EU/3/06/401: Orphan designation for the treatment of familial amyloid polyneuropathy

N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate (tafamidis)


On 28 August 2006, orphan designation (EU/3/06/401) was granted by the European Commission to ICON Clinical Research Limited, United Kingdom for N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl) ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate for the treatment of familial amyloid polyneuropathy.

The sponsorship was transferred to FoldRx Pharmaceuticals Limited, United Kingdom, in September 2009 and to Pfizer Limited, United Kingdom, in May 2012.

N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl) ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate has been authorised in the EU as Vyndaqel since 16 November 2011.

The sponsorship was transferred to Pfizer Europe MA EEIG, Belgium, in July 2018.

This medicine is now known as tafamidis. 

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in November 2021 at the end of the 10-year period of market exclusivity.

Key facts

Active substance
N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate (tafamidis)
Medicine name
Intended use
Treatment of familial amyloid polyneuropathy
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/3/06/401 for Vyndaqel (tafamidis) at the time of the change to the terms of the 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

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Vyndaqel (N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate) for treatment ... (PDF/113.47 KB)


    First published: 21/12/2011
    Last updated: 25/11/2021

  • 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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