A new analysis of data pooled from three clinical trials backs early intervention with Pfizer's Vyndaqel in patients with the rare neurodegenerative disease transthyretin familial amyloid polyneuropathy (TTR-FAP).
According to the data, published online in Amyloid: The Journal of Protein Folding Disorders, treatment with Vyndaqel initiated during the early stage of the condition resulted in minimal neurological disease progression and preserved body weight, which often declines as the disease progresses because of gastrointestinal issues.
On a further positive note, the drug was also found to be well tolerated with no new safety signals observed, the most common adverse reactions being urinary tract infections (28.2 percent), cold (25.4 percent), influenza (23.9 percent), diarrhoea (22.5 percent), and headache and pain in an extremity (both 19.7 percent).
"These findings underscore the long-term benefits of early intervention with Vyndaqel for symptomatic patients with TTR-FAP," said Dr Kevin Williams, chief medical officer, Rare Disease, at 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 genetic, progressive and irreversible neurodegenerative disease 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.
The disease, which is estimated to affect around 10,000 people globally, significantly impairs quality of life and, if left untreated, patients die within 10 years of symptom onset, on average.
Vyndaqel is designed to stabilise TTR to prevent or slow the formation of abnormal proteins and subsequent amyloid deposits. The drug is approved for use in the European Union but not in the US.