Kura Oncology, a biopharmaceutical company focused on precision medicines for cancer treatment, and Kyowa Kirin, a specialty pharmaceutical company, announced FDA approval of Komzifti (ziftomenib) for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) on November 13th, 2025. This approval grants patients with a susceptible NPM1 mutation, who have no satisfactory alternative treatment options, eligibility for the treatment.
According to a company press release, Ziftomenib is an oral, once-daily menin inhibitor designed to target NPM1 mutations, KMt2A translocations, and FLT3 mutations, which are known to be associated with AML. The treatment intends to address unmet need in R/R NPM1-mutated AML, which occurs in approximately 30% of AML cases and has historically had poor outcomes and low survival rates at relapse.
Approval was based on the KOMET-001 trial, a Phase I/II investigational trial evaluating ziftomenib in R/R NPM1-mutated AML patients. Patients dosed with ziftomenib had a 21.4% rate of complete remission (CR) or complete remission with partial hematologic recovery (CRh). Median duration of CR+CRh was 5.0 months, and the median time to first response in patients who achieved CR or CRh was 2.7 months. 88% of patients who achieved CR or CRh did so within 6 months of treatment.
“[Ziftomenib] combines compelling efficacy, a favorable safety profile, compatibility with concomitant medications, and convenient once-daily oral administration in a population with few effective treatment options. These features highlight [ziftomenib’s] potential to serve as the menin inhibitor of choice in its approved indication,” said Troy Wilson, President, CEO, Kura Oncology, in the release. “Together with our partner, Kyowa Kirin, we remain committed to advancing development of [ziftomenib] across the treatment continuum for AML, where its best-in-class profile offers potential for even greater impact in combination regimens and earlier lines of therapy. We are fully prepared to launch [ziftomenib] today and deliver this new medicine to patients in need.”
“The approval of [ziftomenib] underscores our commitment to advancing precision medicines to address the genetic drivers of disease in hematology and oncology,” said Takeyoshi Yamashita, Executive Vice-President, Chief Medical Officer, Kyowa Kirin, in the release. “In AML, where many patients face severe disease progression and limited treatment options, the evolution toward targeted therapies such as [ziftomenib] represents a major step forward and offers potential to transform existing standards of care. We are proud to partner with Kura Oncology in bringing this important therapy to patients and their families.”
Kura Oncology and Kyowa Kirin entered into a global strategic collaboration for the development and commercialization of ziftomenib in November 2024. According to the release, Kura Oncology leads development, regulatory, and commercial strategy in the United States, and also manufactures ziftomenib. Kyowa Kirin handles development, regulatory, and commercial strategy for territories outside of the United States. Additionally, the companies plan to jointly perform certain commercialization activities as delineated by a co-created U.S. territory commercialization plan.
