Vutrisiran Shows Promising Results in Phase 3 Study for ATTR-CM Treatment

Positive Phase 3 Results:
Vutrisiran, an investigational RNA interference therapeutic developed by Alnylam Pharmaceuticals, has shown positive topline results in the HELIOS-B phase 3 study for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).

Improved Cardiovascular Outcomes:
The study demonstrated statistically significant improvements in cardiovascular outcomes, including survival, function, and quality of life in all patient groups with ATTR-CM.

Reduction in Mortality and Cardiovascular Events:
Vutrisiran reduced the risk of all-cause mortality and recurrent cardiovascular events by 28% in the overall population and by 33% in the monotherapy population.

Safety and Tolerability:
The safety and tolerability profile of vutrisiran was consistent with its established profile, with similar rates of adverse events between the vutrisiran and placebo groups.

Regulatory Submissions:
Alnylam plans to file a US Supplemental New Drug Application using a Priority Review Voucher and proceed with global regulatory submissions later this year.

Potential New Standard of Care:
The results suggest that vutrisiran has the potential to become a new standard of care for the treatment of ATTR-CM, a progressive and ultimately fatal disease with limited treatment options.