Vutrisiran Snags FDA Approval for TTR Amyloidosis Cardiomyopathy

Cardiology
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CHF

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RNA interference therapeutic was previously limited to treating polyneuropathy from the disease

by
Nicole Lou, Senior Staff Writer, MedPage Today
March 21, 2025 • 2 min read

Gene-silencing therapy vutrisiran (Amvuttra) graduated to a broad indication in treatment of transthyretin amyloidosis cardiomyopathy, as Alnylam Pharmaceuticals announced expanded FDA approval.

Vutrisiran, a transthyretin-directed small interfering RNA, had its approval expanded to treat cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations, and urgent heart failure visits.

This makes vutrisiran the first FDA-approved RNA interference treatment for ATTR-CM, applicable both to those newly diagnosed and those progressing on existing transthyretin-stabilizing therapy (e.g., tafamidis [Vyndamax, Vyndaqel] or acoramidis [Attruby]). Vutrisiran had been initially approved in 2022 for the treatment of the polyneuropathy of ATTR-CM.

“The FDA approval of Amvuttra for ATTR-CM marks a pivotal advancement for patients, providing a new and clinically differentiated treatment option that has been shown to improve outcomes, including cardiovascular mortality, and reduce progression for those living with this devastating disease,” said Alnylam’s CEO Yvonne Greenstreet, MBChB, in a press release.

The company had previously tried and failed to get an ATTR-CM indication for another of its gene silencers, patisiran (Onpattro).

This time around, FDA approved Alnylam’s supplemental new drug application for vutrisiran based on the 655-person HELIOS-B phase III trial. In those study results released last summer, vutrisiran reduced mortality and recurrent cardiovascular events in ATTR-CM through 36 months of treatment, whether patients were also using background tafamidis or not.

“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,” investigator Ronald Witteles, MD, of Stanford University School of Medicine in California, said in a statement.

Vutrisiran is available as a 25 mg/0.5 mL subcutaneous injection to be administered quarterly by a healthcare professional.

Its label warns of reduced serum vitamin A levels among users. The most common adverse reactions include pain in extremity, arthralgia, and dyspnea on top of reduced vitamin A.

Nicole Lou is a reporter for MedPage Today, where she covers cardiology news and other developments in medicine. Follow