Hereditary Transthyretin Amyloidosis (hATTR) Market Trends, Innovation, and Future Growth Outlook to 2036 - DelveInsight |Alnylam Pharma, Ionis Pharma, Novo Nordisk, AstraZeneca, Pfizer, Intellia

The Key Hereditary Transthyretin Amyloidosis Companies in the market include - Alnylam Pharmaceuticals, Ionis Pharmaceuticals, AstraZeneca, Pfizer, Intellia Therapeutics, Regeneron, Neurimmune, Alexion Pharmaceuticals, Novo Nordisk, Eidos Therapeutics, Corino Therapeutics, Prothena, Regeneron Pharmaceutical, and others.

DelveInsight's "Hereditary Transthyretin Amyloidosis Market Insights, Epidemiology, and Market Forecast-2036 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.

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Some of the key facts of the Hereditary Transthyretin Amyloidosis Market Report:

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The hereditary transthyretin amyloidosis (hATTR) market size is estimated to attain a value of nearly USD 3,000 million in 2025 and is anticipated to register a strong CAGR of 11% throughout the forecast period from 2026 to 2036.

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According to estimates, the United States represented the largest share of the hATTR market in 2025, with a market size of nearly USD 2,000 million.

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In March 2026, Intellia Therapeutics presentation, the Nex-Z ATTR program outlines a planned resumption of patient enrollment in the Phase III MAGNITUDE trial in ATTR-CM, resumption of patient enrollment in the Phase III MAGNITUDE-2 trial in ATTRv-PN, and expected completion of enrollment in MAGNITUDE-2 in the second half of 2026.

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In February 2026, according to the Q4 and Full Year 2025 financial results of Alnylam Pharmaceuticals, the company plans to launch Nucresiran, a next-generation RNA silencer for Transthyretin Amyloidosis, with a potential launch timeline of 2028 for polyneuropathy and 2030 for cardiomyopathy indications.

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As per the Ionis Pharmaceuticals Q4 2025 presentation, eplontersen is highlighted as a key asset, with an NDA submission expected in 2026 and a potential launch anticipated in 2027 for hATTR-CM.

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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.

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In August 2025, Pfizer announced that it would discontinue VYNDAQEL (tafamidis meglumine) in the United States effective December 31, 2025, while confirming that VYNDAMAX (tafamidis) would continue to remain available for all eligible patients with a prescription.

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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.

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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.

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In March 2025, Alnylam Pharmaceuticals announced that the US FDA approval of the supplemental New Drug Application (sNDA) for its RNAi therapeutic, AMVUTTRA (vutrisiran), for the treatment of the cardiomyopathy of wild-type or hATTR-CM in adults to reduce cardiovascular mortality, cardiovascular hospitalisations, and urgent heart failure visits. The approval expands the indication for AMVUTTRA, which now becomes the first and only therapeutic approved by the FDA for the treatment of ATTR-CM and the polyneuropathy of hATTR-PN in adults.

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As per DelveInsight's analysis, the US accounted for nearly 65% of the total diagnosed prevalent cases of hATTR in the 7MM in 2025, which is expected to increase further by 2036.

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The secondary analysis indicates that hATTR generally affects both males and females equally, with no clear gender predominance. However, a possible parent-of-origin effect is suggested, as maternal inheritance may increase disease risk. In contrast, late-onset familial cases tend to show a higher prevalence among males.

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A Japan-based study revealed that late-onset and nonendemic cases of hATTR are more common than previously recognised. Disease severity by ambulatory status showed 59% in stage 1, 19% in stage 2, and 14% in stage 3.

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In 2025, France recorded the highest diagnosed prevalence of hATTR among other EU4 countries and the United Kingdom.

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In the USA, the highest proportion of hATTR cases is seen in Familial Amyloid Polyneuropathy (FAP) at 45%, followed by mixed hATTR, while the lowest proportion is observed in Familial Amyloid Cardiomyopathy (FAC).

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In the USA, the New York Heart Association (NYHA) classification of FAC shows that the majority of patients fall under Class II (60%), followed by Class III, while the lowest proportion is seen in Class IV.

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

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Key Hereditary Transthyretin Amyloidosis Therapies: Vutrisiran (AMVUTTRA), Eplontersen (WAINUA/WAINZUA), Vutrisiran (AMVUTTRA), Patisiran (ONPATTRO), Inotersen (TEGSEDI), Tafamidis (VYNDAMAX), Nexiguran ziclumeran (nex-z), Nucresiran, 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

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The Hereditary Transthyretin Amyloidosis epidemiology based on gender analyzed that FAP is the most affected type-specific hATTR in the US

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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 2022 to 2036. 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 2022-2036 in the 7MM segmented into:

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Total Prevalent Cases of hATTR

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Total Diagnosed Prevalent Cases of hATTR

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Type-specific Diagnosed Prevalent Cases of hATTR

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Stage-specific Diagnosed Prevalent Cases of FAP

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Distribution of FAC Patients by New York Heart Association (NYHA) Criteria

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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

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Vutrisiran (AMVUTTRA): Alnylam Pharmaceuticals

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Eplontersen (WAINUA/WAINZUA): Ionis Pharmaceuticals and AstraZeneca

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Vutrisiran (AMVUTTRA): Alnylam Pharmaceuticals

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Patisiran (ONPATTRO): Alnylam Pharmaceuticals

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Inotersen (TEGSEDI): Ionis Pharmaceuticals

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Tafamidis (VYNDAMAX): Pfizer

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Nexiguran ziclumeran (nex-z): Intellia Therapeutics and Regeneron

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Nucresiran: Alnylam Pharmaceuticals

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WAINUA (eplontersen): AstraZeneca/Ionis Pharmaceuticals

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ONPATTRO (patisiran): Alnylam Pharmaceuticals

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TEGSEDI (inotersen): Ionis Pharmaceuticals

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Nexiguran Ziclumeran: Intellia Therapeutics

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ALXN2220 (formerly NI006): Neurimmune/Alexion Pharmaceuticals

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Coramitug: Novo Nordisk

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CRX-1008 (Tolcapone; SOM0226): Corino Therapeutics

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PRX004: Prothena/ Novo Nordisk

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NTLA-2001: Intellia Therapeutics/Regeneron Pharmaceutical

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Eplontersen: Ionis Pharmaceuticals/ AstraZeneca

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Acoramidis (AG 10): Eidos Therapeutics

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Hereditary Transthyretin Amyloidosis Market Strengths

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The development and success of gene silencing therapies in hATTR amyloidosis is a breakthrough for adult-onset, neurodegenerative diseases.

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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.

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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

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The unique mechanisms of action of inotersen and patisiran overcome many limitations of previous therapies for patients with hATTR.

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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.

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Additional evidence for current and emerging therapies for patients with hATTR offers muchneeded hope, along with a promise of better treatment, for this debilitating and lifethreatening disease.

Scope of the Hereditary Transthyretin Amyloidosis Market Report

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Study Period: 2022-2036

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

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

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Key Hereditary Transthyretin Amyloidosis Therapies: Vutrisiran (AMVUTTRA), Eplontersen (WAINUA/WAINZUA), Vutrisiran (AMVUTTRA), Patisiran (ONPATTRO), Inotersen (TEGSEDI), Tafamidis (VYNDAMAX), Nexiguran ziclumeran (nex-z), Nucresiran, 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

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Hereditary Transthyretin Amyloidosis Therapeutic Assessment: Hereditary Transthyretin Amyloidosis current marketed and Hereditary Transthyretin Amyloidosis emerging therapies

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Hereditary Transthyretin Amyloidosis Market Dynamics: Hereditary Transthyretin Amyloidosis market drivers and Hereditary Transthyretin Amyloidosis market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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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 [https://www.delveinsight.com/sample-request/hereditary-transthyretin-amyloidosis-competitive-landscape-and-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

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 (2022-2036)

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

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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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