Myozyme
What Myozyme is used for
Myozyme is indicated for long-term enzyme-replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid-alpha-glucosidase deficiency). In patients with late-onset Pompe disease the evidence of efficacy is limited.
Approved use, as published by the European Medicines Agency.
Key facts
- Brand
- Myozyme
- Active substance
- alglucosidase alfa
- Disease area
- Glycogen Storage Disease Type II
- Medicine group
- Other alimentary tract and metabolism products
- ATC code
- A16AB07
- Marketing authorisation holder
- Sanofi B.V.
- Authorised in the EU since
- 2006
Official information
In the USA
Same active substance: Lumizyme
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 Myozyme used for?
Myozyme is indicated for long-term enzyme-replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid-alpha-glucosidase deficiency). In patients with late-onset Pompe disease the evidence of efficacy is limited.
What is the active substance in Myozyme?
Myozyme contains alglucosidase alfa.
Who makes Myozyme?
The EU marketing authorisation holder is Sanofi B.V..
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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