The FDA has approved a new drug called revumenib that can be taken orally to treat acute leukemia in patients 1 year or older. The drug will be marketed under the brand name Revuforj.
Revuforj is a new class of drug for leukemia, meaning it works differently than other medicines that treat the disease. Unlike existing therapies, Revuforj directly targets the genetic changes that cause leukemia. It selectively blocks a protein (menin) that interacts with the KMT2A gene linked to leukemia, restoring normal function and halting the growth of leukemic cells.
Leukemia that is caused by this genetic change is fast-growing and associated with poor treatment outcomes, frequent relapses, and survival of less than a year.
The new approval is a “major breakthrough,” said Ghayas C. Issa, MD, associate professor of leukemia at The University of Texas MD Anderson Cancer Center, in a statement from Syndax, the drug’s maker.
"The significant clinical benefit and robust efficacy seen with Revuforj represents a substantial improvement over what has been historically observed in these patients with previously available therapies and has the potential to be an important new treatment option for patients," Issa said.
The results of a 104-patient clinical trial assessing the drug’s effectiveness and safety showed that 21% of patients achieved complete remission that lasted for about six and a half months. On average, it took about two months for remission to start. Twenty-four patients were able to proceed to stem cell transplants.
The most common side effects include bleeding, infections, immune reactions, swelling, muscle pain, fatigue, appetite loss, nausea, constipation, diarrhea, electrolyte changes, blood abnormalities such as increased levels of liver enzymes, and a condition called QT prolongation where the heart takes longer than normal between beats.
Syndax expects 110-milligram and 160-milligram tablets to be available at specialty distributors and pharmacies this month. Dosages of 25 milligrams intended for patients under 40 kilograms (about 88 pounds) are expected by early 2025. In the meantime, a liquid oral solution will be available to patients weighing less than 40 kilograms through an Expanded Access Program. You can talk to your health care provider for more details.
