August 8, 2016
Pfizer Inc. has announced the publication of a new post-hoc analysis of data from three studies of VYNDAQEL in patients with mild transthyretin familial amyloid polyneuropathy (TTR-FAP). The analysis, which included patients with the Val30Met mutation treated over varying periods of up to 5.5 years, presented that treatment with VYNDAQEL started during the early stage of the disease resulted in minimal neurological disease progression. It also resulted in preservation of body weight, which frequently declines as the disease progresses. VYNDAQEL was well tolerated with no new safety signals observed. The new findings were published online in ‘Amyloid: The Journal of Protein Folding Disorders’.
TTR-FAP is a rare, progressive, genetic, and irreversible neurodegenerative disease that impairs quality of life significantly and is estimated to affect approximately 10,000 people worldwide. On average, people with TTR-FAP die within 10 years of symptom onset, when left untreated.
“These findings underscore the long-term benefits of early intervention with VYNDAQEL for symptomatic patients with TTR-FAP. This analysis, which is based on the longest prospective evaluation to date of any medication being studied for TTR-FAP, provides health care professionals with important insights into the management of patients with this disease,” said Dr. Kevin W. Williams, Chief Medical Officer, Rare Disease, Pfizer Innovative Health.