On August 6th, 2015, the Phase 3 Transthyretin Cardiomyopathy Study (“ATTR-ACT Study”, B3461028) reached an important milestone by completing enrollment.
ATTR-ACT is a multicenter, international, double-blind, placebo-controlled, randomized study to evaluate the efficacy, safety, and tolerability of daily oral dosing of tafamidis 20 mg or 80 mg in comparison to placebo in subjects diagnosed with transthyretin cardiomyopathy (TTR-CM). The study is designed to assess the potential benefit both variant and wild-type subjects. The primary analysis is based on all-cause mortality and frequency of cardiovascular-related hospitalizations (including heart failure, arrhythmia, myocardial infarction, and stroke as well as other cardiovascular-related events) during the 30 month trial. Key secondary endpoints include the 6 minute walk test and a quality of life scale, the Kansas City Cardiomyopathy Questionnaire (KCCQ). ClinicalTrials.gov Identifier: NCT01994889
Transthyretin amyloid disease is a rare and fatal condition characterized by the deposition of amyloid derived from transthyretin (a plasma protein) in various organs and tissues. Deposition of TTR amyloid is associated with two distinct clinical presentations: transthyretin familial amyloid polyneuropathy (TTR‑FAP) when the peripheral nerves are primarily affected and transthyretin amyloid cardiomyopathy (TTR-CM) when the heart is primarily affected. Both TTR-FAP and TTR‑CM are associated with genetic variants of transthyretin but TTR‑CM may also occur in the absence of any genetic mutation and may be due to wild-type TTR amyloid deposition. At this time, there are no approved pharmacotherapies for TTR Cardiomyopathy.
Tafamidis is an investigational drug and is not approved in the United States.