Transthyretin amyloid polyneuropathy, also known as transthyretin-related hereditary amyloidosis, is a rare inherited, autosomal-dominant, adult-onset, and slowly neurodegenerative progressive disease. Transthyretin amyloid polyneuropathy is characterized by the extracellular deposition of amyloid fibrils, most commonly due to mutated transthyretin (TTR). TTR gene mutations lead to destabilization and dissociation of TTR tetramers into variant TTR monomers, which form amyloid fibrils that deposit in peripheral nerves and various organs, giving rise to peripheral and autonomic neuropathy and several nondisease specific symptoms. The penetrance and age at onset of transthyretin amyloid polyneuropathy among people carrying the same mutation vary between countries. The symptomatology and clinical course of transthyretin amyloid polyneuropathy can be highly variable.
According to reports published on transthyretin amyloidosis, it has been estimated that nearly 1.1 per 100,000 individuals in the world are living with transthyretin amyloid polyneuropathy. The age group of patients suffering from the disorder is between 30 and 40 years of age. Familial amyloid polyneuropathy (TTR-FAP) leads to 100 different types of mutations in the transthyretin gene, which leads to protein misfolding. There is only a 50% chance of transferring the mutation to the next generation from an affected parent. The global transthyretin amyloid polyneuropathy therapeutics market is likely to be driven by an increase in the prevalence of disease, increase in awareness, improvement in diagnostic procedures, improving health care services, rapid economic growth in developing countries, and rise in research and development activity. However, major restraining factors for the transthyretin amyloid polyneuropathy therapeutics market are less awareness in developing economies, misdiagnosis of the disease, high cost associated with disease diagnosis and treatment, limited clinical trials, and lack of efficient medication.
The transthyretin amyloid polyneuropathy therapeutics can be segmented based on treatment, end-user, and region. In terms of treatment, the market can be classified into drug therapy and surgery. GlaxoSmithKline (GSK) is currently in Phase II of development of a novel therapy for systemic amyloidosis, which directly targets the amyloid deposits themselves through a mechanism directed toward serum amyloid P component (SAP). Based on end-user, the market can be classified into hospital pharmacies, retail pharmacies, and online pharmacies.
In terms of region, the transthyretin amyloid polyneuropathy therapeutics market can be segmented into North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America dominates the global transthyretin amyloid polyneuropathy therapeutics market due to new product innovation, high health care expenditure, and government awareness programs. In March 2018, Ionis Pharmaceuticals, Inc. and its affiliate, Akcea Therapeutics, Inc. announced Phase III clinical data showing that inotersen-treated patients with hereditary ATTR (hATTR) amyloidosis who were treated for up to 27 months in the NEURO-TTR and open-label extension (OLE) studies continued to demonstrate sustained benefit in measures of quality of life and neuropathy. Europe is a prominent region of the transthyretin amyloid polyneuropathy therapeutics market. Within Europe, the incidence of TTR-FAP is highly variable. There are large areas in Portugal and Sweden in which the disease is endemic and is localized to one small area, with a traceable family history with a single genetic mutation. The market in Asia Pacific is expected to expand at a considerable rate due to a rapid rise in population, rise in the prevalence of chronic diseases, increase in health care coverage, and rise in investment in research and development. Emerging regions such as Latin America and Middle East & Africa are expected to offer minimal growth opportunities to the global transthyretin amyloid polyneuropathy therapeutics market due to low awareness among people.
Major players operating in the global transthyretin amyloid polyneuropathy therapeutics market include Pfizer, Inc., GlaxoSmithKline Plc., Corino Therapeutics Inc., Ionis Pharmaceuticals Inc., Ltd., Eidos Therapeutics, Inc., Alnylam Pharmaceuticals, Prothena Corporation plc, and Regeneron Pharmaceuticals, Inc.. Approvals and launches of novel therapies for the treatment of transthyretin amyloid polyneuropathy is expected to be a major factor driving the market over the forecast period. For instance, RNAi Therapeutics for transthyretin amyloid polyneuropathy by Alnylam Pharmaceuticals. A robust drug pipeline is expected to boost the transthyretin amyloid polyneuropathy therapeutics market during the forecast period.
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