Syndax's Revuforj (revumenib) Receives FDA Approval for KM2A-Rearranged Leukemia

Key Insights

• Syndax Pharmaceuticals' Revuforj (revumenib) has gained FDA approval for treating adult and pediatric patients with relapsed or refractory KM2A-rearranged acute myeloid leukemia (AML).
• The approval marks Syndax's second FDA nod this year, positioning Revuforj as a significant advancement in leukemia therapy.
• Revuforj's label includes a black box warning for differentiation syndrome and precautions for QT prolongation, necessitating careful patient monitoring.

Syndax Pharmaceuticals Inc. has secured FDA approval for Revuforj (revumenib), a menin inhibitor, for the treatment of adult and pediatric patients with relapsed or refractory KM2A-rearranged (MLLr) acute myeloid leukemia (AML). This marks the company's second FDA approval this year. The drug's label includes a black box warning for differentiation syndrome, a known risk associated with this class of drugs, as well as warnings regarding QT prolongation.

Safety Warnings and Monitoring

According to Syndax CEO Michael Metzger, the FDA has had a "heightened awareness" of differentiation syndrome for approximately six years, leading to the expectation that all drugs in the menin inhibitor class will carry similar warnings. The Revuforj label also mandates monitoring for QT prolongation, an additional safety measure.

Clinical Significance

Revuforj represents a new therapeutic option for patients with KM2A-rearranged leukemia, a subtype of AML associated with poor prognosis. The approval addresses an unmet need in patients who have relapsed or are refractory to existing treatments. While specific clinical trial data was not included in the source article, the approval suggests a clinically meaningful benefit in this patient population.