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.

Key facts

Active substance
Recombinant human lysosomal acid lipase
Medicine name
Disease / condition
Treatment of lysosomal acid lipase deficiency
Date of first decision
EU designation number

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.

Sponsor's contact details

Alexion Europe S.A.S.
103-105 Rue Anatole France
92300 Levallois Perret

Patients' organisations

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.

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