Transthyretin amyloid cardiomyopathy (ATTR-CM) is a condition that can lead to the heart not pumping well.
Attruby (acoramidis) was approved in 2024 by the FDA to treat people with ATTR-CM. It has been shown to lower the risk of heart-related death or hospitalization due to heart problems.
What Is Transthyretin Amyloid Cardiomyopathy (ATTR-CM)?
Transthyretin (TTR) is a protein in the body. Proteins in the body have to fold into different shapes in order to do their jobs. Sometimes the TTR protein does not fold the right way or becomes unfolded. This leads to clumps of defective proteins in the body, which can cause serious health problems.
ATTR-CM is a condition where protein clumps build up in the heart, which can cause the heart walls to stiffen over time. This can lead to the heart becoming so weak that it cannot squeeze hard enough to pump blood.
There are two types of ATTR-CM. Familial ATTR-CM is passed down in families. This is sometimes also called "variant" ATTR-CM. Wild-type ATTR-CM is not passed down in families but has been linked to aging and is more common in men.
How Does Attruby Work for ATTR-CM?
Attruby binds to the TTR protein and stabilizes it. This helps prevent it from folding the wrong way or from unfolding, which helps lower the chance of the body forming new clumps over time.
How Was Attruby Studied?
Attruby was studied in 632 adults with ATTR-CM and heart failure. The people in this study were treated with either oral Attruby 800 mg (in the form of acoramidis hydrochloride, which is equivalent to 712 mg acoramidis) or a placebo pill two times daily for 30 months. Placebo pills do not contain an active medicine.
To find out if Attruby worked, the researchers considered four outcomes.
- Whether the people in the study had death from any cause, a heart transplant, or an implanted cardiac mechanical device
- Whether the people in the study were hospitalized because of heart problems
- Changes in a laboratory value called NT-proBNP, which is a protein that is made by your heart and released when your heart is under stress or working harder than usual
- Changes in how far people could walk in six minutes, otherwise known as the six-minute walk distance (6MWD) test
To find out if Attruby was safe, the researchers collected information on what types of side effects people had during the study.
What types of people were included in the study?
The average age of people in this study was about 77 years, and about 90% were male. People in this study were mostly White (88%), about 5% were Black, about 2% were Asian, and about 5% identified as "other."
About 90% had wild-type ATTR-CM, and about 10% had variant ATTR-CM.
A three-stage system is used to classify ATTR based on how severe it is, with stage one being the least advanced and stage three being the most advanced stage. In this study, about 57% of people were in stage one, about 32% were in stage two, and about 11% were in stage three.
A four-class system is used to classify how severe heart failure is, based on heart failure symptoms. This is called the New York Heart Association (NYHA) classification system. Roman numerals are used in this system (I = one, II = two, III = three, and IV = four).
Class | Symptoms |
I | No limits on physical activity. Usual physical activity does not cause symptoms such as fatigue, a fast heartbeat, or shortness of breath. |
II | Slight limits on physical activity, but people are comfortable while resting. Usual physical activity causes symptoms such as fatigue, a fast heartbeat, or shortness of breath. |
III | A lot of limits on physical activity, but people are comfortable at rest. Less-than-usual activity causes symptoms such as fatigue, a fast heartbeat, or shortness of breath. |
IV | Symptoms of heart failure at rest. Any physical activity can cause further discomfort. |
In this study, about 72% had NYHA class II heart failure, about 17% had NYHA class III, and about 11% of people had NYHA class I.
People in this study were not allowed to take tafamidis (Vyndamax or Vyndaqel), which is another drug approved for ATTR-CM, during the first year of the study. But they were allowed to take it after the first year. About 15% of the people who took Attruby and about 23% of the people who took the placebo pill used a tafamidis-containing product. Most people started taking tafamidis within about 17 months after starting the study.
What are the main benefits of Attruby, based on the study?
After 30 months of treatment, the researchers looked at four outcomes to find out how well the drug worked.
- Death from any cause
- Hospitalization for heart-related reasons
- NT-proBNP laboratory values
- Six-minute walk distance
More weight was placed on death from any cause and hospitalization caused by heart problems. Overall, people who were given Attruby did better than those who were given the placebo.
People who were given Attruby also had a better quality of life than those who took the placebo pill.
Your results may differ from what was seen in clinical studies.
What side effects of Attruby were seen in the study?
People who received Attruby were less likely to have a serious side effect than those who received the placebo. About 55% of people who received Attruby had a serious side effect, whereas about 65% of people who received the placebo had a serious side effect.
Side effects were generally similar between the groups. People who received Attruby were more likely to have diarrhea and stomach pain than those who received the placebo pills. About 11.6% of people who received Attruby had diarrhea, and only about 7.6% who received the placebo pills had diarrhea. About 5.5% of people who received Attruby had stomach pain, and only about 1.4% of people who received the placebo pills had stomach pain.
Your results may differ from what was seen in clinical studies.
How Long Until I See the Full Effects of Attruby?
This medicine works by slowing down progression of ATTR-CM. You may not notice improvements, but this medicine may help you live longer and slow functional impairments like being able to walk a certain distance in a given amount of time.
How Do I Manage Side Effects?
If you have diarrhea while taking Attruby, make sure you are drinking plenty of fluids, especially those that contain electrolytes, such as Gatorade or Pedialyte. Clear liquids such as water, broth, or juice can help you stay hydrated.
The BRAT diet (bananas, rice, applesauce, and toast) may also be helpful. Avoid spicy or fatty/fried foods, dairy, citrus, or foods that are high in sugar or caffeine. Let your health care provider know if your diarrhea is severe or lasts longer than a few days.
Is There Any Cost Assistance Available for Attruby?
There may be cost assistance to help you afford Attruby. You may have options even if you do not have health insurance. ForgingBridges is a service that offers one-on-one personalized support to help with access to medicines and coordination of care. Your health care provider can help you get started, or you can call 888-55-BRIDGE (888-552-7434) or visit ForgingBridges.com.
