This is a summary of the European public assessment report (EPAR) for Myozyme. It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the medicine to reach its opinion in favour of granting a marketing authorisation and its recommendations on the conditions of use for Myozyme.
Myozyme : EPAR - Summary for the public (PDF/76.65 KB)
First published: 15/01/2009
Last updated: 23/01/2014
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|International non-proprietary name (INN) or common name||
|Therapeutic area (MeSH)||
Glycogen Storage Disease Type II
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This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.
Genzyme Europe B.V.
|Date of issue of marketing authorisation valid throughout the European Union||
08/09/2017 Myozyme - EMEA/H/C/000636 - N/0065
- Annex I - Summary of product characteristics
- Annex IIA - Manufacturing-authorisation holder responsible for batch release
- Annex IIB - Conditions of the marketing authorisation
- Annex IIIA - Labelling
- Annex IIIB - Package leaflet
Please note that the size of the above document can exceed 50 pages.
You are therefore advised to be selective about which sections or pages you wish to print.
Other alimentary tract and metabolism products
Myozyme is indicated for long-term enzyme-replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid-α-glucosidase deficiency).
In patients with late-onset Pompe disease the evidence of efficacy is limited.