DelveInsight Business Research LLP

DelveInsight’s ‘Hereditary Transthyretin Amyloidosis (hATTR) – Market Insights, Epidemiology and Market Forecast– 2030’ report deliver an in-depth understanding of the hATTR, historical and forecasted epidemiology as well as the hATTR market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

Key Highlights 

• Key Companies working in hATTR include Pfizer, Alnylam Pharmaceuticals, Akcea Therapeutics, Ionis Pharmaceuticals, AstraZeneca, Eidos Therapeutics

• Key Therapies included in the hATTR market include Vyndaqel, Onpattro and many others 

hATTR Overview

Transthyretin (earlier known as prealbumin) is an abundant, soluble, β-strand rich 55 kDa homotetramer serum protein that is responsible for the transportation of both vitamin A (via retinol-binding protein) and thyroxin throughout the body. TTR is also involved in the binding and redistribution of β-amyloid in the choroid plexus as well as in the retention of T4 in the cerebral spinal fluid (CSF). TTR may sometimes dissociate into its 127-amino acid monomeric subunits and undergo aberrant changes to form amyloidogenic intermediates. These intermediates then might self-associate to become amyloid fibrils that accumulate as amyloid deposits throughout the body, resulting in Transthyretin Amyloidosis. Transthyretin Amyloidosis (ATTR) can be sub-classified as wild-type (wt) or hereditary, and the latter is then further sub-divided into familial amyloid polyneuropathy (FAP) and familial amyloid cardiomyopathy (FAC).

The hereditary forms of amyloidosis are autosomal dominant diseases characterized by the deposition of variant proteins, in distinctive tissues. The most common hereditary form is transthyretin amyloidosis (ATTR) caused by the misfolding of protein monomers derived from the tetrameric protein transthyretin (TTR). Mutations in the gene for TTR frequently result in instability of TTR and subsequent fibril formation. Closely related is wild-type TTR in which the native TTR protein, particularly in the elderly, can destabilize and re-aggregate causing nonfamilial cases of TTR amyloidosis.

hATTR Epidemiology Insights

• The total prevalent cases of hATTR in the 7MM were estimated to be 23,879 in 2021. The numbers are expected to increase during the forecast period (2019-2032).

• Epidemiology assessed for the condition showed that the US, in 2021, accounted for the highest prevalent cases of hATTR.

• Among the EU-5 countries in 2021, France had the highest diagnosed prevalent cases of hATTR. In contrast, Germany had the lowest cases in 2021. Japan accounted for 672 diagnosed prevalent hATTR cases in 2021.

• In the US, in 2021 the highest proportion of type-specific cases were observed in Familial Amyloid

• Polyneuropathy (FAP) i.e. 3,715 in comparison to Familial Amyloid Cardiomyopathy (FAC) and Mixed hATTR Type. Among the stage-specific distribution of FAP in the US, stage 1 has the highest cases followed by stage 2 cases in 2021.

Click here to learn more about the hATTR Market Landscape

The Report Covers the hATTR Epidemiology Segmented by:

• hATTR prevalent cases 

• hATTR incident cases 

• hATTR treatment cases 

• hATTR diagnosed cases 

hATTR Market Outlook 

The hereditary Transthyretin Amyloidosis (hATTR) market has been assessed, based on demand, prescription analysis, and the annual cost of therapy of current and forecasted market value of the approved drugs, Vyndaqel, Onpattro, Tegsedi in the US, Europe, and Japan as well as forecasted patient share and the annual cost of therapy for upcoming medicines. Supportive therapies like Diflunisal, other symptomatic drugs for TTR-FAP, and recommended symptomatic drugs like loop diuretics, aldosterone antagonists, angiotensin-converting enzyme inhibitors, and beta-blockers have been considered for hATTR market estimation. Liver transplantation is the most common treatment method in hATTR amyloidosis, as the liver is the main source of abnormal TTR production. This procedure is most effective in patients who are in the early stages of the disease.

In addition, only a few therapies have been approved for this indication. Each drug has a different purpose or mode of action, such as stabilizing the TTR protein, preventing the production of the TTR protein, or removing amyloid deposits. The market scenario started changing in 2018, with the launch of Tegsedi and Onpattro, and witnessed further growth in 2019, with the US launch of Vyndaqel/Vyndamax.

Key Companies Working in the hATTR Market

• Pfizer

• Alnylam Pharmaceuticals

• Akcea Therapeutics

• Ionis Pharmaceuticals

• AstraZeneca

• Eidos Therapeutics

• Corino Therapeutics

• Prothena

And many others

hATTR Therapies Covered and Analyzed in the Report

• Vyndaqel

• Onpattro

• Tegsedi

• Eplontersen

• Acoramidis 

• CRX-1008 

And many others 

Learn more about the Key Companies and Emerging Therapies in the hATTR Market

Table of Contents 

1.  Key Insights 

2.  hATTR Introduction 

3.  Executive Summary of hATTR           

4.  Disease Background and Overview

5.  Epidemiology and patient population

6.  hATTR Emerging Therapies

7.  hATTR Market Outlook

8.  Market Access and Reimbursement of Therapies

9.  Market Drivers 

10.  Market Barriers 

11.  Appendix

12.  Report Methodology

13.  DelveInsight Capabilities

14.  Disclaimer

Learn about the detailed offerings of the report @ hATTR Market Outlook

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