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. Author manuscript; available in PMC: 2022 May 1.
Published in final edited form as: Curr Opin Cardiol. 2021 May 1;36(3):309–317. doi: 10.1097/HCO.0000000000000841

Table 2:

Pharmacological therapies for ATTR amyloidosis

Therapy Mechanism of action Route/Dose Indications Adverse effects
Stabilizers
•Tafamidis meglumine Binds and stabilizes TTR, preventing misfolding Oral, 20 or 80 mg daily Cardiomyopathy Potential GI distress but clinical trial suggests 80mg dose similar to placebo
•Tafamidis free salt 61 mg daily
•Diflunisal NSAID, binds and stabilizes TTR, preventing misfolding Oral, 250mg bid Neuropathy Cardiomyopathy

  • GI bleeding

  • Fluid retention

  • Renal dysfunction
•AG-10 (acoramidis) Binds and stabilizes TTR, preventing misfolding Oral, 400 or 800 mg bid Not currently available, in clinical trial  Unknown
Silencers
•Patisiran Small interfering RNA that causes degradation of TTR mRNA IV, 0.3mg/kg every 3 weeks Neuropathy

  • Infusion-related reaction

  • Vitamin A deficiency
•Inotersen Anti-sense oligonucleotide that causes degradation of TTR mRNA SC, 284mg every week Neuropathy

  • Thrombocytopenia and glomerulonephritis, require close monitoring.

  • Infusion-related reaction

  • Vitamin A deficiency

NSAID, non-steroidal anti-inflammatory drug; BID, twice daily; GI, gastrointestinal; IV, intravenous; TID, three times daily; SC, subcutaneous; mRNA, messenger RNA