-
Xenpozyme (olipudase alfa) is an enzyme replacement therapy designed to treat acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disorder. It is specifically indicated for non-central nervous system manifestations of ASMD in both pediatric and adult patients.
Xenpozyme is an enzyme replacement therapy for ASMD that helps reduce sphingomyelin accumulation in organs and tissues.
Key facts at a glance
- RxNorm
- 2610412
- Generic
- Xenpozyme
- 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.
Xenpozyme is indicated for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease types A and B. It is used in adult and pediatric patients.
Xenpozyme is a recombinant human acid sphingomyelinase (rhASM) enzyme. It works as an exogenous enzyme replacement that helps break down accumulated sphingomyelin in cells, reducing the buildup in various organs including the liver, spleen, and lungs.
Xenpozyme is administered via intravenous infusion. As a biologic medication, it requires appropriate monitoring during treatment. Common side effects may include infusion-associated reactions. Patients should be monitored for hepatic parameters as adjustments may be needed. This medication should only be administered by healthcare professionals experienced in managing allergic reactions.
Official drug label
WellAlly may earn a referral fee from linked resources at no extra cost to you. Editorial content is never influenced by commercial relationships.
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 27, 2026. · Read our editorial policy