Eteplirsen
Eteplirsen is an antisense oligonucleotide therapy designed to treat Duchenne muscular dystrophy (DMD) in patients whose genetic mutations are amenable to exon 51 skipping. By binding to dystrophin pre-mRNA, it causes exclusion of exon 51 during mRNA processing, enabling production of a truncated but functional dystrophin protein that helps stabilize muscle cells.
Eteplirsen is a targeted genetic therapy for certain DMD patients that promotes production of a shortened dystrophin protein to slow disease progression.
Key facts at a glance
- ATC
- M09AX06
- RxNorm
- 1810569
- Generic
- Eteplirsen
- Class
- atc-m09
- Brands
- Exondys
- 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
use only if potential benefits outweigh potential risks to fetus; discuss with healthcare provider
regular prenatal monitoring
Use with caution and monitor for adverse effects.
lactation
breastfeedingdiscontinue or avoid
it is unknown whether eteplirsen is excreted in human breast milk; breastfeeding mothers should consult physician
monitor infant for adverse effects
Use with caution and monitor for adverse effects.
pediatric_age
children 5 17 yearsno adjustment
indicated for pediatric patients with DMD amenable to exon 51 skipping; dose based on weight
regular monitoring of renal function and efficacy
geriatric
elderlyuse with caution
limited data available in patients over 65 years; not typically used in geriatric population due to DMD being a pediatric disease
monitor renal function closely
renal_impairment
mild to moderatemonitor renal function
eteplirsen is primarily excreted by kidneys; dose adjustment may be required based on renal function
regular monitoring of renal function tests
Use with caution and monitor for adverse effects.
renal_impairment
severereduce dose or increase interval
use with extreme caution; consider dose reduction in severe renal impairment
frequent renal function monitoring
Use with caution and monitor for adverse effects.
hepatic_impairment
mild to moderateno specific adjustment
eteplirsen is not metabolized by CYP450 enzymes; limited data on hepatic impairment effects
monitor liver function tests
Use with caution and monitor for adverse effects.
hepatic_impairment
severeuse with caution
limited clinical data available; individual assessment required
regular hepatic function monitoring
Use with caution and monitor for adverse effects.
Statistics and clinical data
Drug class
Eteplirsen is a phosphorodiamidate morpholino oligomer (PMO) designed to skip exon 51
FDA approval year
Accelerated approval granted September 2016
Primary indication
Treatment of DMD in patients with confirmed mutation of the DMD gene that is amenable to exon 51 skipping
Manufacturer
Commercial name: Exondys 51
Treatment of Duchenne muscular dystrophy in patients with confirmed dystrophin gene mutations amenable to exon 51 skipping (approximately 13% of DMD cases). Diagnosis requires genetic testing to confirm eligible mutation.
As an antisense oligonucleotide, eteplirsen specifically binds to exon 51 of dystrophin pre-mRNA, redirecting cellular machinery to exclude this exon during mRNA splicing. This produces a shortened but partially functional dystrophin protein, which may help preserve muscle integrity.
Administered as weekly intravenous infusion. Requires genetic testing to confirm exon 51 skip eligibility prior to treatment. Clinical benefits vary; some patients show increased dystrophin production while others may experience disease progression. Monitor for infusion reactions and kidney function.
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