DelveInsight’s “Hereditary Transthyretin Amyloidosis Market Insights, Epidemiology, and Market Forecast-2034″ 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 (2020-2034).

• In September 2025, Intellia Therapeutics, Inc. (NASDAQ: NTLA), a clinical-stage gene editing company advancing CRISPR-based therapies, announced longer-term follow-up data from its ongoing Phase 1 study of nexiguran ziclumeran (nex-z) for treating hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). The findings were presented in an oral session on September 25 at the 5th International ATTR Amyloidosis Annual Meeting for Patients and Doctors in Baveno, Italy. The results were also published in the New England Journal of Medicine, and the presentation is available on the Scientific Publications & Presentations section of intelliatx.com.

• In August 2025, Prothena Corporation announced that Novo Nordisk, during its Q2 2025 update, confirmed plans to advance coramitug, a potential first-in-class amyloid-clearing antibody, into a Phase III trial for ATTR amyloidosis with cardiomyopathy (ATTR-CM) in 2025. Originally developed by Prothena, coramitug was acquired by Novo Nordisk in July 2021.

• In June 2025, Alnylam Pharmaceuticals announced that the European Commission (EC) has approved an expanded indication for AMVUTTRA (vutrisiran), an orphan RNAi therapy, for treating wild-type or hereditary transthyretin amyloidosis (ATTR-CM) in adult patients with cardiomyopathy.

• 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.

• The current treatment landscape includes several US FDA-approved therapies, such as AMVUTTRA (vutrisiran), ONPATTRO (patisiran), TEGSEDI (inotersen), VYNDAQEL (tafamidis meglumine), VYNDAMAX (tafamidis), WAINUA (eplontersen), among others.

• In 2024, the United States represented nearly 65% of the total diagnosed prevalent cases of hATTR across the 7MM, and this figure is projected to rise further by 2034.

• The most frequently observed hATTR variants in the United States include TTR V30M, TTR T60A, TTR L58H, TTR S77Y, and TTR I84S.

• Approximately 45% of hATTR cases in the United States are attributed to familial amyloid polyneuropathy (FAP).

• Key Hereditary Transthyretin Amyloidosis Companies: Alnylam Pharmaceuticals, AstraZeneca, Ionis Pharmaceuticals, Intellia Therapeutics, Neurimmune, Alexion Pharmaceuticals, Novo Nordisk, Eidos Therapeutics, Corino Therapeutics, Prothena, Regeneron Pharmaceutical, and others

• Key Hereditary Transthyretin Amyloidosis Therapies: AMVUTTRA (vutrisiran), WAINUA (eplontersen), ONPATTRO (patisiran), TEGSEDI (inotersen), Nexiguran Ziclumeran, ALXN2220 (formerly NI006), Coramitug, 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 2020 to 2034. 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 2020–2034 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

• AMVUTTRA (vutrisiran): Alnylam Pharmaceuticals

• WAINUA (eplontersen): AstraZeneca/Ionis Pharmaceuticals

• ONPATTRO (patisiran): Alnylam Pharmaceuticals

• TEGSEDI (inotersen): Ionis Pharmaceuticals

• Nexiguran Ziclumeran: Intellia Therapeutics

• ALXN2220 (formerly NI006): Neurimmune/Alexion Pharmaceuticals

• Coramitug: Novo Nordisk

• 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: 2020–2034

• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

• Key Hereditary Transthyretin Amyloidosis Companies: Alnylam Pharmaceuticals, AstraZeneca, Ionis Pharmaceuticals, Intellia Therapeutics, Neurimmune, Alexion Pharmaceuticals, Novo Nordisk, Eidos Therapeutics, Corino Therapeutics, Prothena, Regeneron Pharmaceutical, and others

• Key Hereditary Transthyretin Amyloidosis Therapies: AMVUTTRA (vutrisiran), WAINUA (eplontersen), ONPATTRO (patisiran), TEGSEDI (inotersen), Nexiguran Ziclumeran, ALXN2220 (formerly NI006), Coramitug, Eplontersen, Acoramidis (AG 10), CRX-1008 (Tolcapone; SOM0226), PRX004, NTLA-2001, and others

• 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 (2020–2034)

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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