Us transthyretin amyloidosis treatment market

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 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.

The U.S. transthyretin amyloidosis treatment market 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).

Figure 1. U.S. Transthyretin Amyloidosis Treatment Market Share (%), by Disease Type, 2022

Introduction of novel therapies is expected to boost growth of the U.S. transthyretin amyloidosis treatment market

Introduction of novel therapies is expected to boost growth of the U.S.  Transthyretin amyloidosis treatment market. For instance, in October 2018, according to data published by PubMed novel, therapies using RNA interference also offer clinical promise to treat amyloidosis. Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin. In 2018, patisiran was the first-ever RNA interference (RNAi)-based drug approved by the U.S. Food and Drug Administration. The data across phase I-III clinical trials points to patisiran as an effective and safe drug for the treatment of hereditary transthyretin amyloidosis (hATTR). It is anticipated that real-world data from a larger number of patients treated with patisiran will confirm the effectiveness of this first-approved siRNA-based drug.

Figure 2. U.S. Transthyretin Amyloidosis Treatment Market Share (%), by Drug, 2022

Increasing approval of drugs used in treatment of transthyretin amyloidosis is expected to boost market growth over the forecast period

Major players in the market are engaged in research and development activities for development transthyretin amyloidosis treatment. For instance, in May 2019, Pfizer Inc., multinational pharmaceutical and Biotechnology Corporation based in the U.S. manufactured Vyndaqel and Vyndamax capsules as the first treatment for ATTR-CM. The U.S. Food and Drug Administration (FDA) has approved both VYNDAQEL® (tafamidis meglumine) and VYNDAMAX™ (tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are two oral formulations of the first-in-class transthyretin stabilizer tafamidis, and the first and only medicines approved by the FDA to treat ATTR-CM.

U.S. Transthyretin Amyloidosis Treatment Market-Impact of Coronavirus (COVID-19) Pandemic

Since the COVID-19 outbreak in December 2019, the disease has spread to over 100 countries across the globe and the World Health Organization had declared it a public health emergency on January 30, 2020.

COVID-19 can affect the economy in three main ways: by directly affecting production and demand of drugs, by creating disruptions in distribution channels, and through its financial impact on firms and financial markets. Due to nationwide lockdowns, several countries, such as China, India, Saudi Arabia, U.A.E., Egypt, and others, are facing problems with regards to the transportation of drugs from one place to another.

However, the COVID-19 pandemic had a negative impact on the U.S. transthyretin amyloidosis treatment market, owing to decrease demand for the U.S. transthyretin amyloidosis treatment due to research and development for drugs to be used in the treatment of coronavirus which was on priority. For instance, in April 2020, pandemic associated with the coronavirus disease 2019 (COVID-19) syndrome, which is caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection, has rapidly become a serious threat to global public health. The impact of SARS-CoV-2 infection on patients with most chronic conditions is largely unknown. The pandemic has significantly impacted care for chronic disease by causing a shift in healthcare resources to manage acutely ill patients with COVID-19 with subsequent implementation of telemedicine for many other cases.

U.S. Transthyretin Amyloidosis Treatment Market: Key Developments

In June 22 2022, AstraZeneca plc., a multinational pharmaceutical and biotechnology company, entered into a new global development and agreement with Ionis Pharmaceuticals, Inc., a biotechnology company, for eplontersen, formerly known as IONIS-TTR-L. Eplontersen is a ligand-conjugated antisense investigational medicine currently in Phase III clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN). It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR).

U.S. Transthyretin Amyloidosis Treatment Market: Restraint

The major factors that hinder growth of the U.S. transthyretin amyloidosis treatment market include high cost of U.S. transthyretin amyloidosis treatment. The only approved drug for treatment of familial transthyretin polyneuropathy, Tafamidis, costs around US$ 200,000 a year, although, it has not received approval in the U.S. Patients may not find the therapy affordable (till reimbursement is favorable). This is one of the major factors that is expected to hamper growth of the market over the forecast period.

Key Players

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.

Detailed Segmentation:

• U.S. Transthyretin Amyloidosis Treatment Market, By Drug:
  • Tafamidis
  • Diflunisal
  • Patisiran
  • Inotersen
  • Others
• U.S. Transthyretin Amyloidosis Treatment Market, By Disease Type:
  • Hereditary Transthyretin Amyloidosis
  • Polyneuropathy
  • Cardiomyopathy
  • Mixed Type
  • Wild Type Amyloidosis
• U.S. Transthyretin Amyloidosis Treatment Market, By Distribution Channel:
  • Hospital Pharmacies
  • Retail Pharmacies 
  • Online Pharmacies