What is Vyndaqel (tafamidis)?
Vyndaqel (tafamidis) is a medicine indicated for the treatment of transthyretin amyloidosis in patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment1. Vyndaqel (tafamidis) is the first medication approved to stabilise the protein transthyretin (TTR) from misfolding and forming amyloids3.
Who is Vyndaqel (tafamidis) for?
Vyndaqel (tafamidis) is indicated for adults with stage 1 transthyretin familial amyloid polyneuropathy (TTR-FAP). Polyneuropathies, or peripheral neuropathies, result from damage to the peripheral nerves (nerves that go from the spinal cord to the arms, hands, legs, and feet2), which often result in a sense of weakness, numbness and pain in the extremities1,4.
How does Vyndaqel (tafamidis) work?
Transthyretin amyloidosis is a rare disease characterised by the presence of a defective blood protein called transthyretin (TTR). This defective protein breaks easily, which results in the deposition of a fibrous substance (amyloid fibrils) within the body5. The accumulation of amyloid fibrils causes damage to body structures, like tissues and organs, and prevents their normal function5. When the nerves are affected the disease is called transthyretin familial amyloid polyneuropathy (TTR-FAP)10.
The active substance in Vyndaqel, tafamidis, is a transthyretin stabiliser. It attaches to transthyretin, the defective protein, and makes it stable. In this way, it prevents the protein from breaking up, and it may thus stop the formation of amyloid fibrils and slow down the progression of the neurological disorder5.
Where has Vyndaqel (tafamidis) been approved?
Vyndaqel (tafamidis) has been approved for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP) in the European Union, and also in the following countries:
Japan
Brazil
Argentina
Russia
In 2017, the Food and Drug Administration (FDA), United States, granted it fast track designation for the treatment of transthyretin cardiomyopathy (TTR-CM), in which the amyloids build up in the heart muscle10. Other countries, such as Australia, have granted it orphan drug designation. Orphan drug and fast track designations are processes designed to facilitate and expedite the development of a medicine to treat serious conditions and fill an unmet medical need7,8.
Please note that this medicine may have also been approved in other regions than the ones we’ve listed. If you have a question about its approval in a specific country feel free to contact our support team.
How is Vyndaqel (tafamidis) taken?
The standard dosage is:
20 mg orally once daily1.
Complete information about Vyndaqel (tafamidis) dosage and administration can be found in the official prescribing information listed in our resources section1.
Note: Please consult with your treating doctor for personalised dosing.
Are there any known side effects or adverse reactions of Vyndaqel (tafamidis)?
Possible Side Effects
The most common adverse reactions listed in the prescribing information include1:
diarrhea
urinary tract infection (symptoms may include: pain or a burning sensation when you urinate or a frequent need to urinate)
vaginal infection in women
stomach ache or abdominal pain.
Use in specific populations
It is advised to avoid pregnancy and breastfeeding during and for one month after treatment with Vyndaqel (tafamidis)1.
For a comprehensive list of side effects and adverse reactions please refer to the official prescribing information1.
