Pfizer’s Vyndaqel shows long-term effectiveness in new study

Pfizer has announced new clinical trial data demonstrating the long-term effectiveness of Vyndaqel in the treatment of a rare neurodegenerative disease.

Post-hoc analysis has been carried out on data from three studies in patients with mild transthyretin familial amyloid polyneuropathy (TTR-FAP), including subjects with the Val30Met mutation treated over varying periods of up to 5.5 years.

It was shown that treatment with Vyndaqel initiated during the early stage of the disease resulted in minimised neurological disease progression and optimum preservation of body weight – something that often declines as the disease progresses.

The drug was also shown to be well tolerated, with no new safety signals observed. This demonstrates the benefits associated with early Vyndaqel interventions.

Dr Kevin Williams, chief medical officer for rare diseases at Pfizer Innovative Health, said: "This analysis, which is based on the longest prospective evaluation to date of any medication being studied for TTR-FAP, provides healthcare professionals with important insights into the management of patients with this disease."

This comes after the company recently announced results from a phase III trial demonstrating the strong performance of the drug Xeljanz in the treatment of ulcerative colitis.

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