- The FDA has accepted the New Drug Application for ziftomenib, giving it Priority Review.
- Ziftomenib showed positive results in the Phase 2 KOMET-001 trial, achieving its primary endpoint.
- If approved, ziftomenib would be the first menin inhibitor for treating NPM1-mutant acute myeloid leukemia (AML).
Kura Oncology (KURA, Financial) and Kyowa Kirin have announced that the FDA has accepted their New Drug Application (NDA) for ziftomenib, an investigational menin inhibitor designed to treat adult patients with relapsed or refractory NPM1-mutant acute myeloid leukemia (AML). The application has been granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date set for November 30, 2025.
The NDA is based on data from the Phase 2 KOMET-001 clinical trial, which successfully met its primary endpoint of achieving complete remission plus CR with partial hematological recovery. The study also reported a favorable safety profile, with only 3% of treatment-related discontinuations and limited myelosuppression, a common concern in AML treatments.
Ziftomenib has received multiple designations from the FDA, including Breakthrough Therapy, Fast Track, and Orphan Drug. These designations underscore the drug's potential impact, especially since it would be the first menin inhibitor approved for treating NPM1-mutant AML if cleared by the FDA. This specific mutation occurs in approximately 30% of AML cases, often presenting unique clinical challenges and a high relapse rate.
The acceptance of this NDA marks a significant milestone for Kura Oncology and Kyowa Kirin as they work towards providing new treatment options for patients facing this aggressive form of leukemia. With currently limited options and a poor prognosis for those with NPM1-mutant AML, ziftomenib could play a pivotal role in changing treatment paradigms.
