Abstract
Oral tafamidis (Vyndaqel®) is the first disease-modifying drug approved to delay the progression of peripheral neurological impairment in adults with early-stage transthyretin (TTR) amyloidosis (i.e. stage 1 symptomatic polyneuropathy), with recent treatment guidelines recommending it as first-line treatment. Tafamidis acts by kinetically stabilizing the TTR tetramer, thereby inhibiting amyloidogenesis. In adults with early-stage TTR familial amyloid polyneuropathy, tafamidis 20 mg once daily was superior to placebo in the efficacy evaluable population with regard to neurological deterioration and health-related quality-of-life. Long-term extension studies have demonstrated that the beneficial effects of tafamidis on these endpoints are maintained for up to 5.5 years, and studies in the clinical practice setting found that a marked proportion of tafamidis-treated patients achieved neurological stability. Tafamidis is generally well tolerated.
Published Version
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