Roswell Park’s Role in FDA Approval of New Oral AML Drug Opens Transplant Pathways
USA: Roswell Park has announced a pivotal advance in treatment options for adults with relapsed or refractory acute myeloid leukemia after the FDA approved a new targeted agent called zifomenem three years after the center enrolled the first human patient. The approval applies to patients whose disease carries a specific NMP1 mutation and marks a milestone for the medical team led by Dr. Eunice Wang, chief of the leukemia service, who oversaw the early human use and development at the hospital.
Zifomenem is an oral pill taken once daily and is designed as precision therapy for this molecular subset of AML. In the relapse and refractory setting the drug has produced complete remissions in 23% of treated patients, defined as restoration of normal blood counts and clearance of detectable disease. Roswell’s early clinical work helped move the drug from first-in-human testing to regulatory approval and into broader clinical practice.
Researchers are already studying combinations of zifomenem with other targeted drugs and with conventional chemotherapy, reflecting a strategy of pairing precision agents to increase depth and durability of responses. Clinicians emphasize the importance of less toxic, at-home pill regimens for an illness that most commonly presents in older adults; the average age at diagnosis is about 70, and many patients are in their 60s, 70s, or 80s. AML affects roughly 13,000 people each year in the United States.
Physicians described the impact of the new therapy as measured in months of added survival—examples cited included gains of roughly 6 months, 10–12 months, and 15 months—and, crucially, as a bridge to potentially curative bone marrow transplant. For patients with limited options, the drug offers not only disease control but renewed access to transplantation as a curative goal.
