JANUARY 11, 2024 | J.D. Gillmore, D.P. Judge, F. Cappelli, M. Fontana, P. Garcia-Pavia, S. Gibbs, M. Grogan, M. Hanna, J. Hoffman, A. Masri, M.S. Maurer, J. Nativi-Nicolau, L. Obici, S.H. Poulsen, F. Rockhold, K.B. Shah, P. Soman, J. Garg, K. Chiswell, H. Xu, X. Cao, T. Lystig, U. Sinha, and J.C. Fox, for the ATTRibute-CM Investigators*
The study evaluated the efficacy and safety of acoramidis, a high-affinity transthyretin (TTR) stabilizer, in patients with transthyretin amyloid cardiomyopathy. In a phase 3, double-blind trial, 632 patients were randomly assigned to receive either acoramidis hydrochloride (800 mg twice daily) or placebo for 30 months. The primary analysis favored acoramidis over placebo, with a win ratio of 1.8 (95% CI, 1.4 to 2.2), indicating better outcomes in terms of mortality, cardiovascular-related hospitalization, changes in NT-proBNP levels, and 6-minute walk distance. The overall incidence of adverse events was similar between the two groups, with fewer serious adverse events reported in the acoramidis group. The findings suggest that acoramidis is an effective and safe treatment option for patients with transthyretin amyloid cardiomyopathy.The study evaluated the efficacy and safety of acoramidis, a high-affinity transthyretin (TTR) stabilizer, in patients with transthyretin amyloid cardiomyopathy. In a phase 3, double-blind trial, 632 patients were randomly assigned to receive either acoramidis hydrochloride (800 mg twice daily) or placebo for 30 months. The primary analysis favored acoramidis over placebo, with a win ratio of 1.8 (95% CI, 1.4 to 2.2), indicating better outcomes in terms of mortality, cardiovascular-related hospitalization, changes in NT-proBNP levels, and 6-minute walk distance. The overall incidence of adverse events was similar between the two groups, with fewer serious adverse events reported in the acoramidis group. The findings suggest that acoramidis is an effective and safe treatment option for patients with transthyretin amyloid cardiomyopathy.