What's Happening?
Alnylam Pharmaceuticals has presented new data from the HELIOS-B Phase 3 study at the ESC Congress 2025, demonstrating the long-term cardiovascular benefits of vutrisiran for patients with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM). The study shows that vutrisiran significantly reduces the risk of all-cause mortality and cardiovascular events over a 48-month period. The findings highlight the potential of vutrisiran as a first-line treatment for ATTR-CM, offering sustained improvements in quality of life and cardiac biomarkers.
Why It's Important?
The HELIOS-B study's findings reinforce the potential of RNAi therapeutics like vutrisiran to transform the treatment landscape for ATTR-CM, a progressive and life-threatening condition. By demonstrating long-term benefits, the study supports the use of vutrisiran as a first-line therapy, potentially improving patient outcomes and reducing healthcare costs associated with managing the disease. This research also underscores the broader impact of RNAi technology in developing innovative treatments for genetic and rare diseases.
What's Next?
Following the positive results from the HELIOS-B study, vutrisiran may see increased adoption as a standard treatment for ATTR-CM. Alnylam Pharmaceuticals is likely to continue its efforts to expand the drug's availability and explore additional applications of RNAi technology. The study's findings could also prompt further research into the long-term effects of RNAi therapeutics, potentially leading to new breakthroughs in the treatment of other complex diseases.
