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FDA starts review of Pfizer's genetic disorder drug

Tafamidis meglumine offers alternative to liver transplant for TTR-FAP

The US Food and Drug Administration (FDA) has started to review Pfizer's marketing application for tafamidis meglumine, a new drug for the rare genetic disorder transthyretin familial amyloid polyneuropathy (TTR-FAP).

An earlier attempt to submit the drug for approval in the US resulted in a disappointment after the FDA issued a Refuse to File (RTF) response requesting additional data.

TTR-FAP is a rare, progressive and fatal neurodegenerative disease that affects approximately 8,000 patients worldwide.

The disease usually emerges from age 20-40 and is characterised by the formation of amyloid deposits in nerves and organ systems, leading to breakdown in organ function, pain, weakness and sensory loss.

Life expectancy for untreated patients has typically only been around 10 years from diagnosis, and treatment options are extremely limited, consisting mainly of liver transplantation and/or supportive care.

Pfizer's orally-administered drug represents a major step forward for TTR-FAP patients, with donated livers limited.

There is also a 10 per cent mortality rate associated with transplantation surgery and the requirement for lifelong immunosuppressive therapy.

It has already been approved in the EU under the Vyndaqel trade name and was scheduled for launch there in early 2012.

An 18-month phase II/III study conducted in support of approval showed that tafamidis meglumine delayed peripheral neurologic impairment, particularly for patients in the early stages of the disease.

Additional data from the study showed 51 to 81 per cent less deterioration in neurologic function, large fibre function (a measure of motor strength) and small fibre function (a measure of sensation) with tafamidis meglumine compared with patients treated with placebo.

Treatment with Pfizer's drug also resulted in improved nutritional status, while side effects were manageable and limited to diarrhoea, upper abdominal pain, urinary tract infection and vaginal infection.

Tafamidis meglumine works as a specific transthyretin stabiliser, preventing the protein from dissociating, misfolding and causing amyloid deposition.

In addition to TTR-FAP the compound is also in testing for two related conditions known as senile systemic amyloidosis and familial amyloid cardiomyopathy.

Pfizer acquired the drug when it purchased FoldRx in 2010.

16th February 2012

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