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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