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STRENSIQ (asfotase alfa), enzyme replacement therapy

RARE DISEASE - New medicinal product
Opinions on drugs - Posted on Jun 09 2016

Reason for request

Inclusion

Substantial clinical added value in the treatment of hypophosphatasia 

  • 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.

 


Actual benefit

Important

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Improvement in actual benefit

II (important)

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Therapeutic use

-

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