DelveInsight’s “Hereditary Transthyretin Amyloidosis Market Insights, Epidemiology, and Market Forecast-2032″ report offers an in-depth understanding of the Hereditary Transthyretin Amyloidosis, historical and forecasted epidemiology as well as the Hereditary Transthyretin Amyloidosis market trends in the United States, EU4 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
To Know in detail about the Hereditary Transthyretin Amyloidosis market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Hereditary Transthyretin Amyloidosis Market Forecast
Some of the key facts of the Hereditary Transthyretin Amyloidosis Market Report:
The Hereditary Transthyretin Amyloidosis market size is anticipated to grow with a significant CAGR during the study period (2019-2032)
In November 2024, Intellia Therapeutics, Inc. (NASDAQ:NTLA), a prominent clinical-stage gene editing company specializing in CRISPR-based therapies, has announced promising new clinical data from its ongoing Phase 1 trial of nexiguran ziclumeran (nex-z, also referred to as NTLA-2001) in patients with transthyretin (ATTR) amyloidosis. Nex-z is an experimental in vivo CRISPR-based gene editing treatment under investigation as a one-time therapy for ATTR amyloidosis. Intellia leads the development and commercialization of nex-z in collaboration with Regeneron.
In October 2024, Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced that WAINZUA (eplontersen), developed in collaboration with AstraZeneca, has been recommended for approval by the Committee for Medicinal Products for Human Use (CHMP) in the European Union (EU) for treating hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, also known as hATTR-PN or ATTRv-PN. If approved by the European Commission, WAINZUA will become the first and only medicine in the EU for treating ATTRv-PN that can be self-administered monthly using an auto-injector.
In January 2024, WAINUA™ (eplontersen) is a self-administered auto-injector treatment designed for managing the polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR-PN or ATTRv-PN) in adult patients. Developed by Ionis Pharmaceuticals, a biotech firm headquartered in the United States, WAINUA™ utilizes Ionis’ sophisticated LIgand-Conjugated Antisense (LICA) technology to regulate the production of transthyretin (TTR) protein. This therapeutic approach aims to address the underlying pathology of ATTR for improved patient outcomes.
According to DelveInsight’s examination, the total number of diagnosed prevalent cases of hereditary transthyretin amyloidosis (hATTR) in the Seven Major Markets (7MM) was approximately 15,096 in 2022. These cases are projected to rise over the study period spanning from 2019 to 2032.
In 2022, the United States represented close to 62% of the overall diagnosed prevalent cases of hereditary transthyretin amyloidosis (hATTR) in the Seven Major Markets (7MM). This percentage is anticipated to grow even higher by the year 2032.
In 2022, France had the highest number of diagnosed prevalent cases of hereditary transthyretin amyloidosis (hATTR) among the European Union Four (EU4) and the United Kingdom (UK), totaling approximately 1,593 cases. In contrast, Germany reported the lowest number of cases in this group.
Key Hereditary Transthyretin Amyloidosis Companies: Ionis Pharmaceuticals, AstraZeneca, Eidos Therapeutics, Corino Therapeutics, Prothena, Novo Nordisk, Intellia Therapeutics, Regeneron Pharmaceutical, and others
Key Hereditary Transthyretin Amyloidosis Therapies: Eplontersen, Acoramidis (AG 10), CRX-1008 (Tolcapone; SOM0226), PRX004, NTLA-2001, and others
The Hereditary Transthyretin Amyloidosis epidemiology based on gender analyzed that FAP is the most affected type-specific hATTR in the US
The Hereditary Transthyretin Amyloidosis market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Hereditary Transthyretin Amyloidosis pipeline products will significantly revolutionize the Hereditary Transthyretin Amyloidosis market dynamics.
Hereditary Transthyretin Amyloidosis Overview
Hereditary Transthyretin Amyloidosis (hATTR), also known as transthyretin amyloidosis or ATTRv amyloidosis, is a rare, inherited disorder characterized by the accumulation of abnormal deposits of a protein called transthyretin (TTR) in various tissues and organs throughout the body.
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Hereditary Transthyretin Amyloidosis Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Hereditary Transthyretin Amyloidosis Epidemiology Segmentation:
The Hereditary Transthyretin Amyloidosis market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:
Total Prevalence of Hereditary Transthyretin Amyloidosis
Prevalent Cases of Hereditary Transthyretin Amyloidosis by severity
Gender-specific Prevalence of Hereditary Transthyretin Amyloidosis
Diagnosed Cases of Episodic and Chronic Hereditary Transthyretin Amyloidosis
Download the report to understand which factors are driving Hereditary Transthyretin Amyloidosis epidemiology trends @ Hereditary Transthyretin Amyloidosis Epidemiology Forecast
Hereditary Transthyretin Amyloidosis Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Hereditary Transthyretin Amyloidosis market or expected to get launched during the study period. The analysis covers Hereditary Transthyretin Amyloidosis market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Hereditary Transthyretin Amyloidosis Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Hereditary Transthyretin Amyloidosis Therapies and Key Companies
CRX-1008 (Tolcapone; SOM0226): Corino Therapeutics
PRX004: Prothena/ Novo Nordisk
NTLA-2001: Intellia Therapeutics/Regeneron Pharmaceutical
Eplontersen: Ionis Pharmaceuticals/ AstraZeneca
Acoramidis (AG 10): Eidos Therapeutics
Discover more about therapies set to grab major Hereditary Transthyretin Amyloidosis market share @ Hereditary Transthyretin Amyloidosis Treatment Market
Hereditary Transthyretin Amyloidosis Market Strengths
The development and success of gene silencing therapies in hATTR amyloidosis is a breakthrough for adult-onset, neurodegenerative diseases.
The upcoming therapies with their novel mechanism of action hold potential to combat the unmet need faced by the patients with hATTR-CM and hATTR-PN and provide better treatment options to the patients.
The upcoming pipeline also includes a gene therapy based on CRISPR technology, which is currently in early phase of development. If this therapy gets approved, it could provide a cure for hATTR.
Hereditary Transthyretin Amyloidosis Market Opportunities
The unique mechanisms of action of inotersen and patisiran overcome many limitations of previous therapies for patients with hATTR.
Currently there is only one therapy approved for ATTR-CM in the 7MM, i.e., Vandaqel, this provides a window of opportunity for key players to develop therapies targeting ATTR-CM.
Additional evidence for current and emerging therapies for patients with hATTR offers much‐needed hope, along with a promise of better treatment, for this debilitating and life‐threatening disease.
Scope of the Hereditary Transthyretin Amyloidosis Market Report
Study Period: 2019–2032
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Hereditary Transthyretin Amyloidosis Therapeutic Assessment: Hereditary Transthyretin Amyloidosis current marketed and Hereditary Transthyretin Amyloidosis emerging therapies
Hereditary Transthyretin Amyloidosis Market Dynamics: Hereditary Transthyretin Amyloidosis market drivers and Hereditary Transthyretin Amyloidosis market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
Hereditary Transthyretin Amyloidosis Unmet Needs, KOL’s views, Analyst’s views, Hereditary Transthyretin Amyloidosis Market Access and Reimbursement
To know more about Hereditary Transthyretin Amyloidosis companies working in the treatment market, visit @ Hereditary Transthyretin Amyloidosis Clinical Trials and Therapeutic Assessment
Table of Contents
1. Hereditary Transthyretin Amyloidosis Market Report Introduction
2. Executive Summary for Hereditary Transthyretin Amyloidosis
3. SWOT analysis of Hereditary Transthyretin Amyloidosis
4. Hereditary Transthyretin Amyloidosis Patient Share (%) Overview at a Glance
5. Hereditary Transthyretin Amyloidosis Market Overview at a Glance
6. Hereditary Transthyretin Amyloidosis Disease Background and Overview
7. Hereditary Transthyretin Amyloidosis Epidemiology and Patient Population
8. Country-Specific Patient Population of Hereditary Transthyretin Amyloidosis
9. Hereditary Transthyretin Amyloidosis Current Treatment and Medical Practices
10. Hereditary Transthyretin Amyloidosis Unmet Needs
11. Hereditary Transthyretin Amyloidosis Emerging Therapies
12. Hereditary Transthyretin Amyloidosis Market Outlook
13. Country-Wise Hereditary Transthyretin Amyloidosis Market Analysis (2019–2032)
14. Hereditary Transthyretin Amyloidosis Market Access and Reimbursement of Therapies
15. Hereditary Transthyretin Amyloidosis Market Drivers
16. Hereditary Transthyretin Amyloidosis Market Barriers
17. Hereditary Transthyretin Amyloidosis Appendix
18. Hereditary Transthyretin Amyloidosis Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
About DelveInsight
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It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.
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