Table 3:

Emerging therapies for ATTR-CM

Drug Name	Mechanism of Action	Indication	Route of Administration	Dose	Common, Serious or Potential Side Effects	Concomitant Therapy	Monitoring	Approval
Patisiran	Silencer	Neuropathy	Intravenous	0.3 mg/kg q3weeks up to 30 mg	Infusion related reactions Vitamin A deficiency	With IV infusion: Intravenous corticosteroid (e.g., dexamethaso ne 10 mg, or equivalent) Oral acetaminophe n (500 mg) Intravenous H1 blocker (e.g., diphenhydra mine 50 mg, or equivalent) Intravenous H2 blocker (e.g., ranitidine 50 mg, or equivalent) Daily Vitamin A supplements	None	Approved in US and Europe
Inotersen	Silencer	Neuropathy	Subcutaneous	284 mg q week	Thrombocytopenia/glomerulonephritis, requiring testing before treatment and monitoring during therapy Infusion site reactions, fever Vitamin A deficiency	Daily Vitamin A supplements	Weekly platelet counts. Every 2 weeks measures of serum creatinine, eGFR urinalysis, and UPCR.	Approved in US and Europe
Tafamidis meglumine	Stabilizer	Neuropathy	Oral	20 mg	Reported in hATTR polyneuropathy; Diarrhea UTI Vaginal infection Stomach ache or abdominal pain	None	None	In Europe and Japan
		Cardiomyopathy		20 mg or 80 mg	Side effects were less common than with placebo in cardiomyopathy	None	None	Anticipated 2019
Tafamidis free salt	Stabilizer	Cardiomyopathy	Oral	61 mg	Unknown	None	None	Anticipated 2019
Diflunisal	Stabilizer	Neuropathy Cardiomyopathy	Oral	250 mg PO BID	Related to NSAID properties: Bleeding Hypertension Fluid Retention Renal dysfunction	Proton pump inhibitor	Monitor renal function, platelet count, hemoglobin 1 week after initiation and then every 3–6 months	Approved in US and Europe, off label usage

UPCR: urine protein to creatinine ratio, NSAID: Non-steroidal anti-inflammatory drug