EU/3/06/401

About

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.

Key facts

Active substance
N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate
Medicine name
Vyndaqel
Disease / condition
Treatment of familial amyloid polyneuropathy
Date of decision
28/08/2006
Outcome
Positive
Orphan decision number
EU/3/06/401

Review of designation

During its meeting of 6–8 September 2011, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/06/401 for Vyndaqel (N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate) as an orphan medicinal product for the treatment of familial amyloid polyneuropathy. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other satisfactory methods of treatment. As other satisfactory methods of treatment for patients with this condition exist in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designation of the medicine be maintained1.


1The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.

Sponsor's contact details

Pfizer Europe MA EEIG
Boulevard de la Plaine 17
1050 Bruxelles
Belgium
Tel. +32 2554 6586
E-mail: orphan_enquiries@pfizer.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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