Elosulfase Alfa
Elosulfase Alfa is an enzyme replacement therapy used to treat Mucopolysaccharidosis IVA (MPS IVA, Morquio A Syndrome), a rare genetic disorder. It works by replacing the deficient enzyme N-acetylgalactosamine-6-sulfatase, which helps break down glycosaminoglycans that accumulate in cells throughout the body. This medication is administered via intravenous infusion and is specifically indicated for patients with confirmed MPS IVA diagnosis.
Elosulfase Alfa is an enzyme replacement therapy for MPS IVA (Morquio A Syndrome) that helps reduce glycosaminoglycan accumulation in patients with this rare lysosomal storage disorder.
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
- A16AB12
- RxNorm
- 1489914
- Generic
- Elosulfase Alfa
- Class
- atc-a16
- Brands
- Vimizim
- 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
- 2 mg/kg once weekly
- Maximum
- consult physician
- Frequency
- once weekly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2 mg/kg once weekly
- Maximum
- consult physician
- Frequency
- once weekly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2 mg/kg once weekly
- Maximum
- consult physician
- Frequency
- once weekly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
11 groupspregnancy
first trimesteruse only if benefits outweigh risks
avoid use during pregnancy unless benefits outweigh risks to the mother
regular fetal monitoring and ultrasound assessments
Use with caution and monitor for adverse effects.
pregnancy
second trimesteruse only if benefits outweigh risks
continue therapy only if benefits justify potential risks to fetus
regular fetal monitoring
Use with caution and monitor for adverse effects.
pregnancy
third trimesteruse only if benefits outweigh risks
consider risks versus benefits; may continue if already on therapy
regular fetal monitoring
Use with caution and monitor for adverse effects.
lactation
breastfeedingno specific adjustment
use with caution; monitor infant for adverse effects
monitor infant for signs of adverse reactions
Use with caution and monitor for adverse effects.
pediatric_age
pediatric 2 5 yearsno adjustment needed
approved for use in pediatric patients; follow recommended dosing regimen
regular monitoring of infusion reactions and efficacy
pediatric_age
pediatric 5 12 yearsno adjustment needed
approved for use in pediatric patients
monitor for infusion reactions and treatment response
geriatric
elderlyuse with caution
limited clinical experience in patients over 65 years; monitor closely
close monitoring for adverse effects
Use with caution and monitor for adverse effects.
renal_impairment
mildno specific adjustment in mild impairment
limited data available; use with caution
standard monitoring
Use with caution and monitor for adverse effects.
renal_impairment
moderate to severeuse with caution
not studied in severe renal impairment; use only if benefits outweigh risks
monitor closely for adverse effects
Use with caution and monitor for adverse effects.
hepatic_impairment
mildno specific adjustment
limited data; use with caution
standard monitoring
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate to severeuse with caution
not studied; use only if benefits outweigh risks
monitor closely for adverse effects
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA approval year
FDA approval date for Elosulfase Alfa (Vimizim)
Drug class
Therapeutic classification
Indication
Approved therapeutic indication
Mechanism of action
Biochemical mechanism of action
Treatment of Mucopolysaccharidosis IVA (MPS IVA, Morquio A Syndrome) in pediatric and adult patients to improve functional endurance and mobility.
Elosulfase Alfa is a recombinant human enzyme (N-acetylgalactosamine-6-sulfatase) that catalyzes the breakdown of the glycosaminoglycans keratan sulfate and chondroitin-6-sulfate, which accumulate in lysosomes due to the enzyme deficiency in MPS IVA patients.
Administered via weekly intravenous infusion. Requires appropriate medical supervision with pre-medication consideration for hypersensitivity reactions. Regular monitoring of respiratory function, endurance, and urine GAG levels may be performed during treatment.
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