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 estimated to be valued at US$ 50.7 Million in 2022 and is expected to exhibit a CAGR of 9.0% during the forecast period (2022-2030), as highlighted in a new report published by Coherent Market Insights.
Unavailability of any alternative treatment options, expected to launch drugs and rapid uptake of the newly available therapies are expected to drive U.S. transthyretin amyloidosis treatment market growth. For instance, 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 had announced the U.S. FDA acceptance of NDA and priority review status for Patisiran in February 2018.
U.S. Transthyretin Amyloidosis Treatment Market – Impact of Coronavirus (COVID-19) Pandemic
The COVID-19 pandemic and lockdowns in various countries across the globe have impacted the financial status of businesses across all sectors, including the private healthcare sector. The COVID-19 pandemic has impacted the entire supply chain of the healthcare industry, mainly due to strict lockdown in several regions. Private healthcare is one such sectors that has been impacted significantly by the COVID-19 pandemic.
However, the COVID-19 pandemic had a negative impact on the U.S. transthyretin amyloidosis treatment market, owing to reduction in clinical trials for drugs used in treatment of transthyretin amyloidosis during Covid-19 pandemic. For instance, in September 2020, according to data published by National Center for Biotechnology Information, due to the sudden onset and widespread impact of COVID-19, its influence on managing clinical trials and research remains undetermined. Coronavirus mitigation efforts (self-isolation) interfere with all aspects of clinical trials at multiple levels. Interruption of supply chains and monitoring of clinical trials are additional obstacles. Conduction of clinical trials may require modifications to ensure the rights, safety, and wellbeing of participants as well as medical staff. The ongoing COVID-19 pandemic has a potentially negative impact on the management of clinical trials, which may compromise the scientific integrity of data and may raise concerns for patient safety. Moreover, there is an unprecedented operational burden on trials’ conduction as there is limited access to trial activities and investigations of novel therapies or interventions for various diseases, particularly involving vulnerable populations. The U.S. Food and Drug Administration issued guidelines for conducting clinical trials focusing on this current pandemic which should be followed by research teams currently involved in clinical trial programs.
U.S. Transthyretin Amyloidosis Treatment Market: Key Developments
On July 6 2022, Alnylam Pharmaceuticals Inc., a U.S.-based biopharmaceutical company, launched its new product AMVUTTRA (vutrisiran), an RNAi therapeutic indicated for the treatment of polyneuropathy caused by hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The drug is available in 25mg/0.5ml strength as a clear, colorless-to-yellow solution in a single-dose prefilled syringe for subcutaneous administration once every three months.
Browse 22 Market Data Tables and 25 Figures spread through 116 Pages and in-depth TOC on “U.S. Transthyretin Amyloidosis Treatment Market ”- Forecast to 2030, 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)
To know the latest trends and insights prevalent in the U.S. Transthyretin Amyloidosis Treatment Market, click the link below:
Key Takeaways of the U.S. Transthyretin Amyloidosis Treatment Market:
- The U.S. transthyretin amyloidosis treatment market is expected to exhibit a CAGR of 9.0% during the forecast period due to key players 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. For instance, 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.
- Key players in the market are focused on collaborations, acquisitions, and partnerships, in order to launch their new therapies in the market. For instance, AKCEA-TTR-LRX, a Ligand Conjugated Antisense (LICA), is being developed by Akcea Therapeutics (affiliate of Ionis Pharmaceuticals), and is has cleared clinical trial 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 in January 2018. 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).
- 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.
