Two main types of transthyretin amyloidosis are hereditary transthyretin amyloidosis and wild type transthyretin amyloidosis. Hereditary transthyretin amyloidosis (hATTR) is further classified into familial amyloid polyneuropathy (FAP), and familial amyloid cardiomyopathy (FAC). Whereas, wild type variant (ATTRwt) predominantly affects the heart.
Transthyretin amyloidosis results from transthyretin produced by the liver, which forms dimers and then monomers. These monomers aggregate to form amyloid fibrils, which get deposited in multiple organs such as heart, nervous system, gastrointestinal tract, and kidneys. Familial amyloid polyneuropathy (FAP) is a subtype of hereditary transthyretin amyloidosis (hATTR), and the most common type of FAP is caused by the Val30Met variant of TTR.
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In familial amyloid polyneuropathy, the symptoms are first detected after the patient crosses 30 years of age, however, it can also be detected as early as 20 years or as late as 80 years of age. Symptoms are divided depending on the location such as peripheral neuropathy and autonomic neuropathy. Symptoms may worsen in case excess amyloid protein starts to collect in the nerves.
Available therapies for treatment of transthyretin amyloidosis such as Tafamidis (not approved in the U.S.), Diflunisal (off-label indication), and other therapies are supportive treatment options and only treat symptoms of the disease.
Liver transplant is the only curative option in case of familial transthyretin polyneuropathy, and is generally not used in case of familial transthyretin cardiomyopathy or wild type TTR. New therapies that are expected to receive approval (Patisiran and Inotersen) have a novel therapeutic action known as gene silencing and work by either interfering with the abnormal transthyretin formation or by silencing the gene (antisense) responsible for production of transthyretin protein.
Major players in the market have novel drugs in the pipeline, which are in late-stage clinical trials and are expected to receive approval in the near future. For instance, Alnylam Pharmaceuticals, Inc. and Ionis Pharmaceuticals, Inc. developed Patisiran and Inotersen, respectively, which are expected to receive approval by the U.S. Food & Drug Administration (FDA) in 2018.
In March 2018, Pfizer, Inc. reported positive results from Phase 3 ATTR-ACT study of Tafamidis among patients suffering from transthyretin cardiomyopathy. Alnylam Pharmaceuticals, Inc. is also expected to initiate phase 3 clinical trials for another drug, ALN-TTRsc02, by 2018 (between Q3 and Q4). This drug is primarily indicated for the treatment of ATTR amyloidosis. Transthyretin amyloidosis is a rare disease and treatment for the same is not available yet, however, is expected to be made available by 2018. Robust pipeline of drugs for treatment of transthyretin amyloidosis is expected to significantly drive growth of the U.S. transthyretin amyloidosis market over the forecast period.
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Some major players operating in the U.S. transthyretin amyloidosis treatment market are Alnylam Pharmaceuticals, Inc., Pfizer, Inc., Prothena Corporation Plc, GlaxoSmithKline Plc, Ionis Pharmaceuticals, Inc., Eidos Therapeutics, and SOM Innovation Biotech, S.L.
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