Priority Review of a new drug application for revumenib in the treatment of patients with R/R KMT2Ar AML and ALL

On March 26, 2024, the U.S. Food and Drug Administration (FDA) granted revumenib, a first-in-class menin inhibitor, Priority Review for a New Drug Application that aims to treat patients with relapsed/refractory (R/R) KMT2A-rearranged acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). Also, it has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 26, 2024. This is as a result of positive data from the AUGMENT-101 trial (NCT04065399).

AUGMENT-101 trial¹

AUGMENT-101 investigated revumenib for the treatment of adult and pediatric patients with KMT2Ar AML and ALL. The study met its primary endpoint and had positive results from secondary endpoints (Figure 1).

Figure 1. Primary and secondary endpoints at the interim analysis stage of AUGMENT-101* 

CR/CRh, complete response/complete response with partial hematologic recovery; MRD, minimal residual disease; OR, overall response.
*Adapted from Syndax.^1
†Patients included who achieved a CR/CRh and were assessed for MRD.
^‡Patients included from the efficacy-evaluable subgroup who achieved an overall response.

Conclusion

Priority Review of this New Drug Application could provide an alternative treatment option for patients who have R/R KMT2A-rearranged AML and ALL. This will address the unmet clinical need for patients with ALL and AML after first-line treatment relapse.