The two primary forms of transthyretin amyloidosis are hereditary transthyretin amyloidosis (hATTR) and wild-type transthyretin amyloidosis. Familial Amyloid Polyneuropathy (FAP) and Familial Amyloid Cardiomyopathy (FACM) are two subtypes of hATTR (FAC). The heart is mostly affected by the wild type variation (ATTRwt).
Transthyretin amyloidosis is caused by the liver producing transthyretin, which forms dimers and then monomers. Amyloid fibrils, which are deposited in organs including the heart, neurological system, gastrointestinal tract, and kidneys, are formed when monomers aggregate to create amyloid fibrils. FAP is a kind of hereditary transthyretin amyloidosis, with the Val30Met variation of Transthyretin being the most prevalent cause (TTR). The symptoms of familial amyloid polyneuropathy appear when the patient reaches the age of 30, however it can appear as early as 20 years old or as late as 80 years old. The symptoms of peripheral neuropathy and autonomic neuropathy are different based on their location. If excess amyloid protein begins to build up in nerves, the symptoms may worsen.
Transthyretin amyloidosis treatment with a novel therapy will play a significant role in driving the market growth
For example, Alnylam Pharmaceuticals, Inc. gained FDA clearance in August 2018 for its ONPATTRO (patisiran) lipid complex injectable, an RNA interference (RNAi) therapy, which is intended for the treatment of polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis. The European Commission (EC) granted Ionis Pharmaceuticals, Inc. and Akcea Therapeutics, Inc. marketing authorization clearance for their medication TEGSEDI (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis in July 2018.