EU/3/12/1066: Orphan designation for the treatment of senile systemic amyloidosis



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. 

Key facts

Active substance
Intended use
Treatment of senile systemic amyloidosis
Orphan designation status
EU designation number
Date of designation

Pfizer Europe MA EEIG
Boulevard de la Plaine 17
1050 Bruxelles
Brussels-Capital Region

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation EU/3/12/1066 for Vydaqel (tafamidis) at the time of marketing authorisation and confirmed that the orphan designation should be maintained.

More information is available in the orphan medicine assessment report

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:

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