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.
The sponsor’s address was updated in November 2019.
Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein
Treatment of hypophosphatasia
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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.
Documents related to this orphan designation evaluation
EU/3/08/594: Public summary of positive opinion for orphan designation of recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein for the treatment of hypophosphatasia (PDF/127.4 KB)
First published: 24/04/2009
Last updated: 26/03/2015
EMEA/COMP/547061/2008 Rev. 4
Recommendation for maintenance of orphan designation at the time of marketing authorisation: Strensiq (asfotase alfa) for the treatment of hypophosphatasia (PDF/77.58 KB)
First published: 28/09/2015
Last updated: 28/09/2015
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: