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FDA panel supports Pfizer's tafamidis despite efficacy concerns

Rare nerve disorder drug backed despite missing out on primary objective in phase III trial

A US FDA advisory committee recommended approval of Pfizer's tafamidis meglumine for a rare nerve disorder called transthyretin familial amyloid polyneuropathy (TTR-FAP), despite concluding that a trial of the drug missed its primary objective.

Panellists voted by 13 to 4 that tafamidis had not slowed the progression of TTR-FAP, but by the same margin said that its effects on secondary measures - such as lower limb function - were sufficient to support approval.

According to Pfizer, most of the committee members agreed that the data "provide substantial evidence of efficacy for a surrogate endpoint that is reasonably likely to predict a clinical benefit". The FDA is scheduled to deliver its verdict on the marketing application in June.

TTR-FAP affects around 2,500 patients in the US and is characterised by a build-up of amyloid deposits in peripheral nerves, leading to sensory loss, pain and weakness in the lower limbs. It is a progressive condition, generally leading to disability and death within 10 years of diagnosis.

The panel's willingness to back tafamidis in the face of limited efficacy data stems from the lack of any approved medicine to treat TTR-FAP in the US. At present the only disease-modifying therapy open to patients is a liver transplant.

Pfizer was granted approval in November, 2011, for the drug in Europe, where it is marketed as Vyndaqel.  The company's trials have suggested that the drug can delay peripheral neurologic impairment and reduced progressive loss of neurological and motor function by 51 to 81 per cent compared to placebo.

Commenting on the FDA panel vote, Dr Yvonne Greenstreet, head of the medicines development group at Pfizer's specialty care business unit, said it was "a positive step forward in our goal to provide this much-needed medicine to patients suffering from this rare and fatal disease".

Tafamidis is thought to work by stabilising 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.

25th May 2012

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