Pfizer Publish New Analysis Showing Long Term Therapy Slowed Progression of Rare Neurodegenerative Disease
Pfizer Inc. announced the recent publication of a 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 and a half 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. Left untreated, those afflicted with TTR-FAP die within 10 years of symptom onset, on average. 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.
Patients with TTR-FAP experience a considerable burden of illness early in the course of disease and this burden increases with disease progression. They typically require assistance with walking 5 to 6 years after initial symptoms. As TTR-FAP symptoms progress, patients require a considerable amount of assistance, are unable to care for themselves, and may become bedridden or require hospitalisation.
VYNDAQEL is a novel specific TTR stabilizer indicated in the European Union for the treatment of TTR-FAP in adult patients with early-stage symptomatic polyneuropathy to delay peripheral neurologic impairment. Since its EU approval in 2011, VYNDAQEL has also been approved in Japan, Mexico, Argentina, Israel, and South Korea. VYNDAQEL is not approved in the United States.