Pfizer Inc. 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, showed that treatment with VYNDAQEL 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. VYNDAQEL was well tolerated with no new safety signals observed.
“These findings underscore the long-term benefits of early intervention with VYNDAQEL for symptomatic patients with TTR-FAP,” said Dr. Kevin W. Williams, Chief Medical Officer, Rare Disease, Pfizer Innovative Health. “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 is a rare, genetic, progressive, and irreversible neurodegenerative disease that significantly impairs quality of life and is estimated to affect about 10,000 people worldwide. The disease is caused by a mutation in the gene for the protein transthyretin (TTR), resulting in production of unstable TTR proteins that can accumulate as amyloid deposits in nerves and other organs, interfering with normal function. VYNDAQEL is a medicine designed to specifically stabilize TTR, preventing or slowing the formation of abnormal TTR proteins and subsequent amyloid deposits.
The new findings reported in Amyloid are based on data from three sequential studies: an 18-month randomized, double-blind, placebo-controlled Phase 3 pivotal trial of 125 TTR-FAP patients; its 12-month open-label extension; and a second, ongoing, long-term open-label extension study.
This descriptive analysis examined a subset of 71 of the randomized patients whose neurological impairment was defined as mild just prior to starting treatment with VYNDAQEL, either at study start (for those randomized to VYNDAQEL) or upon entry into the first open-label extension (for those randomized to placebo).
In the 31 patients observed at 5.5 years, the evaluation showed that treatment with VYNDAQEL resulted in minimal neurologic disease progression: a mean change from baseline of 5.3 NIS-LL points. This translates to an annual rate of 1.0 point increase in NIS-LL. The lack of a direct control group is a limitation of this study.
