This page is intended as reference information for the general public.
What is Vyndaqel and what is it used for?
Vyndaqel contains the active substance tafamidis. It is available as a capsule.
Vyndaqel is a medicine which treats a rare disease called transthyretin (TTR) amyloidosis either in patients with transthyretin amyloid polyneuropathy, also known as TTR familial amyloid polyneuropathy (TTR-FAP) or in patients with wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM). TTR amyloidosis is caused by a protein called TTR that does not work properly. TTR is an important protein that carries other substances, such as hormones, through the body.
In patients with this disease, TTR breaks up and may form fibres called amyloid. Amyloid can build up around your nerves (known as transthyretin amyloid polyneuropathy or ATTR-PN) or between the cells in your heart (known as transthyretin amyloid cardiomyopathy or ATTR-CM), preventing them from working normally. Eventually, the amyloid causes the symptoms of ATTR-PN or ATTR-CM.
Vyndaqel can prevent TTR from breaking up and forming amyloid deposits. This medicine is used to treat adult patients with this disease whose nerves have been affected (people with symptomatic polyneuropathy) and is also used to treat adult patients whose heart has been affected (people with symptomatic cardiomyopathy).
The medicine can only be obtained with a prescription.
The European Medicines Agency (EMA) is an agency of the European Union (EU). The Agency is responsible for the scientific evaluation, supervision and safety monitoring of medicines developed by pharmaceutical companies for use in the EU. See EMA guidance on Vyndaqel.
Medicines in Northern Ireland fall under the remit of the European Medicines Agency (EMA) whilst medicines in Great Britain (England, Scotland and Wales) fall under the remit of the Medicines and Healthcare products Regulatory Agency (MHRA) since the UK left the European Union. These agencies are responsible for the scientific evaluation, supervision and safety monitoring of medicines developed by pharmaceutical companies.
If you would like to see the Summary of Product Characteristics (SPC) or Patient Information Leaflet (PIL) for Vyndaqel please follow these links:
▼ This medicine is subject to additional monitoring.
Pfizer is unable to provide advice on personal medical matters. Please consult your doctor or other relevant health professional for specific, health-related advice and support.
If you get any side effects when taking this or any other medicine, talk to your doctor or other relevant health professional e.g. pharmacist, nurse. This includes any possible side effects not listed in the packaging leaflet. You can also report side effects directly via the Yellow Card Scheme at https://yellowcard.mhra.gov.uk or search the Google Play or Apple App Store for MHRA Yellow Card. By reporting side effects, you can help provide more information on the safety of this medicine.