Imetelstat
Imetelstat is a telomerase inhibitor used in the treatment of certain blood disorders. It works by directly targeting and inhibiting the enzyme telomerase, which plays a role in abnormal blood cell production.
Imetelstat is a telomerase inhibitor used to treat certain blood disorders such as myelodysplastic syndromes by blocking the enzyme that contributes to abnormal cell growth.
Key facts at a glance
- ATC
- L01XX80
- RxNorm
- 2685236
- Generic
- Imetelstat
- Class
- atc-l01
- Brands
- Rytelo
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- 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
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
8 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 third trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring and ultrasound
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingnot applicable
do not breastfeed during treatment and for at least 2 weeks after last dose
not applicable
Use of this medication in this population requires careful consideration of risks and benefits.
pediatric_age
children adolescentsnot established
safety and efficacy not established in pediatric patients
not applicable
geriatric
elderly 65no specific dose adjustment required
use with caution due to increased susceptibility to myelosuppression
close monitoring of blood counts
Use with caution and monitor for adverse effects.
renal_impairment
severe renal impairmentreduce dose to 4.7 mg/kg
use with caution in patients with severe renal impairment
monitor renal function and blood counts
Use with caution and monitor for adverse effects.
renal_impairment
moderate renal impairmentno specific adjustment required
use with caution and monitor closely
monitor renal function and blood counts
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate severe hepatic impairmentno specific adjustment required
use with caution in patients with hepatic impairment
monitor liver function tests and blood counts
Use with caution and monitor for adverse effects.
Statistics and clinical data
Drug class
Telomerase inhibitor oligonucleotide
FDA approval year
FDA approval for myelodysplastic syndromes
Indication
Therapeutic indication
Manufacturer
Drug manufacturer information
Imetelstat is indicated for the treatment of myelodysplastic syndromes (MDS), particularly in adult patients with lower-risk disease who have not responded to or lost response to prior erythropoiesis-stimulating agents. It targets hematologic malignancies characterized by elevated telomerase activity.
Imetelstat is an oligonucleotide that specifically binds to the template region of the RNA component of human telomerase (hTR). By binding to this region, it inhibits telomerase enzymatic activity and prevents telomere binding, thereby disrupting the telomere maintenance mechanism essential for abnormal cell survival and proliferation.
As an antineoplastic agent (ATC class L01), Imetelstat may cause significant myelosuppression, including neutropenia and thrombocytopenia. Regular blood count monitoring is required during treatment. Administration is typically via intravenous infusion. Patients should be monitored for signs of hepatotoxicity and infusion-related reactions.
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