FDA Expands Vutrisiran Indication to ATTR-Cardiomyopathy

The US Food and Drug Administration (FDA) has approved a supplemental new drug application for vutrisiran for the treatment of wild-type or hereditary transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) to reduce cardiovascular death, hospitalization, and urgent heart failure visits.

ATTR-CM is a rapidly progressive and ultimately fatal disease affecting roughly 150,000 Americans.

Vutrisiran is an RNA interference therapeutic administered via subcutaneous injection administered once every 3 months. 

By rapidly knocking down TTR production, vutrisiran decreases deposition of TTR fibrils, which form amyloid and cause irreversible cardiovascular damage and premature death in patients with ATTR-CM, the drug’s manufacturer said in a news release. 

The approval was based on the HELIOS-B phase 3 study, which showed that treatment with vutrisiran led to impressive reductions in all-cause death and recurrent cardiovascular events in patients with ATTR-CM. The study achieved statistical significance compared to placebo on all 10 pre-specified primary and secondary endpoints. 

The results were presented at the European Society of Cardiology Congress last year and simultaneously published in The New England Journal of Medicine, and reported by Medscape Medical News at that time. 

“This FDA approval provides an opportunity to further transform ATTR-CM treatment with a new mechanism of action. The HELIOS-B clinical trial found that vutrisiran allowed patients to live longer, experience fewer hospitalizations, and improve how they function and feel,” Ronald Witteles, MD, HELIOS-B investigator, professor of medicine at Stanford University School of Medicine and co-director of the Stanford Amyloid Center, said in the release. 

“The trial enrolled patients who mirror the real-world population with this disease, and I am very encouraged by vutrisiran’s ability to demonstrate meaningful clinical benefits across both cardiovascular outcomes and multiple measures of disease progression. This is a very exciting day for patients with this challenging disease,” Witteles added.

As reported by Medscape Medical News, the FDA initially approved vutrisiran in 2022 for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.