Xenpozyme
What Xenpozyme is used for
Xenpozyme is indicated as an enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD) in paediatric and adult patients with type A/B or type B.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- PRIME: a priority medicine that the EMA supported early because it may meet an unmet need.
Key facts
- Brand
- Xenpozyme
- Active substance
- olipudase alfa
- Disease area
- Acid sphingomyelinase deficiency (ASMD) type A/B or type B
- Medicine group
- Other alimentary tract and metabolism products
- ATC code
- A16AB25
- Marketing authorisation holder
- Sanofi B.V.
- Authorised in the EU since
- 2022
Official information
In the USA
Same active substance: Xenpozyme
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Xenpozyme used for?
Xenpozyme is indicated as an enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD) in paediatric and adult patients with type A/B or type B.
What is the active substance in Xenpozyme?
Xenpozyme contains olipudase alfa.
Who makes Xenpozyme?
The EU marketing authorisation holder is Sanofi B.V..
Is Xenpozyme an orphan medicine?
Yes. Xenpozyme is designated an orphan medicine in the EU, for a rare disease (Acid sphingomyelinase deficiency (ASMD) type A/B or type B).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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