Understanding Amvuttra and Amyloidosis

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Understanding Amvuttra and Amyloidosis

Amyloidosis is a rare condition that can feel overwhelming—especially with names like “hereditary transthyretin-mediated amyloidosis” and “ATTR-cardiomyopathy.” But let’s break it down simply, so you can understand what’s happening in your body and how Amvuttra may help.


What Is Amyloidosis?

Amyloidosis happens when abnormal proteins—called amyloid—build up in your organs and tissues. These proteins shouldn’t be there, and over time, they can interfere with how your body works.

One specific type of amyloidosis involves a protein called transthyretin (TTR), which normally helps carry vitamin A and thyroid hormone through your bloodstream. But when TTR becomes unstable, it can misfold and clump together, forming amyloid deposits.


Two Main Types of TTR Amyloidosis

There are two major forms of transthyretin amyloidosis that Amvuttra treats:

1. hATTR Polyneuropathy (Hereditary ATTR)

  • This form is inherited—passed down through families.

  • It mainly affects the nerves, causing symptoms like:

    • Numbness or tingling in the hands and feet

    • Muscle weakness

    • Digestive issues

    • Dizziness or fainting

  • It’s progressive, meaning symptoms can worsen over time.

2. ATTR-CM (ATTR Cardiomyopathy)

  • This form affects the heart and can be either hereditary or “wild-type” (not inherited).

  • It causes the heart walls to stiffen, making it harder to pump blood.

  • Symptoms may include:

    • Shortness of breath

    • Swelling in the legs

    • Fatigue

    • Irregular heartbeat

Both types are serious, but they’re treatable—and that’s where Amvuttra comes in.


What Is Amvuttra?

Amvuttra (generic name: vutrisiran) is a medication designed to reduce the production of abnormal TTR protein. It’s given as a small injection under the skin every three months by a healthcare provider.

Think of Amvuttra as a “gene silencer.” It tells your liver to make less of the faulty TTR protein, which means fewer amyloid deposits and less damage to your nerves or heart.


FDA-Approved for Two Conditions

Amvuttra was originally approved to treat hATTR polyneuropathy. But in 2024, the FDA expanded its use to include ATTR cardiomyopathy as well. That means it’s now the only approved medication that treats both nerve and heart complications caused by TTR amyloidosis.

In clinical trials, patients taking Amvuttra showed:

  • Fewer hospitalizations for heart failure

  • Improved ability to walk and perform daily activities

  • Slower progression of nerve damage

  • Better overall quality of life


What This Means for You

If you or a loved one has been diagnosed with either form of TTR amyloidosis, Amvuttra may offer a way to slow the disease and improve symptoms. It’s not a cure, but it’s a powerful tool to help manage this complex condition.

And while the diagnosis may sound intimidating, you’re not alone. There are care teams, support groups, and new treatments like Amvuttra that are changing the outlook for patients every day.

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