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Pfizer gets EU approval for neurodegenerative disease drug

Vyndaqel authorised for the treatment of transthyretin familial amyloid polyneuropathy in Europe

The European Commission (EC) has approved a new Pfizer drug for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP), a rare and fatal neurodegenerative disease affecting around 8,000 people worldwide.

Vyndaqel (tafamidis; formerly Fx-1006A) becomes the first treatment approved anywhere in the world to treat the disease. The condition is characterised by deposits of amyloid fibrils in nerves and organs, typically the heart and kidneys, impairing their function.

The disease usually begins in an individual's third or fourth decade and the outcome is invariably progressive and fatal, with liver transplantation currently the only disease-modifying treatment available to patients.

"Today marks a real breakthrough for patients in the EU living with TTR-FAP," said Yvonne Greenstreet, senior vice president and head of the Medicines Development Group at Pfizer's Specialty Care Business Unit. "This community urgently needs an effective therapy."

In trials, Vyndaqel delayed peripheral neurologic impairment and reduced progressive loss of neurological and motor function by 51 to 81 per cent compared to placebo, with accompanying increases in nutritional status and body mass index.

Vyndaqel is a disease-modifying agent that stabilises the TTR protein, preventing the dissociation of the usual tetramer structure into monomers, and as a result blocking amyloid fibril formation. The compound was originally discovered by FoldRx Pharmaceuticals, which was acquired by Pfizer in 2010 and now operates as a wholly-owned subsidiary.

Pfizer said it hopes to make Vyndaqel available to patients in Europe early next year. US patients will have to wait for access to the drug, however, as early this year the US Food and Drug Administration (FDA) refused to accept its marketing application, saying it needed additional data.

Pfizer said at the time it would be able to supply the required information without having to conduct any additional preclinical or clinical studies. The treatment has orphan drug designation in both the EU and US.

18th November 2011


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