Table 1.

Treatments for polyneuropathy of hATTR

	Inotersen [24, 25]	Patisiran [22, 23]	Tafamidis [18, 29]	Diflunisal [20, 36]
Agent type	ASO	siRNA	Stabilizer	Nonsteroidal anti-inflammatory drug
Indication	Approved for ATTR-PN in the United States and for stage 1 or 2 hATTR-PN in the European Union, Canada, Brazil, and other countriesa	Approved for ATTR-PN in the United States, Canada, and Japan and for stage 1 or 2 hATTR-PN in the European Union, Brazil, and Switzerland	Approved in the European Union (stage 1) and in over 40 countriesb	Off-label
Dose	284 mg subcutaneously once weekly	0.3 mg/kg intravenously every 3 weeks	20 mg orally once daily	250–1000 mg daily
Premedication	None	Intravenous corticosteroid Oral acetaminophen Intravenous H1 blocker Intravenous H2 blocker	None	None
Precautions	Vitamin A supplementation	Vitamin A supplementation	None	None
Safety monitoring	Thrombocytopenia Glomerulonephritis Liver function	Infusion-related reactions	None	Serious cardiovascular thrombotic events, myocardial infarction, stroke, and serious gastrointestinal adverse events including bleeding, ulceration, and perforation of the stomach or intestines

ASO antisense oligonucleotide, ATTR transthyretin amyloidosis, ATTR-PN transthyretin-mediated amyloid polyneuropathy, hATTR hereditary transthyretin amyloidosis, hATTR-PN hereditary transthyretin-mediated amyloid polyneuropathy, siRNA small interfering ribonucleic acid

^aIceland, Liechtenstein, Norway, United Kingdom (license issued based on European Medicines Agency)

^bApproved for ATTR-PN in over 40 countries, including Japan, European countries, Brazil, Mexico, Argentina, Israel, Russia, and South Korea