(Reuters) -The U.S. Food and Drug Administration on Thursday approved Kura Oncology and Japanese partner Kyowa Kirin’s drug to treat a rare form of blood cancer that has returned or stopped responding to treatment.
The approval offers new hope for patients with a particularly aggressive form of blood cancer who have exhausted other treatment options.
The once-daily pill, which will be sold under the brand name Komzifti, is the first approved therapy for acute myeloid leukemia patients with an NPM1 mutation, a genetic change found in about 30% of cases.
Acute myeloid leukemia is an aggressive blood cancer that affects the bone marrow and blood.
The approval was based on a study of 112 patients in which about 21% achieved complete or near-complete remission, with responses lasting a median of five months. Some patients also became independent of blood transfusions during treatment.
The recommended dose is 600 milligrams taken once daily until the disease worsens or side effects become unacceptable, the agency said.
The therapy will carry warnings for risks such as differentiation syndrome, heart rhythm changes and potential harm to unborn babies.
Komzift is the first menin inhibitor, a protein that plays a role in cancer cell growth, to receive full FDA approval for this indication.
Shares of California-based Kura were up about 1% in morning trading.
(Reporting by Kamal Choudhury and Christy Santhosh in Bengaluru; Editing by Shailesh Kuber)
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