EU/3/00/002: Orphan designation for the treatment of Fabry disease
Table of contents
Please note that this product was withdrawn from the Community register of designated orphan medicinal products in August 2011 at the end of the period of market exclusivity.
On 8 August 2000, orphan designation (EU/3/00/002) was granted by the European Commission to TKT Europe-5S AB, Sweden, for alpha-galactosidase A for the treatment of Fabry disease.
The sponsor applied for a change of name to Shire Human Genetic Therapies AB in September 2010.
α-Galactosidase A has been authorised in the EU as Replagal since 3 August 2001.
Treatment of Fabry disease
|Orphan designation status||
|EU designation number||
|Date of designation||
Shire Human Genetic Therapies AB
Tel. +46 8544 96400
Fax +46 8544 96429
Review of designation
Please note that this product (marketed as Replagal) was withdrawn from the Community register of designated orphan medicinal products in August 2011 at the end of the period of market exclusivity.
Documents related to this orphan designation evaluation
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: