The U.S. Food and Drug Administration (FDA) has approved vutrisiran for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), Alynlam Pharmaceuticals announced in a press release on March 20, 2025.
The drug, which is marketed as AMVUTTRA®, is the first therapy to be approved to treat both ATTR-CM and hereditary transthyretin amyloid polyneuropathy (hATTR-PN).
Vutrisiran is an RNA interference therapeutic that binds to transthyretin messenger RNA to prevent its translation into protein, inhibiting the accumulation of misfolded transthyretin in the body. It is administered to patients via a subcutaneous injection four times per year.
Approval was granted based on the results of the HELIOS-B trial, which demonstrated the safety and efficacy of vutrisiran in patients with ATTR-CM. The findings were published in the New England Journal of Medicine and presented at the European Society of Cardiology Congress.
Read more about ATTR-CM therapies
Over a three-year period, the Phase 3 clinical trial found that the combined risk of all-cause mortality and cardiovascular events was reduced by 28% in patients receiving vutrisiran compared to those receiving a placebo.
Additionally, patients receiving vutrisiran reported overall improved quality of life and functional abilities compared to patients receiving the placebo. NT-proBNP and troponin I levels, which are indicators of cardiac damage, were also reduced in the treatment group.
The safety of vutrisiran was studied extensively in the HELIOS-A trial in patients with hATTR-PN. The most commonly reported side effects included extremity pain, joint pain and shortness of breath. No additional adverse reactions were identified in individuals with ATTR-CM during the HELIOS-B study.
“AMVUTTRA in hATTR-PN is covered by insurers for ~99% of patients with the majority paying $0 out-of-pocket,” Alnylam Pharmaceuticals wrote in their press release. “Similar broad coverage and out-of-pocket costs are expected in ATTR-CM given comparable payer dynamics and the clinical value demonstrated in the HELIOS-B clinical trial.”
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