Transthyretin-Related Amyloidosis Medication

Updated: Jul 22, 2020
  • Author: Jefferson R Roberts, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Medication

Medication Summary

Despite recent drug approvals, liver transplantation remains the gold standard for treating transthyretin-related amyloidosis (ATTR). Multi-organ transplantation (heart, liver and kidney) has been successful in slowing the natural course of the disease. In 2018, the US Food and Drug Administration (FDA) approved patisiran and inotersen for treatment of polyneuropathy caused by hereditary ATTR (hATTR) in adults. [40, 42] In 2019, the FDA approved tafamidis and tafamidis meglumine for ATTR cardiomyopathy in adults. [44] In Europe, tafamidis is approved for stage I hATTR. Research continues to identify and refine effective medical treatments for preventing the deposition of amyloid fibrils.

 

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siRNA Agents

Class Summary

Anti-transthyretin small interfering ribonucleic acid (siRNA) agents causes degradation of mutant and wild-type TTR mRNA through RNA interference.

Patisiran (Onpattro)

Patisiran is an siRNA agent that reduces serum transthyretin (TTR) protein and TTR protein deposits in tissues. It is indicated for treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults.

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Antisense Oligonucleotides

Class Summary

In a clinical trial, inotersen, an antisense oligonucleotide, has improved neurological scores and therefore less neurologic decline compared with placebo. [42, 57]  

Inotersen (Tegsedi)

Inotersen is an antisense oligonucleotide that causes degradation of mutant and wild-type transthyretin mRNA by binding TTR mRNA. This action results in reduced TTR protein in serum and tissue. It is indicated for polyneuropathy of hATTR in adults.

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Cardiovascular, Other

Tafamidis (Vyndamax)

Selectively binds to transthyretin tetramer to prevent transthyretin transport protein destabilization and amyloid formation that causes ATTR-CM

Tafamidis meglumine

Selectively binds to transthyretin tetramer to prevent transthyretin transport protein destabilization and amyloid formation that causes ATTR-CM

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