Three post-hoc analysis studies of Vyndaqel (tafamidis meglumine) in patients with mild TTR-FAP published in Amyloid: The Journal of Protein Folding Disorders.- Pfizer

Pfizer Inc. has 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.

The new findings were published online in Amyloid: The Journal of Protein Folding Disorders.The new findings reported in Amyloid are based on data from three sequential studies: an 18-month randomized, double-blind, placebo-controlled Phase III 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 .TTR-FAP is typically accompanied by gastrointestinal issues that can lead to malnutrition and unintentional weight loss, resulting in a decline in modified body mass index (mBMI), a clinical indicator of disease progression and treatment response. The published analysis showed that mBMI was preserved during long-term Vyndaqel treatment, with less than one percent decrease at 5.5 years from baseline. No new safety issues or side effects of Vyndaqel were identified in the long-term evaluation of these 71 patients.

See- Waddington Cruz M, Amass L, Keohane D, Schwartz J, Li H, Gundapaneni B. "Early intervention with tafamidis provides long term (5.5 year) delay of neurologic progression in transthyretin familial amyloid polyneuropathy". Amyloid 2016. doi:10.1080/13506129.2016.1207163.