FDA Greenlights Revumenib for Relapsed Leukemia Treatment, First Administered at FCS

The U.S. Food and Drug Administration (FDA) has approved revumenib, a pioneering drug for relapsed or refractory acute leukemia with KMT2A translocation, marking a transformative step in oncology. First administered at the Florida Cancer S&R Institute (FCS) during a Phase 1 trial in 2019, this innovative therapy is now available for patients with limited options facing this aggressive cancer.

A New Era in Leukemia Treatment

Revumenib's approval heralds a breakthrough in the treatment of acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL), rare and aggressive blood cancers often resistant to standard therapies. By specifically targeting genetic abnormalities driving the disease, the drug has demonstrated remarkable efficacy in clinical trials, improving both remission rates and overall survival.

“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 Dr. Lucio N. Gordan. “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.”

The very first patient to ever receive this groundbreaking treatment was referred by FCS hematologist Dr. Muhammad Imam to FCS Director of Drug Development Dr. Manish R. Patel, at the FCS Sarasota DDU, one of three early-phase clinical trial sites. This milestone underscores FCS’ fundamental role in the development of this drug, which has now gained FDA approval for its remarkable ability to target and treat MLL-rearranged leukemia.

Transforming Patient Outcomes

Acute leukemia patients now have renewed hope. Revumenib offers a targeted approach, designed to disrupt the mechanisms that fuel the progression of MLL-rearranged leukemia. While challenges remain in treating these complex cancers, this breakthrough represents a significant step forward.

Dr. Patel, the principal investigator for the clinical trial at FCS, shared his perspective 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.”

Leading Oncology Research

The Florida Cancer S&R Institute’s extensive oncology research program, one of the largest in the U.S., continues to play a pivotal role in the development of new cancer treatments. With three early-phase drug development sites and 30 late-phase locations, FCS has participated in trials leading to the approval of the most recent cancer drugs in the U.S. Its pioneering efforts, in collaboration with partners like the Sarah Cannon Research Institute, ensure that patients benefit from advancements defining the future of cancer care.