This medicine is now known as alipogene tiparvovec.
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 was authorised in the EU as Glybera on 25 October 2012.
The marketing authorisation was withdrawn on 29 October 2017. Please note that this product was withdrawn from the Community Register of designated orphan medicinal products by the European Commission in October 2017 at the time of withdrawal of the marketing authorisation.
Adeno-associated viral vector expressing lipoprotein lipase (alipogene tiparvovec)
Treatment of lipoprotein-lipase deficiency
|Orphan designation status||
|EU designation number||
|Date of designation||
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.
Documents related to this orphan designation evaluation
Recommendation for maintenance of orphan designation at the time of marketing authorisation: Glybera (alipogene tiparvovec) for the treatment of lipoprotein-lipase deficiency (PDF/113.59 KB)
First published: 03/12/2012
Last updated: 31/05/2021
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: