Amyloidosis Ireland

Treatments

Treatments for ATTR amyloidosis include stabilisers, gene-silencing therapies and gene-editing approaches.

Please check with your clinical team

Treatment availability changes over time and varies by country, diagnosis and individual circumstances. This page is not medical advice. Always discuss treatment decisions with the doctors and specialists treating you.

At a glance

  • Stabilisers aim to stop the TTR protein breaking apart.
  • Gene-silencing therapies aim to reduce production of TTR protein.
  • Gene-editing therapies are being studied as a way to alter the TTR gene in liver cells.

Stabilisers

The transthyretin protein breaking apart is the initial step in forming amyloid deposits. A stabiliser binds to the TTR protein, helps prevent it breaking apart and reduces amyloid formation.

Tafamidis Vyndaqel · Pfizer
An oral prescription medicine used in cardiac ATTR amyloidosis.
Diflunisal Merck & Co
A repurposed non-steroidal anti-inflammatory drug, available only on prescription.
Acoramidis AG10 · BridgeBio
An oral TTR stabiliser currently in development.

Gene-silencing therapies

Gene-silencing therapies reduce the amount of transthyretin protein the body produces, limiting further amyloid build-up.

Patisiran Alnylam
A transthyretin-directed small interfering RNA used for polyneuropathy of hereditary ATTR amyloidosis.
Vutrisiran Alnylam
A second-generation formulation related to Patisiran, given by subcutaneous injection every three months.
Inotersen Tegsedi · Sobi
A weekly subcutaneous RNA-targeting drug requiring ongoing platelet monitoring.
Eplontersen AstraZeneca / Ionis
A second-generation formulation related to Inotersen, given by subcutaneous injection every four weeks.

Gene-editing therapies

Gene-editing approaches are designed to edit the TTR gene in liver cells — a potential one-time treatment rather than ongoing management.

CRISPR-Cas9 Intellia Therapeutics
An investigational in-vivo gene-editing approach currently in clinical trials for ATTR amyloidosis.

Questions worth asking your clinical team

  • Which type of amyloidosis do I have?
  • Which organs or nerves are affected?
  • What is the goal of this treatment for me?
  • How will we monitor whether treatment is working?
  • Who should I contact if symptoms change?

Next: types of amyloidosis

Learn the difference between hereditary ATTR, wild-type ATTR and other amyloidosis types.

Read types of amyloidosis