Pfizer Inc. has announced that analysis of data from three studies involving VYNDAQEL have been published.
The studies researched how patients with mild transthyretin familial amyloid polyneuropathy (TTR-FAP) responded to the drug.
The studies dealt with patients who suffer from the Val30Met mutation at the beginning stages of the disease. The individuals were treated for five-and-a-half years, and they showed very little progression in the neurological disease as well as preservation of weight, which normally decreases with the progression of the disease. VYNDAQEL proved to be tolerable during the studies with no new safety signals observed.
“These findings underscore the long-term benefits of early intervention with VYNDAQEL for symptomatic patients with TTR-FAP,” Pfizer Innovative Health Chief Medical Officer Dr. Keith W. Williams said. “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.”
TTR-FAP affects an estimated 10,000 people on a global basis. It is a rare and genetic neurodegenerative disease that decreases quality of life. It is currently irreversible.
The analysis of data related to the studies was published in Amyloid: the Journal of Protein Folding Disorders.