Olipudase Alfa
Olipudase Alfa is an enzyme replacement therapy designed to treat Acid Sphingomyelinase Deficiency (ASMD), a rare lysosomal storage disease. It works by replacing the deficient acid sphingomyelinase enzyme, helping to break down accumulated sphingomyelin in cells. This medication is used primarily in pediatric and adult patients with chronic visceral ASMD.
Olipudase Alfa is an enzyme replacement therapy for ASMD that replaces deficient acid sphingomyelinase to reduce sphingomyelin accumulation in cells.
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
- A16AB25
- RxNorm
- 2610407
- Generic
- Olipudase Alfa
- Class
- atc-a16
- Brands
- Xenpozyme
- 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.
Treatment of Acid Sphingomyelinase Deficiency (ASMD), including chronic visceral manifestations of Niemann-Pick disease type A and type B. It is indicated for pediatric and adult patients with confirmed ASMD diagnosis.
Olipudase Alfa is a recombinant human acid sphingomyelinase (rhASM) that supplements or replaces the deficient endogenous enzyme. It catalyzes the breakdown of sphingomyelin into ceramide and phosphocholine, thereby reducing the accumulation of sphingomyelin in various tissues and organs characteristic of ASMD.
As a parenteral enzyme replacement therapy, it is administered via intravenous infusion. Patients should be monitored for potential infusion-related reactions and immune responses. Treatment should be initiated and managed by specialists experienced in treating lysosomal storage diseases.
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