On 23 March 2010, orphan designation (EU/3/10/726) was granted by the European Commission to Protalix B.V., the Netherlands, for taliglucerase alfa for the treatment of Gaucher disease.
The sponsorship was transferred to Pfizer Limited, United Kingdom, in September 2010 and subsequently to Pfizer Europe MA EEIG, Belgium, in November 2018.
Treatment of Gaucher disease
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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: