Early intervention with tafamidis provides long-term benefit in delaying neurological progression in patients with transthyretin familial amyloid polyneuropathy

Abstract

Tafamidis is a transthyretin (TTR) stabilizer approved to delay neurological progression associated with stage 1 TTR familial amyloid polyneuropathy (FAP). A placebo-controlled, randomized 18-month registration trial allowed for continued evaluation of patients receiving tafamidis (20 mg oral once-daily) through an ongoing open label extension study. The effectiveness of tafamidis for delaying long-term neurological progression relative to baseline levels of neuropathy impairment at the start of treatment has not been reported previously.