Ziftomenib
Ziftomenib is an investigational menin inhibitor being studied for the treatment of acute leukemias, particularly those driven by NPM1 mutations. It works by blocking the interaction between menin and KMT2A (lysine-specific methyltransferase 2A), which prevents recruitment of this complex to leukemogenic gene promoters. This mechanism targets the specific molecular driver in susceptible NPM1-mutant acute leukemias.
Ziftomenib is an experimental menin inhibitor for treating NPM1-mutant acute leukemias by disrupting the menin-KMT2A interaction critical to cancer cell proliferation.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- RxNorm
- 2725985
- Generic
- Ziftomenib
- Class
- unclassified
- Brands
- Komzifti
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- capsule
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 600mg once daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 300mg once daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 600mg once daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
10 groupspregnancy
first trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
second trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring and growth assessments
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
third trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
fetal monitoring and assessment for preterm labor
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingavoid
discontinue breastfeeding during treatment and for at least 1 week after last dose
not applicable
Use of this medication in this population requires careful consideration of risks and benefits.
geriatric
elderly 65 and olderno specific adjustment
use with caution; monitor for adverse effects
regular monitoring of blood counts and organ function
Use with caution and monitor for adverse effects.
pediatric_age
children under 18not established
safety and efficacy not established in pediatric patients
not applicable
renal_impairment
mild to moderateno adjustment needed
no dose adjustment for mild to moderate renal impairment
monitor renal function regularly
Use with caution and monitor for adverse effects.
renal_impairment
severeuse with caution
use with caution; consider dose reduction if needed
close monitoring of renal function
Use with caution and monitor for adverse effects.
hepatic_impairment
mild to moderateno adjustment needed
no dose adjustment for mild to moderate hepatic impairment
monitor liver function tests
Use with caution and monitor for adverse effects.
hepatic_impairment
severeuse with caution
use with caution; consider dose reduction
close monitoring of liver function
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA Approval Date
FDA approval for relapsed or refractory AML with NPM1 mutation
Drug class
Targeted therapy classification
Indication
Therapeutic indication
Mechanism of action
Molecular mechanism of action
Investigational agent for acute leukemias, with primary focus on acute myeloid leukemia (AML) harboring NPM1 mutations. Clinical trials are ongoing to establish efficacy and safety in this patient population.
Ziftomenib is a small molecule menin inhibitor that blocks the protein-protein interaction between menin and KMT2A. In NPM1-mutant leukemias, this complex is recruited to leukemogenic gene promoters, driving cancer progression. By inhibiting this interaction, Ziftomenib disrupts oncogenic gene expression programs essential for leukemia cell survival.
Ziftomenib is an investigational drug not yet FDA-approved. As an unclassified agent, it remains under clinical evaluation. Safety and efficacy data are still being collected through ongoing trials. Patients should only receive this medication through participation in clinical studies or expanded access programs under physician supervision.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy