Pfizer's Vyndaqel shows promise in slowing transthyretin familial amyloid polyneuropathy

Findings from three studies have been published which suggest that Vyndaqel (tafamidis), a drug being developed by US pharma giant Pfizer (NYSE: PFE), could slow the progression of a rare neurodegenerative disease.

The drug was trialled in patients with mild transthyretin familial amyloid polyneuropathy (TTR-FAP), a rare, genetic, progressive, and irreversible neurodegenerative disease that significantly impairs quality of life and is estimated to affect about 10,000 people worldwide.

In the analysis, which included patients with the Val30Met mutation treated over varying periods of up to 5.5 years, treatment with Vyndaqel which was initiated during the early stage of the disease resulted in minimal neurological disease progression and in preservation of body weight, which often declines as the disease progresses.