On 8 November 2012, orphan designation (EU/3/12/1066) was granted by the European Commission to Pfizer Limited, United Kingdom, for tafamidis for the treatment of senile systemic amyloidosis.
The sponsorship was transferred to Pfizer Europe MA EEIG, Belgium, in July 2018.
Tafamidis (for treatment of senile systemic amyloidosis) has been authorised in the EU as Vyndaqel since 17 February 2020.
Treatment of senile systemic amyloidosis
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
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: