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MYOZYME (alglucosidase alpha), enzyme replacement therapy
METABOLISM - Update
Opinions on drugs -
Posted on
Oct 18 2017
Reason for request
Re-assessment of the actual benefit and the improvement in actual benefit
Moderate clinical added value in the therapeutic strategy of infantile-onset forms of Pompe disease
Minor clinical added value in the therapeutic strategy of late-onset forms of Pompe disease
- MYOZYME has marketing authorisation in long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid alpha-glucosidase deficiency).
- In the infantile-onset forms of the disease, the new data available confirm the benefit on the overall survival of patients. On the other hand, no data document their quality of life (very deteriorated), nor the resulting neurological damage which causes major disability and a significant mortality.
- In the late-onset forms of the disease, the data available only on functional criteria are still very heterogeneous according to patient characteristics. They do not allow the assessment of MYOZYME in terms of disability progression or overall survival.
Clinical Benefit
| Substantial |
- |
| Low |
Clinical Added Value
| moderate |
- |
| minor |
Therapeutic use
|
- |
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