Vutrisiran for Cardiomyopathy Associated with Transthyretin Amyloidosis

The RNA silencer reduced the risk for death and cardiovascular events in both hereditary and wild-type disease.

Transthyretin (TTR) amyloidosis causes amyloid fibrils to accumulate in tissue, including cardiac muscle, which can lead to cardiomyopathy. The condition, stemming from a genetic abnormality (variant) or aging (wild-type), is characterized by progressive physical decline and risk for early mortality. Tafamidis, a TTR stabilizer, is the only FDA-approved treatment for TTR amyloidosis with cardiomyopathy (ATTR-CM), but a recent trial showed the efficacy of another stabilizer, acoramidis (NEJM JW Cardiol Jan 17 2024 and N Engl J Med 2024; 390:132). Researchers have now conducted a manufacturer-funded trial (NCT04153149) of vutrisiran, which inhibits hepatic synthesis of both wild-type and variant TTR messenger RNA.

The investigators randomized …

Reviewing Author

Frederick A. Masoudi, MD, MSPH, MACC, FAHA

Disclosures

Consultant/Advisory BoardBristol Myers Squibb; CPC Clinical Research

Grant/Research SupportNational Heart, Lung, and Blood Institute

Editorial BoardsUpToDate; American College of Cardiology Self-Assessment Program (SAP)

Leadership Positions in Professional SocietiesAmerican College of Cardiology (Chair, Innovations Committee)