Tafamidis (Vyndaqel)

A Medicine for Transthyretin Amyloidosis

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Transthyretin amyloidosis is a rare hereditary disease in which a fibrous substance called amyloid is deposited in tissues and organs around the body, including the nerves. These deposits interfere with the normal nerve function and can result in a condition called polyneuropathy, characterised by a sense of weakness, numbness, and pain in the hands and feet. Tafamidis, also known by the brand name Vyndaqel, is used to slow down nerve damage in patients with early-stage polyneuropathy.

Tafamidis (Vyndaqel): a medicine to slow down nerve damage in patients with early-stage polyneuropathy

Tafamidis (Vyndaqel) is used to treat a rare disease known as transthyretin familial amyloid polyneuropathy (TTR-FAP). It has been approved in many countries, including Brazil, Israel, South Korea, and those in the European Union. In some other countries, e.g. Australia, it has orphan designation*, and in the United States the Food and Drug Administration (FDA) granted it fast track designation* for the treatment of transthyretin cardiomyopathy (TTR-CM)–in which the disease affects the heart muscle.

* With the orphan drug and the fast track designations, the evaluation and development of this medicine can be done more quickly, in the hope that people with these serious and rare conditions will be able to benefit from new treatment options that would otherwise take longer to become available, if at all.

What is tafamidis (Vyndaqel)?

Tafamidis is the active ingredient in Vyndaqel which comes in capsule form. It is indicated for patients affected by transthyretin amyloidosis who also suffer from polyneuropathy. Polyneuropathies, or peripheral neuropathies, result from damage to the peripheral nerves (nerves that go from the spinal cord to the arms, hands, legs, and feet), which often leads to a sense of weakness, numbness, and pain in the extremities.

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How effective is tafamidis (Vyndaqel)?

The efficacy of tafamidis (Vyndaqel) has been tested in clinical trials. The main study lasted 18 months and included 128 patients carrying a specific genetic mutation (V30M). These participants were in stage 1 of the disease, meaning they were able to walk without support most of the time. Those taking tafamidis (Vyndaqel) showed results superior to the placebo, with less nerve damage and a higher quality of life.

Final analysis after 18 months

Neuropathy Impairment Score of the Lower Limb (NIS-LL) Percent of patients that showed improvement. NIS-LL is a score based on a physician's assessment of the
neurologic exam of the lower limbs.

Final analysis after 18 months

Change in Total Quality of Life Score (TQOL). TQOL is a total quality of life score based on patient-reported data. A change close to zero indicates that the general quality of life (QOL) was maintained, while a larger change indicates a worsening of the patients' QOL.

For more details refer to the prescribing information at the bottom of the page.

Which side effects can I expect with tafamidis (Vyndaqel)?

The most common side effects listed in the prescribing information include diarrhoea, urinary tract infection, stomach ache or abdominal pain, and vaginal infection in women.

Please note this is not intended to be a comprehensive guide. Consult your treating doctor and the prescribing information at the bottom of the page for full details of side effects.

How is tafamidis (Vyndaqel) taken?

The capsules should be taken orally, once daily. They should be swallowed whole, not cut or crushed, and can be taken with or without food.

Please note this is not intended to be a treatment plan. For a personalised treatment plan consult your doctor. For more details, you can also reference the full prescribing information at the bottom of the page.

How does tafamidis (Vyndaqel) work?

TTR-FAP is characterised by the presence of a defective blood protein called transthyretin (TTR).
This defective protein breaks easily which causes the accumulation of amyloids (a fibrous substance) within tissues, nerves and organs, and prevents their normal function.
The damage to the nerves results in a sense of weakness, numbness, and pain in the extremities.
Tafamidis (Vyndaqel) stabilises the defective protein. In this way, it reduces the symptoms and slows down the progression of the neurological disorder.

For more details about the way tafamidis (Vyndaqel) works, consult your doctor or see the prescribing information at the bottom of the page. This is not intended to be a comprehensive scientific explanation.

How can I get tafamidis (Vyndaqel)?

It may be the case that tafamidis (Vyndaqel) is not yet approved or available in your country, which may mean that you will experience delays in accessing this medicine for yourself, your patient or a relative or friend. We’re here to help.

With a prescription from the patient’s treating doctor TheSocialMedwork can support you in obtaining tafamidis (Vyndaqel) from outside your country. Our team of pharmacists and regulatory experts delivers elsewhere approved medicines around the world every day. They are ready to assist with sourcing and delivery, and will guide you through every step of the process. We always advise patients to ask their health insurance provider about reimbursement of the medicine.

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If you or one of your relatives or friends are a patient with transthyretin amyloidosis, you might want to ask questions about the medicine for this disease. If you’d like to ask questions about how to get tafamidis (Vyndaqel), or about our service in general, our team of qualified pharmacists and experts are ready to offer support and assist with queries. Our team speaks 17 different languages and is ready to help.