Vyndaqel (tafamidis)

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Vyndaqel (tafamidis) is a medication used for the treatment of transthyretin amyloidosis in adults with early-stage symptomatic polyneuropathy.

DISEASE INDICATIONS

Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP)

MANUFACTURER

Pfizer Limited

USAGE

Oral

MEDICINE APPROVED BY

  • European Medical Agency (EMA)

  • Food and Drug Administration (FDA)

  • Health Canada

  • Pharmaceuticals and Medical Devices Agency (PMDA)

  • Therapeutic Goods Administration (TGA)

    PATIENTS HELPED

    • Over 1.2 million patients reached

    • More than 11,000 patients helped

    • 100% successful deliveries to 88 countries

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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 impairment[1]. Vyndaqel (tafamidis) is the first medication approved to stabilise the protein transthyretin (TTR) from misfolding and forming amyloids[3].

It is available in capsule form each containing 20mg of tafamidis.

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 feet[2]), which often result in a sense of weakness, numbness and pain in the extremities[1,4].

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 body[5]. The accumulation of amyloid fibrils causes damage to body structures, like tissues and organs, and prevents their normal function[5]. 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 disorder[5].

Where has Vyndaqel (tafamidis) been approved?

Vyndaqel (tafamidis) has been approved for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP) by the:

  • EMA (EU), November 2011[1]
  • FDA (USA), May 2019[7]
  • Health Canada, January 2020[11]
  • PMDA (Japan), September 2013[12]
  • TGA (Australia), March 2020[13]

It is also approved in Brazil, Argentina and Russia for this indication. 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 need[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 for assistance.

Please be aware that any decision to use a prescription generic or brand name medicine should always be taken in consultation with a medical professional. The FDA has sent warning letters to drugmakers in India concerning the quality of their medicines.

How is Vyndaqel (tafamidis) taken?

The standard dosage is:

  • 20 mg orally once daily[1].

Complete information about Vyndaqel (tafamidis) dosage and administration can be found in the official prescribing information listed in our references section[1].

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 include[1]:

  • diarrhoea
  • 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 information[1].

References

  1. Summary of Product Characteristics [EMA]: Vyndaqel (tafamidis)[PDF],

Pfizer Limited, November 2011

  1. European public assessment report (EPAR) for Vyndaqel[PDF],

Pfizer Limited, November 2011

  1. Pfizer Press Release, Pfizer’s Vyndaqel (tafamidis) first therapy approved in the European Union for the rare and fatal neurodefenerative disease transthyretin familial amyloid polyneuropathy (TTR-FAP),

www.pfizer.com, October 2018

  1. Mayo Clinic. Peripheral neuropathy,

www.mayoclinic.com, October 2018.

  1. Benson M., Maurer M. 2016, Amyloidosis Foundation, A Guide to Transthyretin Amyloidosis,

October 2018

  1. Orphan drug designation[TGA],

www.tga.gov.au, October 2018

  1. FDA label,

www.fda.com, May 2019.

  1. Orphan Medicine,

ema.europe.eu, November 2018

  1. Merlini, G., et al. Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis.

Journal of cardiovascular translational research. 2013 October 8; 6(6), 1011-20, October 2018

  1. Pfizer Inc., n.d., Transthyretin amyloidosis,

www.pfizer.com, October 2018

  1. Product Monograph [Health Canada]: Vyndaqel (tafamidis)[PDF],

Pfizer Limited, January 2020

  1. Summary of Product Characteristics [PMDA]: Vyndaqel (tafamidis)[PDF],

Pfizer Japan, September 2013

  1. Summary of Product Characteristics [TGA]: Vyndaqel (tafamidis)

TGA Australia, March 2020

For more information please visit our blog page“What is Vyndaqel?”

This content has been reviewed by a Medical Doctor.

VYNDAQEL (TAFAMIDIS) Package

30 capsules of 20 mg

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