Therapeutic efficacy of Niraparib

Relapsed or Refractory Acute Leukemia with KMT2A Translocation

A single-arm, open-label, multicenter trial evaluated the efficacy of revumenib in 104 adult and pediatric patients. The median age of the patients was 37 years, with 83% diagnosed with acute myeloid leukemia, 15% with acute lymphoblastic leukemia, and 2% with mixed-phenotype acute leukemia. Patients had received a median of 2 lines of prior therapy, and 44% had undergone stem cell transplantation.

The primary efficacy endpoint was the complete remission rate plus the complete remission rate with partial hematologic recovery (CR+CRh). The results showed that the CR+CRh rate was 21.2%, with a median duration of response of 6.4 months. The complete remission (CR) rate was 12.5%, with a median duration of 4.3 months. The complete remission with partial hematologic recovery (CRh) rate was 8.7%, with a median duration of 6.4 months. The median time to achieve CR or CRh was 1.9 months. Among patients who were transfusion-dependent at baseline, 14% achieved transfusion independence; among patients who were transfusion-independent at baseline, 48% maintained transfusion independence.

Relapsed or Refractory Acute Myeloid Leukemia with NPM1 Mutation

Another cohort of the same trial evaluated the efficacy of revumenib in 65 patients. The median age of the patients was 65 years, and 50.8% were aged 65 years or older.

The results showed that the CR+CRh rate was 23.1%, with a median duration of response of 4.5 months. The CR rate was 18.5%, with a median duration of 3.7 months. The CRh rate was 4.6%, with observed durations of response of 1.8, 2.0, and 4.5 months, respectively. The median time to achieve CR or CRh was 2.8 months. Among patients who were transfusion-dependent at baseline, 17% achieved transfusion independence; among patients who were transfusion-independent at baseline, 68% maintained transfusion independence.

FDA，2025.10