Efficacy And Safety Of Tafamidis Doses In The Tafamidis In Transthyretin Cardiomyopathy Clinical Tri

The Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR‐ACT) demonstrated the effectiveness of tafamidis for treatment of transthyretin amyloid cardiomyopathy (ATTR‐CM). Although ATTR‐ACT was not designed for a dose‐specific assessment, guidance for determination of the optimal dose can be gained from further analysis of ATTR‐ACT and its long‐term extension study (LTE). In ATTR‐ACT, randomization was performed of patients (2:1:2) to tafamidis 80 mg, 20 mg, or placebo for 30 months. Enrollment of patients completing ATTR‐ACT in the LTE was done (with placebo‐treated patients randomized to tafamidis 80 or 20 mg; 2:1) and subsequent switching of all patients to high‐dose tafamidis was performed. Outcomes support the effectiveness of tafamidis, both 80 and 20 mg, in lowering mortality and cardiovascular‐related hospitalizations in patients with ATTR‐CM. Tafamidis 80 mg was supported to be the optimal dose considering the longer‐term survival data and the lack of dose‐related safety concerns.

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