Studied in people with ATTR‑CM and hATTR-PN

Safety and effectiveness of AMVUTTRA® (vutrisiran) were shown in 2 clinical studies

The ATTR-CM study

  • 654 adults with cardiomyopathy of wild-type or hereditary ATTR

  • Up to 3½ years of data

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The hATTR-PN study

  • 164 adults with polyneuropathy of hereditary ATTR

  • 18 months of data

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How AMVUTTRA was studied in people with ATTR-CM 

Chart showing how AMVUTTRA limits TTR production in the liver

The safety and effectiveness of AMVUTTRA was determined by comparing the results of people taking AMVUTTRA to people taking placebo (treatment without any active medication). Both groups were made up of people who were either taking a stabilizer or not taking a stabilizer at the start of the study.​

  • ​The main measure of effectiveness was the combined risk of death and heart-related hospital stays and urgent visits over 3 years

  • Additional measures of effectiveness included exercise capacity and health-related quality of life at 2½ years

After the first 3 years of the study, people receiving placebo could switch to AMVUTTRA. This means that everyone still in the study received AMVUTTRA for up to an additional 6 months.​​​

  • The study then measured the risk of death over 3½ years and compared people who originally received AMVUTTRA to those who originally received placebo

A range of people with ATTR-CM took part in the study 

Heart failure severity is based on the New York Heart Association class, a system that categorizes heart failure into 4 levels depending on the level of activity that causes symptoms such as extra tiredness, shortness of breath or pounding chest.

  • No symptoms—Normal physical activity (like jogging or walking) does not cause symptoms

  • Mild—Normal physical activity (like jogging or walking) causes symptoms

  • Moderate—Lighter than normal activity (like climbing one flight of stairs at a normal pace) causes symptoms

  • Severe—Any physical activity causes symptoms. The ATTR-CM study did not include people with severe symptoms

Explore results in ATTR-CM

How AMVUTTRA was studied in people with hATTR-PN 

Chart showing how AMVUTTRA limits TTR production in the liver

The study compared 122 people who took AMVUTTRA to 77 people from a similar study who were given a placebo (treatment without any active medication).

  • At 9 months and 18 months, nerve function and quality of life were measured

  • At 18 months, walking speed, nutritional health, and activities of daily living were also measured

A range of people with hATTR-PN took part in the study 

Nerve disability was based on Polyneuropathy Disability (PND) scores.​​

  • Mild—No walking assistance is required

  • Moderate—Walking assistance (such as a crutch or walker) is required

  • Severe—Inability to walk leads to wheelchair use or being bedridden. The hATTR-PN study did not include people with severe symptoms

Explore results in hATTR-PN

Up Next

How AMVUTTRA may help ATTR-CM

Important Safety Information

What are the most important things I should know about AMVUTTRA® (vutrisiran)?

AMVUTTRA can cause low vitamin A levels

Treatment with AMVUTTRA lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day. You should not take more than the amount of vitamin A recommended by your doctor.

Low vitamin A levels can affect vision. If you have problems with your vision (e.g., night blindness) while taking AMVUTTRA, talk to your doctor. Your doctor may refer you to an eye specialist.

What are the common side effects of AMVUTTRA?

The most common side effects of AMVUTTRA were pain in the arms or legs, pain in the joints, shortness of breath, and low vitamin A levels.

These are not all the possible side effects of AMVUTTRA. Talk to your doctor about side effects that you experience. You are encouraged to report negative side effects of prescription drugs to the U.S. Food and Drug Administration (FDA). Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

For additional information about AMVUTTRA, please see the full Prescribing Information.

Indications

What is AMVUTTRA?

AMVUTTRA is a prescription medicine that treats the:

  • cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce heart-related death, hospital stays and urgent visits.
  • polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

cardiomyopathy of
ATTR amyloidosis

polyneuropathy of hereditary
ATTR amyloidosis

transthyretin

transthyretin-mediated
amyloidosis

Kansas City Cardiomyopathy
Questionnaire

6-minute walk test

modified Neuropathy
Impairment Score +7

Quality of Life-Diabetic
Neuropathy