ATTR-CM

Alnylam Pharmaceuticals’ AMVUTTRA (vutrisiran) showed sustained benefits in transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM) patients in the HELIOS-B Phase 3 study. Data presented at ESC Congress 2025 revealed that vutrisiran reduced the risk of all-cause mortality or first cardiovascular event by 37% in the overall population and 42% in the monotherapy group over 48 months. The open-label extension maintained improvements in quality of life and cardiac biomarkers, supporting vutrisiran as a potential first-line ATTR-CM therapy.

BridgeBio Pharma’s acoramidis demonstrated a 44% reduction in cardiovascular mortality risk in ATTR-CM patients after 42 months in the ATTRibute-CM open label extension study. The study also showed a 46% hazard reduction in cardiovascular mortality or first cardiovascular hospitalization. Approximately 50% of participants exhibited disease stabilization compared to less than 20% on placebo at month 30. Acoramidis, marketed as Attruby® and BEYONTTRA®, exhibited the fastest benefit observed in a Phase 3 ATTR-CM study, with positive changes seen in 3 months.