Pfizer study shows long-term benefits of tafamidis for TTR-FAP

Pfizer has announced the publication of new clinical data that demonstrates the significant long-term benefits offered by its transthyretin amyloid polyneuropathy (TTR-FAP) therapy tafamidis.

A new interim analysis of long-term data from four studies has been published by Amyloid: The Journal of Protein Folding Disorders, showing that treatment with tafamidis is associated with delays in disease progression in patients with hereditary TTR-FAP.

The analysis included patients with the most common TTR-FAP mutation, Val30Met, and showed that Pfizer's drug was well-tolerated, with no unexpected side effects.

Results covered a six-year trial period, making this the longest evaluation of any medication for this fatal neurodegenerative disease, which affects around 10,000 people worldwide.

Dr Kevin Williams, chief medical officer for rare diseases at Pfizer Innovative Health, said the findings "build on previous studies suggesting tafamidis provides benefit when given early in the disease and is associated with delay in disease progression over the long term".

Tafamidis is marketed under the trade name Vyndaqel and has been approved in 40 countries for the treatment of TTR-FAP in adult patients with early-stage symptomatic polyneuropathy, although it is not yet available in the US.

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