EU/3/00/003: Orphan designation for the treatment of Fabry disease

Alpha-galactosidase A

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Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in August 2011 at the end of the period of market exclusivity.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/3/00/003) was granted by the European Commission to Genzyme BV, the Netherlands, for alpha-galactosidase A for the treatment of Fabry disease.

Genzyme BV changed name to Genzyme Europe BV in 2002.

α-Galactosidase A has been authorised in the EU as Fabrazyme since 3 August 2001.

Key facts

Active substance
Alpha-galactosidase A
Medicine name
Intended use
Treatment of Fabry disease
Orphan designation status
EU designation number
Date of designation
Genzyme Europe BV
Gooimeer 10
1411 DD Naarden
The Netherlands
Tel. +31 35 699 1200
Fax +31 35 694 3214

Documents related to this orphan designation evaluation

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.

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:

How useful was this page?

Add your rating
1 rating
1 rating