This is a summary of the European public assessment report (EPAR) for Vyndaqel. It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the medicine to reach its opinion in favour of granting a marketing authorisation and its recommendations on the conditions of use for Vyndaqel.
This product originally had an orphan designation for familial amyloid polyneuropathy, granted on 28 August 2006. This designation was withdrawn from the Community register of orphan medicinal products in November 2021 at the end of the 10-year period of market exclusivity.
Vyndaqel : EPAR - Summary for the public (PDF/78.69 KB)
First published: 18/11/2011
Last updated: 02/08/2016
Vyndaqel : EPAR - Risk-management-plan summary (PDF/99.91 KB)
First published: 21/02/2020
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This medicine was authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.
Pfizer Europe MA EEIG
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Boulevard de la Plaine 17
06/12/2021 Vyndaqel - EMEA/H/C/002294 - IAIN/0077/G
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.
Other nervous system drugs
Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage-1 symptomatic polyneuropathy to delay peripheral neurologic impairment.