Elosulfase Alfa
Vimizim (elosulfase alfa) is an enzyme replacement therapy used to treat Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, a rare genetic disorder. It works by providing a recombinant form of the enzyme N-acetylgalactosamine-6-sulfatase to help break down and reduce the accumulation of glycosaminoglycans in the body. This medication is specifically indicated for patients with confirmed MPS IVA diagnosis and is administered via intravenous infusion.
Vimizim is an enzyme replacement therapy for MPS IVA (Morquio A syndrome) that helps reduce glycosaminoglycan buildup in the body.
Key facts at a glance
- RxNorm
- 1490048
- Generic
- Vimizim
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- 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.
Vimizim is indicated for the treatment of Mucopolysaccharidosis type IVA (MPS IVA), a rare autosomal recessive lysosomal storage disorder caused by deficiency of the N-acetylgalactosamine-6-sulfatase (GALNS) enzyme. It is used in pediatric and adult patients with confirmed diagnosis of Morquio A syndrome.
Vimizim is a recombinant human enzyme that replaces the deficient GALNS enzyme in patients with MPS IVA. The exogenous enzyme helps catalyze the breakdown of the glycosaminoglycans keratan sulfate and chondroitin-6-sulfate, reducing their accumulation in tissues, which is the underlying cause of the disease symptoms and progression.
Vimizim carries a risk of serious allergic reactions including anaphylaxis during infusion. Spinal cord compression due to cervical spine involvement may occur and requires monitoring. Sleep apnea is common in MPS IVA patients and should be evaluated before and during treatment. Regular assessments of endurance, pulmonary function, and growth are recommended. The medication is administered as an intravenous infusion, typically every week.
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 August 26, 2026. · Read our editorial policy