EU/3/08/594

About

On 3 December 2008, orphan designation (EU/3/08/594) was granted by the European Commission to Europa Rx Limited, United Kingdom, for recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein for the treatment of hypophosphatasia.

The sponsorship was transferred to Dr Ulrich Granzer, Germany, in September 2010 and Alexion Europe SAS, France, in September 2012.

Update: recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein, asfotase alfa (Strensiq) has been authorised in the EU since 28 August 2015 for long-term enzyme replacement therapy in patients with paediatric-onset hypophosphatasia to treat the bone manifestations of the disease.

Key facts

Active substance
Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein
Disease / condition
Treatment of hypophosphatasia
Date of first decision
04/12/2008
Outcome
Positive
EU designation number
EU/3/08/594

Review of designation

During its meeting of 14 to 16 July 2015, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/08/594 for Strensiq (asfotase alfa1) as an orphan medicinal product for the treatment of hypophosphatasia. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. The COMP recommended that the orphan designation of the medicine be maintained2.


1Previously known as 'recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein'.

2The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.

Sponsor's contact details

Alexion Europe S.A.S.
1-15, avenue Edouard Belin
92500 Rueil-Malmaison
France
Tel. +33 1 47 10 06 15
E-mail: medicalinformation.europe@alxn.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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