Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy

Is the efficacy of acoramidis similar between those with transthyretin amyloid cardiomyopathy with wild-type (ATTRwt-CM) and with a pathogenic/likely pathogenic variant (ATTRv-CM)?Among 611 participants, in this study, 9.6% had ATTRv-CM with similar reduction in all-cause mortality and first cardiovascular hospitalization compared with ATTRwt-CM at 30-month trial completion. Of 380 participants who continued in the open-label extension, 7.1% had ATTRv-CM with a similar reduction in all-cause mortality at 42-month follow-up.In patients randomized to acoramidis and followed up in the open-label extension, reduction in all-cause mortality and cardiovascular hospitalization was similar in those with ATTRv-CM and ATTRwt-CM.

Corresponding Author: Kevin M. Alexander, MD, Stanford University, 300 Pasteur Dr, Floor 2, Room A260, Palo Alto, CA 94305 (kevalex@stanford.edu).

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