Data from a Phase II/III trial of transthyretin familial amyloid polyneuropathy (TTR-FAP) showed that Pfizer and FoldRx's drug tafamidis slowed disease progression over 30 months. No safety concerns were identified, and none of the 86 patients in the study discontinued treatment due to adverse events. TTR-FAP is a rare and fatal neurodegenerative disease that, left untreated, is fatal.
Those who received tafamidis for the entire 30 months showed less progression than those who got the drug after receiving placebo for the first 18 months and the drug for just 12 months. Large and small nerve fiber function was also better preserved in the longer treatment group. "We are encouraged by the consistency of the results and preservation of neurologic function across all endpoints, as well as the long-term maintenance effect over 30 months," stated Dr. Teresa Coelho of the Hospital Santo Antonio in Porto, Portugal, a principal investigator in the study.
- here's Pfizer's release