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.