On 8 June 2004 orphan designation (EU/3/04/199) was granted by the European Commission to Dr. Gertrud Thormann, France, for tetrahydrobiopterin for the treatment of hyperphenylalaninemia.
The sponsorship was transferred first to BioMarin Europe Ltd, Ireland, in March 2005, then to Serono Europe Limited, United Kingdom, in August 2006, then to Merck KGaA, Germany, in September 2007, then to Merck Serono Europe Limited, United Kingdom, in March 2011 and subsequently to BioMarin International Limited, Ireland, in December 2015.
Tetrahydrobiopterin has been authorised in the EU as Kuvan since 2 December 2008.
|Disease / condition||
Treatment of hyperphenylalaninaemia
|Date of first decision||
|EU designation number||
Sponsor's contact details
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.