What treatments are available for patients with ATTR amyloidosis?

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What treatments are available for patients with ATTR amyloidosis?

Several medications have been approved by the Food and Drug Administration (FDA) for treating patients with ATTR amyloidosis. Other medications continue to be investigated.

  • ATTR silencers. These medications act on the liver to decrease the production of TTR. Two ATTR silencers approved by the FDA to treat patients with the hereditary type of ATTR who also have neuropathy.
    • Patisiran (Onpattro®) is an infusion that is given every three weeks.
    • Inotersen (Tegsedi®) is an injection given once a week and requires weekly lab work.
  • ATTR stabilizers. These medications stabilize the TTR protein, which in turn prevents it from breaking apart and forming amyloid fibrils.
    • Tafamidis (Vyndamax®, Vyndaqel®) is approved by the FDA for patients with hereditary or wild-type ATTR that has affected their heart.
    • AG10 is a medication currently being tested in a clinical trial.
    • Diflunsial (Dolobid®) is a nonsteroidal anti-inflammatory drug (NSAID) that has been shown to also stabilize the TTR protein. However, this medication has not been fully studied in patients with ATTR that has affected the heart and also may not be tolerated due to side effects.
  • Fibril disruptors. These medications may help break up and clear ATTR amyloid fibrils. Doxycycline (antibiotic) and green tea extract (over-the-counter supplement)have only been tested in small studies and there is limited evidence that these medications would be helpful in treating amyloidosis.

An antibody that removes TTR amylod fibrils, called PRX-004, is being tested in clinical trials.

Because TTR is made in the liver, a liver transplant is also a possible treatment for patients with some hereditary forms of ATTR amyloidosis, but not for those with wild-type form.

Your treatment team will likely include:

  • Cardiologist: A doctor that specializes in the heart.
  • Gastroenterologist: A doctor who specializes in the gastrointestinal tract.
  • Genetic Counselor: A healthcare worker who helps patients and family members understand the risk of inheriting certain medical conditions.
  • Hematologist: A doctor that specializes in blood problems.
  • Nephrologist: A doctor that specializes in the kidneys.
  • Neurologist: A doctor that specializes in the nerves.

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