The US Food and Drug Administration has approved the menin inhibitor ziftomenib (KOMZIFTI) for the treatment of relapsed or refractory NMP1-mutated acute myeloid leukemia in adult patients who have no alternative treatment options.
The FDA approval is supported by the pivotal KOMET-001 trial, which evaluated the safety and efficacy of ziftomenib in 112 patients with relapsed or refractory NMP1-mutated AML. The rate of complete remission plus CR with partial hematologic recovery was 21.4% (95% CI, 14.2, 30.2). The median duration of CR+CRh was 5.0 months (95% CI, 1.9, 8.1) and the median time to first response in patients who achieved a CR or CRh was 2.7 months (range, 0.9 to 15 months), according to a press release from Kura Oncology.
“KOMZIFTI addresses a critical need for adult patients with R/R NPM1-m AML, many of whom are older and unable to tolerate intensive chemotherapy or transplant,” said Eunice Wang, MD, chief of the leukemia service and professor of oncology at Roswell Park Comprehensive Cancer Center. “The clinical data demonstrate deep and durable responses with a manageable safety profile, including no drug-drug interactions and no Boxed Warnings for QTc prolongation or Torsades de Pointes — key advantages for patients on multiple concurrent medications. This approval equips physicians with a new oral therapy to integrate into care and improve outcomes for this vulnerable patient population.”
