The U.S. Food and Drug Administration (FDA) approved revumenib on November 15, 2024, for relapsed or refractory acute leukemia with a KMT2A translocation. This marks a significant development in cancer treatment, as the drug, first administered to a patient at Florida Cancer Specialists & Research Institute, LLC (FCS) in 2019 during a Phase 1 clinical trial, is now accessible nationwide.
The first patient to receive this treatment was referred by FCS medical oncologist/hematologist Muhammad Imam, MD, to FCS Director of Drug Development Manish R. Patel, MD, at the Sarasota Drug Development Unit (DDU). The Sarasota DDU is one of three early-phase clinical trial sites within the statewide practice. This milestone highlights FCS' role in the journey of this drug from trial to FDA approval, emphasizing its potential to target and treat MLL-rearranged leukemia.
"This latest FDA approval of revumenib is a testament to the power of innovation and collaboration—values deeply embedded in our practice's history," said FCS President & Managing Physician Lucio N. Gordan, MD. "Since treating the first patient in 2019, we've believed in the transformative potential of this therapy for leukemia patients. It's moments like these that affirm our longstanding commitment to advancing oncology care through research. This milestone not only validates that belief but also brings hope to countless individuals facing this devastating disease, reflecting the unwavering dedication of our team to improving lives."
Acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) are rare and aggressive blood cancers often resistant to standard treatments. Revumenib is designed to target the genetic abnormalities driving these diseases, showing significant promise in clinical trials by improving remission rates and overall survival.
Dr. Patel, principal investigator for the FCS clinical trial, reflected on the approval: "Conducting this trial was an extraordinary scientific and clinical journey. We observed firsthand the profound impact this therapy had on patients who previously faced limited treatment options. The FDA's approval represents the culmination of years of rigorous research, collaborative effort, and the unwavering commitment of our team. Most importantly, it honors the bravery of the patients who participated and trusted us to pursue this innovative approach."
FCS contributed to the drug's development through its collaboration with Sarah Cannon Research Institute. The FDA approval reflects years of global medical and scientific collaboration.
With three early-phase drug development sites and 30 late-phase locations across Florida, FCS operates one of the nation's largest oncology research programs. Patients have access to over 180 clinical trials at any time, and more than 600 individuals receive cutting-edge treatments annually. Many of the cancer drugs recently approved for use in the U.S. included trials involving FCS' contributions, underscoring its pivotal role in advancing oncology care.
