On 17 December 2010, orphan designation (EU/3/10/827) was granted by the European Commission to HungaroTrial Ltd, Hungary, for recombinant human lysosomal acid lipase for the treatment of lysosomal acid lipase deficiency.
The sponsorship was transferred to Synageva BioPharma Ltd, United Kingdom, in August 2011.
The sponsorship was transferred to Alexion Europe SAS, France, in October 2015.
Update: recombinant human lysosomal acid lipase, sebelipase alfa (Kanuma) has been authorised in the EU since 28 August 2015 for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) deficiency.
The sponsor’s address was updated in November 2019.
Recombinant human lysosomal acid lipase
Treatment of lysosomal acid lipase deficiency
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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/3/10/827 for Kanuma (sebelipase alfa, previously known as recombinant human lysosomal acid lipase) as an orphan medicinal product for the treatment of lysosomal acid lipase deficiency. 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 maintained1.
1The 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
Recommendation for maintenance of orphan designation at the time of marketing authorisation: Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency (PDF/77.51 KB)
First published: 08/09/2015
Last updated: 08/09/2015
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: