Ziftomenib Submits New Drug Application to FDA for R/R NPM1-Mutant AML

The FDA previously granted breakthrough therapy designation to ziftomenib for relapsed/refractory NPM1-mutant acute myeloid leukemia.

A New Drug Application (NDA) has been submitted to the Food and Drug Administration for ziftomenib for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM 1) mutation.

The submission of the application was announced in a news release issued by Kura Oncology, Inc., and Kyowa Kirin Co., Ltd., which described the investigational drug as a highly selective, once-daily, oral, investigational menin inhibitor.

The FDA had previously granted breakthrough therapy designation to the drug for the treatment of adults with relapsed or refractory NPM1-mutant AML in April 2024.

Ziftomenib, according to the news release, has additionally received fast track and orphan drug designations from the regulatory agency. The FDA has a 60-day filing review period to determine if the NDA is complete and accepted for review. Priority review was requested and, if granted, would provide a target FDA review period of six months after acceptance of the NDA, the news release explained.

“This NDA submission brings us one step closer to our goal of advancing ziftomenib to market as a new therapeutic option for adult patients with R/R NPM1[-mutant] AML, a devastating disease for which there are currently no FDA-approved targeted therapy options,” said Troy Wilson, president and CEO of Kura Oncology. “We look forward to working closely with the FDA throughout the review process and are optimistic about the potential of ziftomenib to impact patients with NPM1-mutant AML.

“We extend our gratitude to the team at Kura, our dedicated investigators, study site teams, and most importantly, to the patients who participated in our clinical trials, and their families and caregivers, who all helped make this possible. We appreciate the support and cooperation we enjoy with our partner Kyowa Kirin, and we look forward with confidence to the continued progress of this program and our collaboration.”

Ziftomenib, as explained on the National Cancer Institute’s website, prevents the interaction between two proteins, known as menin and MLL, that are needed for cancer cells to grow. The drug is currently being evaluated in nine clinical trials for cancers including AML, acute lymphoblastic leukemia, acute leukemia and advanced gastrointestinal stromal tumors.

The breakthrough therapy designation, the companies stated, was based on findings from the phase 2 KOMET-001 clinical trial.

Earlier this year, the companies announced positive topline results from the KOMET-001 trial, which was submitted for presentation at an upcoming medical conference in the second quarter of 2025. The trial, the companies stated, achieved its primary endpoint of complete response plus complete response with partial hematological recovery, according to a news release issued in February 2025.

An NDA, as the FDA explains on its website, is how drug sponsors formally propose that the agency approve a new pharmaceutical product for sale and marketing in the United States. breakthrough therapy designation, as the agency explained, is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and where preliminary clinical evidence indicates that the drug may be a substantial improvement over available therapy.

Approximately 30% of patients with AML have NPM1 mutations, the companies stated, noting that while patients with NPM1-mutant AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% of patients surviving at 12 months in the relapsed/refractory setting. There are, the companies stated, no currently FDA-approved therapies targeting NPM1-mutant AML.

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