The FDA beforehand granted breakthrough remedy designation to ziftomenib for relapsed/refractory NPM1-mutant acute myeloid leukemia.
A New Drug Utility (NDA) has been submitted to the Meals and Drug Administration for ziftomenib for the therapy of grownup sufferers with relapsed or refractory acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM 1) mutation.
The submission of the appliance was introduced in a information launch issued by Kura Oncology, Inc., and Kyowa Kirin Co., Ltd., which described the investigational drug as a extremely selective, once-daily, oral, investigational menin inhibitor.
The FDA had beforehand granted breakthrough remedy designation to the drug for the therapy of adults with relapsed or refractory NPM1-mutant AML in April 2024.
Ziftomenib, in keeping with the information launch, has moreover acquired quick observe and orphan drug designations from the regulatory company. The FDA has a 60-day submitting evaluation interval to find out if the NDA is full and accepted for evaluation. Precedence evaluation was requested and, if granted, would supply a goal FDA evaluation interval of six months after acceptance of the NDA, the information launch defined.
“This NDA submission brings us one step nearer to our objective of advancing ziftomenib to market as a brand new therapeutic possibility for grownup sufferers with R/R NPM1[-mutant] AML, a devastating illness for which there are presently no FDA-approved focused remedy choices,” stated Troy Wilson, president and CEO of Kura Oncology. “We stay up for working carefully with the FDA all through the evaluation course of and are optimistic in regards to the potential of ziftomenib to influence sufferers with NPM1-mutant AML.
“We lengthen our gratitude to the workforce at Kura, our devoted investigators, examine website groups, and most significantly, to the sufferers who participated in our scientific trials, and their households and caregivers, who all helped make this doable. We admire the assist and cooperation we take pleasure in with our associate Kyowa Kirin, and we glance ahead with confidence to the continued progress of this program and our collaboration.”
Ziftomenib, as defined on the Nationwide Most cancers Institute’s web site, prevents the interplay between two proteins, referred to as menin and MLL, which can be wanted for most cancers cells to develop. The drug is presently being evaluated in 9 scientific trials for cancers together with AML, acute lymphoblastic leukemia, acute leukemia and superior gastrointestinal stromal tumors.
The breakthrough remedy designation, the businesses said, was primarily based on findings from the section 2 KOMET-001 scientific trial.
Earlier this 12 months, the businesses introduced optimistic topline outcomes from the KOMET-001 trial, which was submitted for presentation at an upcoming medical convention within the second quarter of 2025. The trial, the businesses said, achieved its major endpoint of full response plus full response with partial hematological restoration, in keeping with a information launch issued in February 2025.
An NDA, because the FDA explains on its web site, is how drug sponsors formally suggest that the company approve a brand new pharmaceutical product on the market and advertising and marketing in america. breakthrough remedy designation, because the company defined, is a course of designed to expedite the event and evaluation of medicine which can be meant to deal with a critical situation and the place preliminary scientific proof signifies that the drug could also be a considerable enchancment over out there remedy.
Roughly 30% of sufferers with AML have NPM1 mutations, the businesses said, noting that whereas sufferers with NPM1-mutant AML have excessive response charges to frontline remedy, relapse charges are excessive and survival outcomes are poor, with solely 30% of sufferers surviving at 12 months within the relapsed/refractory setting. There are, the businesses said, no presently FDA-approved therapies focusing on NPM1-mutant AML.
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