TTR Stabilizers

Living with ATTR amyloidosis means managing a complex condition. This condition involves a protein in your body called transthyretin, or TTR. Normally, this protein does its job without any problems. However, in ATTR amyloidosis, this protein becomes unstable – it breaks apart and builds up in organs like the heart and nerves.¹

This buildup can cause serious health issues. There are different ways to treat ATTR amyloidosis. One important treatment is a class of drugs called TTR stabilizers.¹

How do TTR stabilizers work?

Your body makes the TTR protein mostly in the liver. Its normal job is to carry thyroid hormone and vitamin A through your blood. To do this, the TTR protein forms a shape with 4 parts. This shape is called a tetramer.^2,3

In ATTR amyloidosis, this 4-part tetramer becomes unstable. It breaks apart into single pieces, called monomers. These monomers then misfold, or change into the wrong shape.^2,3

These misfolded pieces stick together. They form hard, damaging clumps called amyloid fibrils. These fibrils build up in your organs. When they build up in the heart, it is called ATTR cardiomyopathy, or ATTR-CM. This buildup makes the heart muscle stiff and weak.^1-3

TTR stabilizers are drugs that help stop this process. They work by attaching to the TTR protein. This binding helps hold the 4-part tetramer together. It makes the protein stable, so it is less likely to break apart.^2,3

If the protein does not break apart, it cannot form the harmful amyloid fibrils. This action is key to the treatment. TTR stabilizers can help slow down the progression of the disease.^2,3

Examples of TTR stabilizers

The US Food and Drug Administration (FDA) has approved 2 TTR stabilizers for the treatment of the cardiomyopathy of hereditary or wild-type ATTR amyloidosis (ATTR-CM) in adults:^2-5

• Vyndaqel® and Vyndamax® (tafamidis)
• Attruby® (acoramidis)

Vyndaqel® and Vyndamax® (tafamidis)

These 2 brand names contain the same active drug: tafamidis. The FDA has approved tafamidis for adults with ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN). It is shown to reduce death and hospital stays related to heart problems, improving overall quality of life.^2-4

Attruby® (acoramidis)

Attruby contains the active drug acoramidis. The FDA approved Attruby in November 2024. It is also approved for adults with ATTR-CM.^2-5

Like tafamidis, acoramidis helps reduce cardiovascular death and hospitalizations. It was designed to mimic a natural, very stable version of the TTR protein.^2-5

What are the possible side effects?

Side effects can vary depending on the specific drug you are taking.^4,5

For tafamidis, there are no known side effects. However, before taking tafamidis, tell your healthcare provider about all your medical conditions, including if you:⁴

• Have liver problems
• Are pregnant or plan to become pregnant – tafamidis may harm an unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with either of these drugs.

For acoramidis, the side effects may include:⁵

• Gastrointestinal, or stomach, issues
• Upper abdominal pain

These are not all the possible side effects of TTR stabilizers. Talk to your doctor about what to expect when taking these drugs. You should also call your doctor if you have any changes that concern you when taking these drugs.

Other things to know

These drugs are taken as pills. You should swallow the capsules or tablets whole. Do not cut, crush, or chew them. Your doctor will prescribe the correct dose for you.^4,5

TTR stabilizers can sometimes interact with other medicines. For example, tafamidis may interact with drugs like methotrexate or rosuvastatin. Acoramidis may change how your body processes certain other drugs.^4,5

Tell your doctor if you are pregnant or plan to become pregnant. Tafamidis may harm an unborn baby. You should use effective birth control while taking it. It is not known if acoramidis is safe to use during pregnancy.^4,5

Before beginning treatment for ATTR amyloidosis, tell your doctor about all your health conditions and any other drugs, vitamins, or supplements you take. This includes over-the-counter drugs.