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Vyndaqel Approved to Delay Nerve Damage Caused by Transthyretin Amyloidosis

Vyndaqel is used to delay nerve damage caused by transthyretin amyloidosis, a hereditary disease in which fibres called amyloid build up in tissues around the body including around the nerves. Vyndaqel is used in adult patients with the early stage of nerve disease (stage 1).

Because the number of patients with transthyretin amyloidosis is low, the disease is considered ‘rare’, and Vyndaqel was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 28 August 2006.

The medicine can only be obtained with a prescription.

Treatment should be started and supervised by a doctor knowledgeable in the management of patients with this disease.

The recommended dose is one capsule once a day, swallowed whole. Vyndaqel is used alongside standard care for patients with transthyretin amyloidosis. Doctors will need to monitor patients and assess their need for other treatments including liver transplantation. Patients undergoing liver transplantation will have to stop taking Vyndaqel.

In patients with transthyretin amyloidosis, the amyloid fibres that surround and damage the nerves are formed of a protein called transthyretin. The transthyretin in these patients is defective: it breaks easily and is deposited in tissues and organs around the body.

The active substance in Vyndaqel, tafamidis, is a transthyretin stabilizer. It attaches to the protein, which prevents the protein from breaking up, thereby stopping the formation of amyloid and slowing down the progression of the nerve disease.

The effects of Vyndaqel were first tested in experimental models before being studied in humans. Vyndaqel was compared with placebo (a dummy treatment) in 128 patients with transthyretin amyloidosis. The main measure of effectiveness was based on an assessment of the patients’ nerve damage and quality of life after 18 months of treatment.

Vyndaqel was more effective than placebo at treating patients with transthyretin amyloidosis. In 45% of patients taking Vyndaqel, nerve function either improved or stabilized, compared with 30% of patients taking placebo. The results did not include patients with more severe disease who left the study early because they needed to undergo a liver transplant.

Patients taking Vyndaqel also had better quality of life scores, but the difference with placebo was not considered significant.

The most common side effects with Vyndaqel (seen in more than 1 patient in 10) are urinary tract infection (infection of the structures that carry urine) and diarrhoea. For the full list of all side effects reported with Vyndaqel, see the package leaflet.

Vyndaqel must not be used in people who are hypersensitive (allergic) to tafamidis or to any of the other ingredients.

The CHMP noted that, apart from in patients who left the study because they needed a liver transplant, Vyndaqel was more effective than placebo in delaying nerve damage in patients with transthyretin amyloidosis. The medicine presented no major safety concerns and the risks were considered to be manageable. The CHMP therefore concluded that the benefits of Vyndaqel are greater than its risks and recommended that it be granted marketing authorisation.

Vyndaqel has been authorised under ‘exceptional circumstances’. This means that because transthyretin amyloidosis is rare, for scientific reasons, it has not been possible to obtain complete information about Vyndaqel. Every year, the European Medicines Agency will review any new information that may become available and this summary will be updated as necessary.

The company that makes Vyndaqel will carry out a study on the effects of the medicine in a subgroup of patients with certain genetic mutations. The company will also provide long-term post-marketing safety data.

The company will provide educational material to all doctors expected to prescribe Vyndaqel. The material will include a leaflet containing information on the correct use of the medicine and on how to contribute to a post marketing study and a surveillance programme.


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Source :

European Medicines Agency

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