The U.S. Transthyretin Amyloidosis Treatment Market by Drug (Tafamidis, Diflunisal, Patisiran, Inotersen, and Others), By Disease Type (Hereditary Transthyretin Amyloidosis (Polyneuropathy, Cardiomyopathy, Mixed), and Wild Type), and By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies) is expected to be valued at US$ 36.9 million in 2018 and is projected to exhibit a CAGR of 52.4% over the forecast period (2018–2026). Unavailability of any alternative treatment options, expected drug launches in 2018, and rapid uptake of the newly available therapies are expected to drive U.S. transthyretin amyloidosis treatment market growth.

In 2017, the U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation (BTD) to Patisiran for the treatment of hereditary ATTR (hATTR) amyloidosis with polyneuropathy. Moreover, in November 2017, Alnylam Pharmaceuticals, Inc. initiated a New Drug Application (NDA) for Patisiran and the company announced U.S. FDA acceptance of NDA and priority review status for Patisiran in February 2018.

Manufacturers are actively focused on developing new therapies for the treatment transthyretin amyloidosis. There is no treatment available in the U.S. for transthyretin amyloidosis, which is expected to create lucrative opportunities for manufacturers in the U.S. transthyretin amyloidosis market over the forecast period.

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In January 2018, the new drug application (NDA) of Inotersen was accepted for priority review by the U.S. Food and Drug Administration and a date of July 2018 was set for Prescription Drug User Fee Act (PDUFA), which was later postponed to October 6, 2018. Inotersen launch is expected to further boost the transthyretin amyloidosis treatment market growth.

Browse 22 Market Data Tables and 25 Figures spread through 116 Pages and in-depth TOC on U.S. Transthyretin Amyloidosis Treatment Market by Drug (Tafamidis, Diflunisal, Patisiran, Inotersen, and Others), By Disease Type (Hereditary Transthyretin Amyloidosis (Polyneuropathy, Cardiomyopathy, Mixed), and Wild Type), and By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies) - Forecast to 2026

Key players in the market are focused on collaborations, acquisitions, and partnerships, in order to launch their new therapies into the market. For instance, AKCEA-TTR-LRX, a Ligand Conjugated Antisense (LICA), is being developed by Akcea Therapeutics (affiliate of Ionis Pharmaceuticals), and is expected to enter clinical trials in 2018 for the treatment of all forms of ATTR amyloidosis.

In March 2018, Ionis Pharmaceuticals completed a previously announced transaction with its affiliate Akcea Therapeutics that was related to commercialization rights of Inotersen and AKCEA-TTR-LRX globally (given to Akcea Therapeutics). Alnylam Pharmaceuticals, Inc. developed the drug in collaboration with Sanofi and the deal related to Patisiran was restructured inJanuary 2018.

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Under the new deal, Alnylam regained rights for global development and commercialization of its investigational RNAi therapeutics such as Patisiran and ALN-TTRsc02, whereas Sanofi assumed full responsibility for the development and commercialization of Fitusiran (Hemophilia A and B).

Key Takeaways of the U.S. Transthyretin Amyloidosis Treatment Market:

The U.S. transthyretin amyloidosis treatment market is expected to exhibit a CAGR of 52.4% over the forecast period (2018–2026), owing to product launches by manufacturers such as Alnylam Pharmaceuticals, Inc., and Ionis Therapeutics

Patisiran (Alnylam Pharmaceuticals, Inc.) is currently ahead of its competitors in terms of approval and subsequent commercialization, and Alnylam may benefit from the first-mover advantage

Therapies in the pipeline belong to the class of gene therapies (RNAi therapeutics and antisense drugs) which have the potential to cure the underlying cause of the disease

Major players operating in the U.S. transthyretin amyloidosis treatment market include 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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