Attruby reduces risk of death by 49% in rare heart condition in long-term study

Key Takeaways

• Transthyretin amyloid cardiomyopathy is increasingly diagnosed in the US (~120,000 affected), particularly among older adults and men, and remains associated with progressive heart failure and substantial mortality.
• Acoramidis (Attruby), a small-molecule oral TTR stabilizer designed to mimic protective TTR effects, entered the market in November 2024 with a listed monthly wholesale acquisition cost of $19,790.
• In the ATTRibute-CM open-label extension (n=389), continuous acoramidis through 54 months reduced all-cause mortality by 44.7% and cardiovascular mortality by 49.3%.
• Biomarker and patient-centered measures favored acoramidis, including mitigation of NT-proBNP progression after placebo crossover and maintenance of quality-of-life scores with ongoing therapy.
• Safety remained consistent long term, with 10 treatment-related discontinuations and treatment-emergent events contributing to hospitalizations (n=191) and fatal outcomes (n=65).