through our sponsorship of the Pediatric Acute Leukemia (PedAL) and Beat AML master clinical trials.
Blood Cancer United has supported research on the menin protein complex for more than 20 years. Our funding contributed to the discovery of how its normal function changes to drive the development of leukemia and how menin inhibitors can reverse this.
“Blood Cancer United has pursued a clear goal for 75 years—to find cures and improve the quality of life of people with blood cancer and their families,” says Dr. Gruenbaum. “Our latest round of research funding, which brings us to over $2 billion in total commitments since our founding, includes a $5 million grant to a group of scientists working to uncover additional mechanisms that drive AML and find ways to target them with known and novel drugs.”