The U.S. Food and Drug Administration (FDA) has approved Komzifti (ziftomenib) for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with susceptible NPM1 mutation.
Acute myeloid leukemia (AML) is a cancer that affects the blood and bone marrow. Conditions are generally called “acute” when they develop quickly and have an aggressive course. The signs and symptoms of AML vary but may include easy bruising; bone pain or tenderness; fatigue; fever; frequent nosebleeds; bleeding from the gums; shortness of breath; and/or weightloss. AML is one of the most common types of leukemia among adults and is rarely diagnosed in people under age 40. There are many potential causes of AML such as certain blood disorders, inherited syndromes, environmental exposures, and drug exposures; however, most people who develop AML have no identifiable risk factor.
Ziftomenib is the first and only once-daily, oral menin inhibitor approved for R/R NPM1-mutated AML. The FDA approval is supported by results from the KOMET-001 (NCT04067336) clinical trial evaluating the safety and efficacy of ziftomenib in patients with R/R NPM1-mutated AML.
The rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) was 21.4%. The median duration of CR plus CRh was 5 months and the median time to first response in patients who achieved a CR or CRh was 2.7 months. Of the patients who achieved CR or CRh, 88% did so within six months of initiating ziftomenib.
The most common adverse reactions included increased aspartate aminotransferase, alanine aminotransferase, creatinine, alkaline phosphatase, bilirubin, potassium, and transaminases, decreased potassium, albumin, and sodium, infection without an identified pathogen, hemorrhage, diarrhea, nausea, fatigue, edema, bacterial infection, musculoskeletal pain, differentiation syndrome, pruritus, and febrile neutropenia.
Ziftomenib also includes a Boxed Warning for differentiation syndrome.
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To learn more about AML and other rare cancers, visit https://checkrare.com/diseases/cancers/