On Nov. 13, 2025, the US Food and Drug Administration (FDA) approved Komzifti^™ (ziftomenib – Kura Oncology and Kyowa Kirin), a selective oral menin inhibitor, for the treatment of adults who have relapsed or refractory (R/R) acute myeloid leukemia (AML) harboring a susceptible nucleophosmin 1 (NPM1) mutation, who have no satisfactory alternative treatment options. The recommended dose is 600 mg orally once daily until disease progression or unacceptable toxicity. According to the company, the drug will launch within the next few business days at a cost of $48,500 per month of therapy. Access and network information is not known at this time. For complete prescribing information, see here.

At a Glance

• Brand Drug: Komzifti^™ (ziftomenib)
• Manufacturer: Kura Oncology and Kyowa Kirin
• Date Approved: Nov. 13, 2025
• Indication: Treatment of adult patients with relapsed or refractory AML with a susceptible NPM1 mutation who have no satisfactory alternative treatment options.
• Dosage Forms Available: Available as 200mg capsules
• Launch Date: within the next few business days
• Estimated Annual Cost: $582,000 per year
• FDA Designation:  Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review
• Komzifti is a menin inhibitor that disrupts the menin–KMT2A interaction, a critical driver of aberrant HOX/MEIS1 gene expression in NPM1-mutated and KMT2A-rearranged AML. This inhibition restores normal hematopoietic differentiation and induces apoptosis of leukemic blasts.
• AML is an aggressive cancer of the blood and bone marrow, where immature white blood cells, called myeloblasts, multiply uncontrollably. These abnormal cells impair the body’s ability to fight infections, carry oxygen and stop bleeding. AML is the most common acute leukemia in adults, with approximately 22,000 new cases and 11,000 deaths expected in 2025. NPM1 mutations occur in approximately 30% of newly diagnosed AML cases.
• Approval was based on the KOMET-001 trial, a multicenter, open-label, single-arm study of 112 adults. The trial showed a 21.4% complete remission (CR) plus CR with partial hematologic recovery (CRh), with a median duration of five months.
• Komzifti carries a boxed warning for differentiation syndrome, a potentially fatal condition caused by the rapid maturation and proliferation of leukemic cells. Symptoms may include fever, low blood pressure, fluid buildup in the lungs or heart, kidney injury, and respiratory distress. If suspected, treatment should be interrupted and corticosteroids initiated with close monitoring until symptoms resolve. There are also warnings for QTc interval prolongation and embryo-fetal toxicity.
• One Nov. 15, 2024, the FDA approved Revuforj^™ (revumenib -Syndax Pharmaceuticals), the first oral menin inhibitor for (R/R) acute leukemia who have a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older.
• Komzifti is being evaluated in combination regimens in ongoing Phase III trials for both intensive and non-intensive combination regimens in patients with newly diagnosed NPM1 or KMT2A-rearranged AML. Taiho Oncology has submitted a New Drug Application for Inqovi^® (decitabine/cedazuridine) tablets, with a PDUFA target action date of February 25, 2026, for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) who are not candidates for intensive induction chemotherapy.