The current treatment options of transthyretin amyloidosis majorly revolve around relieving patients from symptoms associated with the condition, and treating the underlying cause with a liver transplant. Drugs such as Tafamidis (not approved in the U.S.) and Diflunisal, help in stabilizing the transthyretin tetramer and are majorly used in the treatment of familial amyloid polyneuropathy (FAP). Tafamidis (manufactured by Pfizer, Inc.) is currently in the phase 3 clinical trials in the U.S. Supporting therapies include treatments for heart diseases associated with cardiomyopathy as well as for neuropathy, depending on the severity of the symptoms
Diagnosis of transthyretin amyloidosis is carried out in various ways such as imaging, histopathology, and molecular and genetic testing. Cardiac biomarkers such as NT-proBNP (N-terminal fragment of brain natriuretic peptide) and troponin are present in abnormally high concentrations in the heart, following amyloid deposits that can be tested for disease diagnosis. In case of diagnosis of familial transthyretin amyloidosis, genetic testing for transthyretin gene mutations is carried out.
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Absence of the U.S. Food & Drug Administration (FDA) approved therapies for transthyretin amyloidosis is expected to be a major factor responsible for growth of drugs in the pipeline such as Patisiran and Inotersen (expected to receive approval in 2018). The market is expected to exhibit exponential growth, owing to rapid uptake of approved therapies.
Various other drugs in the pipeline besides Patisiran and Inotersen, such as Tafamidis and ALN-TTRsc02 are expected to boost growth of the market in the near future. These therapies in the pipeline are meant to treat the underlying cause of the disease, which is expected to be a major factor for the previously untreated disease that lacks availability of robust therapy.
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.
Two major therapies that are expected to receive approval in 2018 (Patisiran and Inotersen) are expected to generate yearly cost of six figures (between US$ 200,000 and US$ 400,000). Although, the only approved drug for treatment of familial transthyretin polyneuropathy, Tafamidis, costs around US$ 200,000 a year, it has not received approval in the U.S. Patients may not find the therapy affordable (till reimbursement is favorable) and adoption of the therapy may thus be slower than expected. These factors in turn, are expected to hinder growth of the 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.