On 8 March 2004, orphan designation (EU/3/04/194) was granted by the European Commission to Mr Aart Brouwer, the Netherlands, for adeno-associated viral vector expressing lipoprotein lipase for the treatment of lipoprotein-lipase deficiency.

The sponsorship was transferred to Amsterdam Molecular Therapeutics, the Netherlands, in December 2006.

In July 2012, Amsterdam Molecular Therapeutics changed name to uniQure biopharma B.V.

Adeno-associated viral vector expressing lipoprotein lipase has been authorised in the EU as Glybera since 25 October 2012.

Key facts

Active substance
Adeno-associated viral vector expressing lipoprotein lipase
Medicine name
Disease / condition
Treatment of lipoprotein-lipase deficiency
Date of first decision
EU designation number

Review of designation

During its meeting of 4-5 September 2012, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/04/194 for Glybera (alipogene tiparvovec, previously known as adeno-associated viral vector expressing lipoprotein lipase) as an orphan medicinal product for the treatment of lipoprotein-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 satisfactory 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 European Union (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

uniQure biopharma B.V.
Paasheuvelweg 25
1105 BP
The Netherlands
Tel: +31 (0)20 240 6154

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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