KOMZIFTI (ziftomenib)
Komzifti treats adults with acute myeloid leukemia (AML) that has either returned or failed to respond to previous therapies. It specifically helps patients with a susceptible nucleophosmin 1 (NPM1) mutation who lack other effective treatment options. This medication belongs to a class of drugs known as menin inhibitors.
How KOMZIFTI Works
Ziftomenib works by blocking the interaction between menin and the KMT2A protein, which prevents the activation of genes that drive cancer growth. By disrupting this specific protein-protein bond, the drug stops the oncogenic activity caused by mutant NPM1 and encourages leukemic cells to mature into normal, differentiated cells.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2025-11-13
- Patent Cliff
- 2044
- Routes
- ORAL
- Dosage Forms
- CAPSULE
KOMZIFTI Approval History
What KOMZIFTI Treats
1 indicationsKOMZIFTI is approved for 1 conditions since its original approval in 2025. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Acute Myeloid Leukemia
KOMZIFTI Boxed Warning
DIFFERENTIATION SYNDROME Differentiation syndrome, which can be fatal, has occurred with KOMZIFTI. Signs and symptoms may include fever, joint pain, hypotension, hypoxia, dyspnea, rapid weight gain or peripheral edema, pleural or pericardial effusions, pulmonary infiltrates, acute kidney injury, and rashes. If differentiation syndrome is suspected, interrupt KOMZIFTI and initiate oral or intravenous corticosteroids with hemodynamic and laboratory monitoring until symptom resolution; resume KOMZI...
WARNING: DIFFERENTIATION SYNDROME Differentiation syndrome, which can be fatal, has occurred with KOMZIFTI. Signs and symptoms may include fever, joint pain, hypotension, hypoxia, dyspnea, rapid weight gain or peripheral edema, pleural or pericardial effusions, pulmonary infiltrates, acute kidney injury, and rashes. If differentiation syndrome is suspected, interrupt KOMZIFTI and initiate oral or intravenous corticosteroids with hemodynamic and laboratory monitoring until symptom resolution; resume KOMZIFTI upon symptom improvement [see Dosage and Administration ( 2.5 ), Warnings and Precautions ( 5.1 ), and Adverse Reactions ( 6.1 )] . WARNING: DIFFERENTIATION SYNDROME See full prescribing information for complete boxed warning. Differentiation syndrome, which can be fatal, has occurred with KOMZIFTI. If differentiation syndrome is suspected, interrupt KOMZIFTI and initiate oral or intravenous corticosteroids with hemodynamic and laboratory monitoring until symptom resolution; resume KOMZIFTI upon symptom improvement. ( 2.5 , 5.1 , 6.1 )
KOMZIFTI Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in KOMZIFTI's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications KOMZIFTI treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to KOMZIFTI
3 of 18FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
14 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT04067336 | KO-MEN-001 | Ph 1, Ph 2 | active not recruiting | First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia |
| NCT07007312 | KO-MEN-017 2025-521314-25-00 | Ph 3 | recruiting | Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML |
| NCT07411586 | 2025-1468 NCI-2026-01058 | Ph 1 | not yet recruiting | Phase 1/1b Trial Of Olutasidenib And Ziftomenib For NPM1 And IDH1 Co-Mutated Acute Myeloid Leukemia |
| NCT05848687 | IRB-68271 PEDSHEMALL0015, NCI-2023-04129 | Ph 1, Ph 2 | recruiting | TINI 2: Total Therapy for Infants With Acute Lymphoblastic Leukemia II |
| NCT06001788 | KO-MEN-008 | Ph 1 | recruiting | Safety and Tolerability of Ziftomenib Combinations in Patients With Relapsed/Refractory Acute Myeloid Leukemia |
| NCT06440135 | 24-096 | Ph 1 | recruiting | Ziftomenib Maintenance Post Allo-HCT |
| NCT05735184 | KO-MEN-007 | Ph 1 | recruiting | A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML |
| NCT06930352 | OSU-24309 NCI-2025-02414 | Ph 2 | not yet recruiting | Ziftomenib for the Treatment of Patients With NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia Not Eligible for Standard Therapy |
| NCT06769490 | 2024-1193 NCI-2024-10248, NCI-2025-00080 | Ph 1 | recruiting | Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia |
| NCT06655246 | KO-MEN-015 | Ph 1 | recruiting | A Study of Ziftomenib in Combination With Imatinib in Patients With Advanced Gastrointestinal Stromal Tumors (GIST) |
| NCT06397027 | 2023-1008 NCI-2024-03798 | Ph 1 | recruiting | A Phase I Study Investigating the Combination of the Ziftomenib, Venetoclax and Azacitidine in Pediatric Relapsed and Refractory Acute Leukemias |
| NCT07355335 | 25-804 | Ph 1 | not yet recruiting | Ziftomenib + Mezigdomide in Adolesc. and Adults w/ R/R AML |
| NCT06448013 | 2023-0911 NCI-2024-04821 | Ph 1 | recruiting | A Phase I Study Investigating the Combination of the Menin Inhibitor Ziftomenib With Venetoclax and Gemtuzumab in Pediatric Patients With Acute Myeloid Leukemia |
| NCT06376162 | ITCC-101/APAL2020K 2023-505262-28-00 | Ph 1 | recruiting | Ziftomenib in Combination With Chemotherapy for Children With Relapsed/Refractory Acute Leukemia |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
KOMZIFTI FDA Label Details
Indications & Usage
FDA Label (PDF)KOMZIFTI is indicated for the treatment of Acute Myeloid Leukemia.
WARNING: DIFFERENTIATION SYNDROME Differentiation syndrome, which can be fatal, has occurred with KOMZIFTI. Signs and symptoms may include fever, joint pain, hypotension, hypoxia, dyspnea, rapid weight gain or peripheral edema, pleural or pericardial effusions, pulmonary infiltrates, acute kidney in...
KOMZIFTI Patents & Exclusivity
Patents (8 active)
Exclusivity
Pro Intelligence Preview
Deep insights for KOMZIFTI
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2044
- • 13 active patents
Trial Analysis
- • 14 total trials
- • Stage: Growth
Competitive Landscape
- • 18 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment