Viltolarsen
Viltolarsen is an antisense oligonucleotide medication designed to treat Duchenne muscular dystrophy (DMD) in patients with genetic mutations amenable to exon 53 skipping. It works by binding to dystrophin pre-mRNA to exclude exon 53 during mRNA processing, allowing production of a truncated but functional dystrophin protein. This therapy is specifically indicated for patients with confirmed DMD mutations eligible for exon 53 exclusion.
Viltolarsen is a targeted genetic therapy for Duchenne muscular dystrophy that enables exon 53 skipping to produce a partially functional dystrophin protein.
Key facts at a glance
- ATC
- M09AX12
- RxNorm
- 2389840
- Generic
- Viltolarsen
- Class
- atc-m09
- Brands
- Viltepso
- 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
10 groupspregnancy
pregnancy overallavoid if possible
use only if benefits outweigh risks to the fetus
regular fetal monitoring including ultrasound
Use with caution and monitor for adverse effects.
pregnancy
first trimesteravoid if possible
avoid use during first trimester unless benefits outweigh risks
regular fetal monitoring
Use with caution and monitor for adverse effects.
pregnancy
third trimesteruse with caution
use only if benefits outweigh risks, consider timing of delivery
fetal monitoring and assessment of labor timing
Use with caution and monitor for adverse effects.
lactation
breastfeedingdiscontinue breastfeeding or avoid use
discontinue breastfeeding during treatment or avoid use
monitor infant for adverse effects
pediatric_age
pediatricweight-based dosing: 5 mg/kg or 40 mg/kg once weekly
approved for patients 2 years and older amenable to exon 53 skipping therapy
regular monitoring of renal function, urine protein
geriatric
elderlyno specific adjustment recommended
use with caution, limited clinical experience in elderly
monitor renal function closely
Use with caution and monitor for adverse effects.
renal_impairment
mild renal impairmentno specific dose adjustment in product label
use with caution, monitor renal function
regular monitoring of renal function and urine protein
Use with caution and monitor for adverse effects.
renal_impairment
moderate severe renal impairmentuse with caution, consider dose adjustment
use with caution, limited data in severe renal impairment
intensive renal function monitoring
Use with caution and monitor for adverse effects.
hepatic_impairment
mild hepatic impairmentno specific dose adjustment in product label
use with caution, limited data
monitor liver function tests
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate severe hepatic impairmentuse with caution
limited clinical data, use only if benefits outweigh risks
regular monitoring of liver function
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA approval year
FDA approval for Duchenne muscular dystrophy treatment
Drug class
Classification as antisense oligonucleotide for exon skipping
Therapeutic indication
Approved indication for patients with specific genetic mutation
Administration route
Method of drug administration
Treatment of Duchenne muscular dystrophy (DMD) in patients with documented genetic mutations confirmed to be amenable to exon 53 skipping. Diagnosis requires genetic testing to confirm eligible mutations.
Viltolarsen is an antisense oligonucleotide that binds specifically to exon 53 of dystrophin pre-mRNA. This binding causes exon 53 to be excluded during mRNA splicing, resulting in production of an internally truncated but partially functional dystrophin protein.
This is a targeted genetic therapy requiring confirmed genetic eligibility prior to treatment. Administered via intravenous infusion. Monitor for renal function as with other antisense oligonucleotide therapies. Treatment should be initiated and supervised by specialists experienced in DMD management.
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