By 2026, the U.S. Transthyretin Amyloidosis Treatment Market is expected to reach a value of $1,077.7million.

Transthyretin (TTR) amyloidosis is a slowly progressing disease in which abnormal deposits of a protein called amyloid (amyloidosis) form in the body's organs and tissues. Hereditary transthyretin amyloidosis (hATTR) and wild-type transthyretin amyloidosis are the two main kinds of transthyretin amyloidosis (ATTRwt). Transthyretin amyloidosis is an uncommon condition with limited treatment options.

Genetic mutations in the genes responsible for transthyretin production cause hATTR. Familial amyloid polyneuropathy (FAP) and familial amyloid cardiomyopathy (FACM) are two subtypes (FAC). The heart is primarily affected by ATTRwt. The Val30Met variation of TTR is the most common cause of FAP. After the patient reaches the age of 30, the signs of FAP become apparent.

The symptoms might appear as young as 20 years old or as late as 80 years old. The symptoms are categorized as peripheral neuropathy or autonomic neuropathy depending on where they occur. When extra amyloid protein builds up in the nerves, the symptoms grow more severe.

Some supportive treatment options for transthyretin amyloidosis include Tafamidis (not licensed in the United States) and Diflunisal (off-label indication), which solely treat symptoms of the condition. For familial transthyretin polyneuropathy, doctors advocate a liver transplant as a viable treatment option. For hereditary transthyretin cardiomyopathy or wild-type TTR, this method is not suggested.

The  U.S. Transthyretin  Amyloidosis Treatment Market is estimated to reach US$ 36.9 million in 2018, with a CAGR of 52.4 percent over the forecast period (2018–2026).

The market's growth is also likely to be aided by a strong pipeline of innovative medications. Patisiran from Alnylam Pharmaceuticals, Inc. and Inotersen from Ionis Pharmaceuticals, Inc., for example, are likely to be approved by the US Food and Drug Administration in 2018. (FDA). These treatments are based on gene silencing and work by either interfering with aberrant transthyretin formation or silencing the gene responsible for transthyretin protein production (antisense). Patisiran and Inotersen are estimated to produce six-figure annual costs ranging from US$ 200,000 to US$ 400,000.

Over the projection period, the market's expansion is expected to be hampered by the high cost of therapies. Tafamidis, for example, is used to treat familial transthyretin polyneuropathy and costs over $200,000 per year.

Alnylam Pharmaceuticals, Inc., Pfizer, Inc., Prothena Corporation Plc, GlaxoSmithKline Plc, Ionis Pharmaceuticals, Inc., Eidos Therapeutics, and SOM Innovation Biotech, S.L. are among the major participants in the transthyretin amyloidosis therapy market in the United States.