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In May 2019, the FDA approved two tafamidis preparations for the treatment of transthyretin-mediated cardiomyopathy, but has not approved it for the treatment of transthyretin familial amyloid polyneuropathy. [15] In August 2018, the FDA approved patisiran, an siRNA-based treatment, at an expected cost of up to $450,000 per year. [16]
Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis. [3] It is a transthyretin-directed antisense oligonucleotide. [3] It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca. [4] [5] [6] [7]
Tafamidis was approved in the European Union in 2011 for the treatment of transthyretin amyloidosis with polyneuropathy, and in Japan in 2013. [6] [17] In the United States, it was rejected for the treatment of transthyretin amyloidosis with polyneuropathy because the Food and Drug Administration saw insufficient evidence for its efficacy. [20] [7]
The familial amyloid neuropathies (or familial amyloidotic neuropathies, neuropathic heredofamilial amyloidosis, familial amyloid polyneuropathy) are a rare group of autosomal dominant diseases wherein the autonomic nervous system and/or other nerves are compromised by protein aggregation and/or amyloid fibril formation. [1] [2] [3]
Treatments aimed at symptom relief are available, and include diuretics, pacemakers, and arrhythmia management. Thus, Senile systemic amyloidosis and familial amyloid polyneuropathy are often treatable diseases that are misdiagnosed. [9] [10] [11]
Vutrisiran, sold under the brand name Amvuttra, is a medication used for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. [ 7 ] [ 5 ] [ 8 ] It is a double stranded small interfering RNA (siRNA) (also called RNA interference, or RNAi therapeutic) that interferes with the expression of the ...
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