FDA Approves Revumenib for Relapsed, Refractory Acute Leukemia With a KMT2A Translocation

The FDA has approved revumenib (Revuforj) for the treatment of adult and pediatric patients aged 1 year and older with relapsed or refractory acute leukemia and a KMT2A translocation.

Data from the phase 1/2 AUGMENT-101 study (SNDX-5613-0700; NCT04065399) showed that the menin inhibitor led to a complete remission (CR) plus CR with partial hematologic recovery (CRh; CR+CRh) rate of 21.2% (95% CI, 13.8%-30.3%) with a median duration of 6.4 months (95% CI, 2.7-not estimable). In the patients who achieved a CR or CRh with revumenib (n = 22), the median time to response was 1.9 months range, 0.9-5.6).

Moreover, 14% of 83 patients who were dependent at baseline achieved independence of red blood cell (RBC) and platelet transfusions during any 56-day post-baseline period.

Regarding safety, the most common toxicities experienced by at least 20% of patients who received the drug included hemorrhage, nausea, increased phosphate levels, musculoskeletal pain, infection, increased aspartate and alanine aminotransferase levels, febrile neutropenia, increased intact parathyroid hormone, bacterial infection, diarrhea, differentiation syndrome, prolonged electrocardiogram QT, decreased phosphate, increased triglycerides, decreased potassium, reduced appetite, constipation, edema, viral infection, fatigue, and increased alkaline phosphatase.