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 amyloidosis either in patients with Transthyretin (TTR) 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), between cells in your heart (known as transthyretin amyloid cardiomyopathy or ATTR-CM), and in other places in your body, 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:
PP-VYN-GBR-0884 / October 2021