Vestronidase Alfa
Vestronidase alfa is an enzyme replacement therapy used to treat Mucopolysaccharidosis VII (MPS VII or Sly syndrome), a rare genetic lysosomal storage disorder. It works by providing recombinant human beta-glucuronidase to break down accumulated glycosaminoglycans in cells throughout the body, reducing multisystem damage.
An enzyme replacement therapy for MPS VII that reduces glycosaminoglycan accumulation in patients with confirmed beta-glucuronidase deficiency.
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
- ATC
- A16AB18
- RxNorm
- 1989819
- Generic
- Vestronidase Alfa
- Class
- atc-a16
- Brands
- Mepsevii
- 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
- 4 mg/kg
- Maximum
- consult physician
- Frequency
- every 2 weeks
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 4 mg/kg
- Maximum
- consult physician
- Frequency
- every 2 weeks
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 4 mg/kg
- Maximum
- consult physician
- Frequency
- every 2 weeks
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
8 groupspregnancy
first trimesteravoid if possible
use only if benefits outweigh potential risks to fetus
regular fetal monitoring and ultrasound assessments
Use with caution and monitor for adverse effects.
pregnancy
second trimesteravoid if possible
use only if benefits outweigh potential risks
fetal growth monitoring
Use with caution and monitor for adverse effects.
pregnancy
third trimesteravoid if possible
use only if benefits outweigh potential risks
fetal monitoring and assessment of labor
Use with caution and monitor for adverse effects.
lactation
breastfeedingnot recommended
discontinue breastfeeding or discontinue drug
monitor infant for any adverse effects
pediatric_age
pediatricno adjustment
indicated for patients 5 months and older with confirmed MPS VII
regular monitoring of infusion reactions and antibody development
geriatric
elderlyno specific adjustment
use with caution due to limited clinical experience
monitor renal and cardiac function
Use with caution and monitor for adverse effects.
renal_impairment
mild to moderateno adjustment
use with caution, no formal studies conducted
monitor renal function during treatment
Use with caution and monitor for adverse effects.
hepatic_impairment
mild to moderateno adjustment
use with caution, no formal studies conducted
monitor liver function tests
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA Approval Year
FDA approval date for Vestronidase Alfa (Mepsevii)
Drug class
Therapeutic classification of Vestronidase Alfa
Indication
Approved therapeutic indication
Mechanism of action
Molecular mechanism of action
Treatment of Mucopolysaccharidosis VII (MPS VII or Sly syndrome) in pediatric and adult patients with confirmed beta-glucuronidase deficiency.
Vestronidase alfa is a recombinant form of human beta-glucuronidase (GUS) that replaces the deficient endogenous enzyme, allowing proper breakdown of accumulated glycosaminoglycans within lysosomes and reducing cellular damage across multiple organ systems.
Administered via intravenous infusion; requires appropriate medical setting for administration. Monitor for infusion-related reactions including anaphylaxis. Use caution in patients with compromised respiratory or cardiac function. Limited clinical data available due to rare disease prevalence.
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