Abstract
Background Tafamidis is a transthyretin (TTR) stabilizer approved to delay neurological progression associated with stage 1 TTR familial amyloid polyneuropathy (FAP). A placebocontrolled, 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.
Highlights
Tafamidis is a transthyretin (TTR) stabilizer approved to delay neurological progression associated with stage 1 TTR familial amyloid polyneuropathy (FAP)
A placebocontrolled, 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
For 3 patients, there was evidence after 3-5 years of treatment of an increase in the Neuropathy Impairment Scale for lower limbs (NIS-LL) score to 30 or more. This analysis describes the trajectory of the disease progression over 5.5 years or more for 71 patients with the V30M mutation and stage 1 TTR-FAP who received tafamidis either at the start of the registration trial or after the switch from placebo following 18 months of study participation
Summary
Tafamidis is a transthyretin (TTR) stabilizer approved to delay neurological progression associated with stage 1 TTR familial amyloid polyneuropathy (FAP). Intervention with tafamidis provides long-term benefit in delaying neurological progression in patients with transthyretin familial amyloid polyneuropathy From First European Congress on Hereditary ATTR amyloidosis Paris, France. Background Tafamidis is a transthyretin (TTR) stabilizer approved to delay neurological progression associated with stage 1 TTR familial amyloid polyneuropathy (FAP).
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