STRENSIQ (asfotase alfa), enzyme replacement therapy
RARE DISEASE - New medicinal product
Opinions on drugs - Posted on Jun 09 2016
Reason for request
- STRENSIQ has marketing authorisation in long-term enzyme replacement therapy in patients with hypophosphatasia (HPP) whose first signs appeared before age 18 years to treat the bone manifestations of the disease.
- In perinatal/infantile forms of the disease, its efficacy was observed in two non-comparative studies and one comparative study versus historical controls in terms of radiological improvement of the wrist and knee joints (n=75 children). Exploratory data suggest a favourable outcome in respiratory function in patients treated with STRENSIQ.
- In juvenile forms of the disease, its efficacy has been observed on a smaller study group (20 children and adults) in a comparative study versus historical controls and a study with an untreated control group in terms of radiological improvement of wrist and knee joints, growth, disability and reduction in plasma PLP.
- Its prescription should be restricted to reference centres for rare diseases of calcium and phosphorus metabolism and constitutional bone diseases (OSCAR network) and only for perinatal/infantile forms (first clinical manifestations ≤ 6 months) and symptomatic juvenile forms (early clinical manifestations between 6 months and 18 years) showing criteria for progression.
Improvement in actual benefit
Évaluation des médicaments
HAS opinions and decisions
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