Vyndaqel

tafamidis

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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.

What is Vyndaqel?

Vyndaqel is a medicine that contains the active substance tafamidis. It is available as capsules (20 mg).

What is Vyndaqel used for?

Vyndaqel is used to delay nerve damage caused by transthyretin amyloidosis, a hereditary disease in which fibres called amyloid build up in tissues around the body including around the nerves. Vyndaqel is used in adult patients with the early stage of nerve disease (stage 1).

Because the number of patients with transthyretin amyloidosis is low, the disease is considered ‘rare’, and Vyndaqel was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 28 August 2006.

The medicine can only be obtained with a prescription.

How is Vyndaqel used?

Treatment should be started and supervised by a doctor knowledgeable in the management of patients with this disease.

The recommended dose is one capsule once a day, swallowed whole. Vyndaqel is used alongside standard care for patients with transthyretin amyloidosis. Doctors will need to monitor patients and assess their need for other treatments including liver transplantation. Patients undergoing liver transplantation will have to stop taking Vyndaqel.

How does Vyndaqel work?

In patients with transthyretin amyloidosis, the amyloid fibres that surround and damage the nerves are formed of a blood protein called transthyretin, which is defective in these patients. As a result it breaks easily and is deposited in tissues and organs around the body where it interferes with the normal functions of the organs.

The active substance in Vyndaqel, tafamidis, is a transthyretin stabiliser. It attaches to transthyretin, which prevents the protein from breaking up, thereby stopping the formation of amyloid and slowing down the progression of the nerve disease.

How has Vyndaqel been studied?

Vyndaqel was compared with placebo (a dummy treatment) in 128 patients with transthyretin amyloidosis. The main measure of effectiveness was based on an assessment of the patients’ nerve damage and quality of life after 18 months of treatment.

What benefit has Vyndaqel shown during the studies?

Vyndaqel was more effective than placebo at treating patients with transthyretin amyloidosis. In 45% of patients taking Vyndaqel, nerve function either improved or stabilised, compared with 30% of patients taking placebo. The results did not include patients with more severe disease who left the study early because they needed to undergo a liver transplant.

Patients taking Vyndaqel also had better quality-of-life scores, but the difference with placebo was not considered significant.

What is the risk associated with Vyndaqel?

The most common side effects with Vyndaqel (seen in more than 1 patient in 10) are urinary tract infection (infection of the structures that carry urine), vaginal infection, upper abdominal pain (stomach ache) and diarrhoea. For the full list of all side effects reported with Vyndaqel, see the package leaflet.

Vyndaqel must not be used in people who are hypersensitive (allergic) to tafamidis or to any of the other ingredients.

Why has Vyndaqel been approved?

The CHMP noted that, apart from in patients who left the study because they needed a liver transplant, Vyndaqel was more effective than placebo in delaying nerve damage in patients with transthyretin amyloidosis. The medicine presented no major safety concerns and the risks were considered to be manageable. The CHMP therefore concluded that the benefits of Vyndaqel are greater than its risks and recommended that it be granted marketing authorisation.

Vyndaqel has been authorised under ‘exceptional circumstances’. This means that because transthyretin amyloidosis is rare, for scientific reasons, it has not been possible to obtain complete information about Vyndaqel. Every year, the European Medicines Agency will review any new information that may become available and this summary will be updated as necessary.

What information is still awaited for Vyndaqel?

The company that makes Vyndaqel will carry out a study on the effects of the medicine in a subgroup of patients with certain genetic mutations. The company will also provide long-term post-marketing safety data.

What measures are being taken to ensure the safe use of Vyndaqel?

The company will provide educational material to all doctors expected to prescribe Vyndaqel. The material will include a leaflet containing information on the risks of the medicine including the risks to the unborn baby and on how to contribute to a post marketing study and a surveillance programme for pregnant women.

Other information about Vyndaqel

The European Commission granted a marketing authorisation valid throughout the European Union for Vyndaqel on 16 November 2011.

For more information about treatment with Vyndaqel, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

Name Language First published Last updated
Vyndaqel : EPAR - Summary for the public BG = bălgarski 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public ES = español 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public CS = čeština 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public DA = dansk 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public DE = Deutsch 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public ET = eesti keel 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public EL = elliniká 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public EN = English 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public FR = français 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public IT = italiano 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public LV = latviešu valoda 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public LT = lietuvių kalba 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public HU = magyar 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public MT = Malti 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public NL = Nederlands 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public PL = polski 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public PT = português 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public RO = română 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public SK = slovenčina 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public SL = slovenščina 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public FI = suomi 18/11/2011 08/08/2013
Vyndaqel : EPAR - Summary for the public SV = svenska 18/11/2011 08/08/2013

This EPAR was last updated on 30/09/2014 .

Authorisation details

Product details

Product details for Vyndaqel
NameVyndaqel
Agency product numberEMEA/H/C/002294
Active substance

tafamidis

International non-proprietary name (INN) or common name

tafamidis

Therapeutic area Amyloidosis
Anatomical therapeutic chemical (ATC) code N07XX08
Additional monitoring

This medicine is under additional monitoring. This means that it is being monitored even more intensively than other medicines. For more information, see medicines under additional monitoring.

Treatment of rare diseases

This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.

Exceptional Circumstances

There were "exceptional circumstances" concerning the approval of this medicine. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicine for which authorisation is being sought, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.

Publication details

Publication details for Vyndaqel
Marketing-authorisation holder

Pfizer Ltd.

Revision5
Date of issue of marketing authorisation valid throughout the European Union16/11/2011

Contact address:

Pfizer Ltd.
Ramsgate Road
Sandwich
Kent
CT13 9NJ
United Kingdom

Product information

Product information

01/09/2014  Vyndaqel -EMEA/H/C/002294 -IAIN/0019

Name Language First published Last updated
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014
Vyndaqel : EPAR - Product Information SV = svenska 18/11/2011 30/09/2014

Contents

  • Annex I - Summary of product characteristics
  • Annex IIA - Manufacturing-authorisation holder responsible for batch release
  • Annex IIB - Conditions of the marketing authorisation
  • Annex IIIA - Labelling
  • Annex IIIB - Package leaflet

Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.


Name Language First published Last updated
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  
Vyndaqel : EPAR - All Authorised presentations SV = svenska 18/11/2011  

Name Language First published Last updated
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  
Vyndaqel : EPAR - Conclusions on the granting of the marketing authorisation under exceptional circumstances - Annex IV SV = svenska 08/08/2013  

Pharmacotherapeutic group

Other nervous-system drugs

Therapeutic indication

Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage-1 symptomatic polyneuropathy to delay peripheral neurologic impairment.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated
Vyndaqel : EPAR - Procedural steps taken and scientific information after authorisation SV = svenska 22/04/2013 30/09/2014

Initial marketing-authorisation documents

Name Language First published Last updated
Vyndaqel : EPAR - Public assessment report SV = svenska 18/11/2011  
CHMP summary of positive opinion for Vyndaqel SV = svenska 22/07/2011  

Authorised

This medicine is approved for use in the European Union

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