Treatments for ATTR amyloidosis include stabilisers, gene-silencing therapies and gene-editing therapy. We try to keep this list updated though new treatments may not be listed here. Please consult with the medical professionals treating you.
Stabilisers
The transthyretin protein breaking apart is the initial step in forming amyloid deposits. A stabiliser binds to the TTR protein, prevents it from breaking apart and reduces the level of amyloid formation.
TAFAMIDIS from Pfizer
Tafamidis (Vyndaqel) was the first drug to be prescribed for cardiac amyloidosis. It inhibits amyloid formation, thereby delaying the development of nerve and cardiac muscle damage caused by transthyretin amyloidosis.
It is orally administered and available only on prescription.
DIFLUNISAL from Merck & Co
Diflunisal is a non-steroidal, anti-inflammatory drug (NSAID). This class of drugs is commonly used as painkillers for conditions such as rheumatoid arthritis.
Diflunisal is one of the first examples of repurposing of a generic drug to treat a rare disease. Previously under the brand name Dolobid, generic versions are now widely available.
It is orally administered and available only on prescription.
ACORAMIDIS (AG10) from BridgeBio
Acoramidis is an orally administered TTR stabiliser currently being developed.
Gene-silencers
PATISIRAN from Alnylam
PATISIRAN is a transthyretin-directed small interfering RNA used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis.
VUTRISIRAN
Vutrisiran may be the second-generation formulation of Patisiran. It involves a subcutaneous injection self-administered once every three months.
INOTERSEN from Sobi
Inotersen (Tegsedi), discovered and developed by Ionis Pharmaceuticals, is the world’s first subcutaneous RNA-targeting drug. It involves a subcutaneous injection self-administered once weekly. It requires ongoing monitoring of platelet counts.
EPLONTERSEN
Eplontersen may be the second-generation formulation of Inotersen and involves a subcutaneous injection self-administered once every four weeks.
Gene-editing therapies
CRISPR-Cas9 from Intellia
CRISPR/Cas9 edits genes by precisely cutting DNA and then letting natural DNA repair processes to take over.
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