FDA Approves Amvuttra™ (vutrisiran) for ATTR-CM: More Options for Patients

BREAKING NEWS: The U.S. Food and Drug Administration has approved Alnylam’s AMVUTTRA™ (vutrisiran) for the treatment of patients with transthyretin amyloid cardiomyopathy (ATTR-CM).

The US FDA’s approval of AMVUTTRA adds another option for patients living with transthyretin amyloidosis (ATTR — both wild-type and hereditary) who have heart involvement (cardiomyopathy). Previously approved by the FDA in 2022 for ATTR-PN (transthyretin amyloid polyneuropathy), AMVUTTRA is the first gene-silencing treatment approved for the treatment of ATTR-CM.

What is ATTR-CM?

ATTR-CM is a rare and progressive disease where a protein called transthyretin (TTR) misfolds and forms harmful amyloid deposits in the heart. Over time, these deposits can lead to heart failure, making it difficult for the heart to pump blood properly. Although treatments for ATTR-CM have been limited, the approval of Amvuttra offers more hope for patients affected by this debilitating disease.

What is Amvuttra?

Amvuttra is a transthyretin (TTR) knockdown agent, which works with the body’s natural system to stop the production of the TTR protein and slow down the development of amyloidosis. By decreasing the amount of TTR protein made in the liver, fewer harmful amyloid fibrils end up depositing in the heart and causing cardiomyopathy. The drug is administered by an injection under the skin once every three months in a hospital or clinic setting.

Why This Approval Matters

The FDA’s approval of Amvuttra provides a new type of treatment for ATTR-CM; one that has delivered beneficial results for those living with ATTR-PN. While previously FDA-approved ATTR-CM therapies — Attruby and Vyndamax/Vyndaqel — work by stabilizing the TTR protein made in the liver, Amvuttra works differently by slowing down the production of these proteins entirely. Both types of therapies slow down the progression of ATTR-CM. Patients and their doctors can work together to choose the treatment that best meets their needs, ultimately improving heart health and quality of life.

“The FDA approval of Amvuttra is a major advancement for patients with ATTR-CM, as it introduces the first gene-silencing approach to treating this disease. With this approval, patients now have a choice between two distinct disease-modifying treatment strategies — TTR stabilizers or TTR gene silencers — allowing for a more personalized approach to care. This milestone not only expands treatment options but also represents important progress in addressing the underlying cause of ATTR-CM and improving patient outcomes.”

— Kristen Hsu, Executive Director of Research, ARC

Amvuttra and Insurance

Insurance coverage of Amvuttra will vary depending on the particular plan. If insurance does not provide enough coverage, there are patient assistance programs that will help. This includes the Alnylam patient support program, Alnylam Assist. You can learn more about this program on Alnylam’s website at alnylamassist.com or call 1-833-256-2748.

If you are on Medicare, Amvuttra will be covered under Medicare Part B.

More Information

For more information you can contact us by phone at 617-467-5170 or by email at support@arci.org.

Download ARC’s Amvuttra™ (vutrisiran) ATTR-CM fact sheet here.

Learn about hereditary ATTR amyloidosis here.

Learn about wild-type ATTR amyloidosis here.