Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy.
Julian D GillmoreDaniel P JudgeFrancesco CappelliMarianna FontanaPablo Garcia-PaviaSimon GibbsMartha GroganMazen HannaJames HoffmanAhmad MasriMathew S MaurerJose Nativi-NicolauLaura ObiciSteen Hvitfeldt PoulsenFrank RockholdKeyur B ShahPrem SomanJyotsna GargKaren ChiswellHaolin XuXiaofan CaoTed LystigUma SinhaJonathan C Foxnull nullPublished in: The New England journal of medicine (2024)
In patients with transthyretin amyloid cardiomyopathy, the receipt of acoramidis resulted in a significantly better four-step primary hierarchical outcome containing components of mortality, morbidity, and function than placebo. Adverse events were similar in the two groups. (Funded by BridgeBio Pharma; ATTRibute-CM ClinicalTrials.gov number, NCT03860935.).