Vyndaqel | European Medicines Agency (EMA)

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Overview

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 This designation was withdrawn from the Community register of orphan medicinal products in November 2021 at the end of the 10-year period of market exclusivity.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 is a medicine that contains the active substance tafamidis. It is available as capsules (20 mg).

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.

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.

In patients with transthyretin amyloidosis, a blood protein called transthyretin is defective and breaks easily. The broken protein forms a fibrous substance called amyloid that is deposited in tissues and organs around the body, including around nerves, where it interferes with their normal functions.

The active substance in Vyndaqel, tafamidis, is a transthyretin stabilizer. 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.

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.

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.

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 and restrictions with Vyndaqel, see the package leaflet.

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.

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.

The company that markets Vyndaqel 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.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Vyndaqel have also been included in the summary of product characteristics and the package leaflet.

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.

English (EN)
(78.69 KB – PDF)

First published: 18/11/2011Last updated: 02/08/2016

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English (EN)
(1.16 MB – PDF)

First published: 21/02/2020Last updated: 22/11/2024

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Product information

English (EN)
(680.08 KB – PDF)

First published: 18/11/2011Last updated: 18/02/2025

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Latest procedure affecting product information:
N/0000245541

17/02/2025

This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.

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).

English (EN)
(31.93 KB – PDF)

First published: 18/11/2011Last updated: 10/12/2021

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English (EN)
(95.21 KB – PDF)

First published: 18/11/2011Last updated: 18/11/2011

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English (EN)
(61.67 KB – PDF)

First published: 08/08/2013Last updated: 08/08/2013

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Product details

Name of medicine: Vyndaqel
Active substance: tafamidis
International non-proprietary name (INN) or common name: tafamidis
Therapeutic area (MeSH): Amyloidosis
Anatomical therapeutic chemical (ATC) code: N07XX08

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.

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Vyndaqel | European Medicines Agency (EMA)

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Vyndaqel | European Medicines Agency (EMA)

Overview

What is Vyndaqel?

What is Vyndaqel used for?

How is Vyndaqel used?

How does Vyndaqel work?

How has Vyndaqel been studied?

What benefit has Vyndaqel shown during the studies?

What is the risk associated with Vyndaqel?

Why has Vyndaqel been approved?

What information is still awaited for Vyndaqel?

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

Other information about Vyndaqel

Product information

Product details

Pharmacotherapeutic group

Therapeutic indication

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