Listen
VIMIZIM (elosulfase alfa), recombinant enzyme
RARE DISEASE - New medicinal product
Opinions on drugs -
Posted on
Jan 20 2015
Reason for request
Inclusion
Moderate clinical added value in mucopolysaccharidosis, type IV A (Morquio A Syndrome)
- VIMIZIM has a Marketing Authorisation in the treatment of mucopolysaccharidosis, type IV A (Morquio A syndrome) regardless of the age of the patients.
- This is a recombinant form of N-acetylgalactosamine-6-sulfatase (rhGALNS). Deficiency of this enzyme is the cause of Morquio A syndrome. VIMIZIM is the only enzyme replacement therapy indicated in this disorder.
- Short-term studies have shown an improvement in the 6-minute walk test. To date there are no efficacy data on the clinical manifestations of the disease and its long-term prognosis.
Clinical Benefit
| Substantial |
the actual benefit of VIMIZIM is substantial in the Marketing Authorisation indication. |
Clinical Added Value
| moderate |
Considering the severity of the disease, the only available palliative treatment alternatives and the modest efficacy of elosulfase alfa on the walking distance, as demonstrated in the short term, the Committee considers that VIMIZIM provides a moderate improvement in actual benefit (level III) to patients with Morquio A syndrome (mucopolysaccharidosis type IVA). |
