The hereditary transthyretin amyloidosis market is experiencing significant growth driven by increasing disease awareness, advancements in gene-silencing therapies, and the rising prevalence of the condition globally. The market is further fueled by emerging therapies such as Nexiguran ziclumeran (Intellia Therapeutics), ALXN2220 (Neurimmune/Alexion Pharmaceuticals), Coramitug (Novo Nordisk), and others.
LAS VEGAS, Oct. 8, 2025 /PRNewswire/ -- DelveInsight's Hereditary Transthyretin Amyloidosis Market Insights report includes a comprehensive understanding of current treatment practices, hereditary transthyretin amyloidosis emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into leading markets (the US, EU4, UK, and Japan).
Hereditary Transthyretin Amyloidosis Market Summary
- The market size for hereditary transthyretin amyloidosis in the leading markets is expected to grow significantly by 2034.
- The United States accounted for the highest hereditary transthyretin amyloidosis treatment market size in 7MM in 2024, in comparison to the other major markets, i.e., EU4 countries, the United Kingdom, and Japan.
- The US accounted for nearly 65% of the total diagnosed prevalent cases of hATTR in the 7MM in 2024, which is expected to increase further by 2034.
- Leading hereditary transthyretin amyloidosis companies developing emerging therapies, such as Intellia Therapeutics, Neurimmune, Alexion Pharmaceuticals, Novo Nordisk, and others, are developing new therapy for hereditary transthyretin amyloidosis that can be available in the hereditary transthyretin amyloidosis market in the coming years.
- The promising hereditary transthyretin amyloidosis therapies in clinical trials include Nexiguran Ziclumeran, ALXN2220 (formerly NI006), Coramitug, and others.
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Key Factors Driving the Growth of the Hereditary Transthyretin Amyloidosis Market
Advancements in Genetic Testing and Early Diagnosis
Improved genetic testing techniques have enhanced the ability to diagnose hATTR at earlier stages. This advancement enables timely interventions, resulting in improved patient outcomes and increased demand for specialized treatments.
Emergence of CRISPR-Cas9-based Therapy
CRISPR-Cas9 gene editing is a revolutionary genome-editing technology that enables precise and targeted changes to DNA within living cells. Intellia Therapeutics' Nexiguran Ziclumeran is an investigational, in vivo CRISPR-Cas9 gene-editing therapy designed to treat hereditary transthyretin-mediated (hATTR) amyloidosis by directly targeting the root cause of the disease. It uses a guide RNA to direct the Cas9 enzyme to the TTR gene in liver cells, where Cas9 introduces a double-stranded break in the DNA.
Expected Launch of Emerging hATTR Therapies
Several potential therapies are currently under investigation for the treatment of hATTR. The expected introduction of promising candidates such as Nexiguran, Ziclumeran, ALXN2220, Coramitug, and others between 2025 and 2034 is likely to drive a notable transformation in the hATTR market landscape.
Hereditary Transthyretin Amyloidosis Market Analysis
Treatment approaches for hereditary transthyretin-mediated amyloidosis have become increasingly diverse, targeting multiple stages of the disease. Gene-silencing agents, such as eplontersen (WAINUA), patisiran (ONPATTRO), and inotersen (TEGSEDI), act by suppressing TTR mRNA in the liver, thereby reducing the production of both mutant and normal transthyretin proteins and slowing disease progression through lowered circulating TTR levels. TTR stabilizers, including tafamidis (VYNDAQEL/VYNDAMAX), bind to the protein to prevent its misfolding into amyloid fibrils, delaying organ damage, most notably in the heart.
Although no therapies are currently approved to degrade amyloid fibrils already deposited in tissues, this remains an area of active research, with some agents offering potential indirect benefits. Liver transplantation, once the standard option for removing the primary source of mutant TTR, is now rarely used given the availability of effective drug therapies. A significant innovation is gene editing, highlighted by Nexiguran Ziclumeran, an in vivo CRISPR-Cas9-based therapy that irreversibly disrupts the TTR gene in liver cells, potentially serving as a one-time curative treatment. In addition to these disease-modifying strategies, supportive care and symptom management remain vital, addressing complications such as peripheral neuropathy, autonomic dysfunction, and cardiomyopathy, which drive much of the disease burden and impact quality of life.
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Hereditary Transthyretin Amyloidosis Competitive Landscape
Several potential therapies are being investigated for the management of hATTR. The anticipated launch of promising candidates like Nexiguran ziclumeran (Intellia Therapeutics), ALXN2220 (Neurimmune/Alexion Pharmaceuticals), Coramitug (Novo Nordisk), and others during the forecast period (2025-2034) is expected to bring about a significant shift in the market dynamics of hATTR.
Intellia Therapeutics' Nexiguran ziclumeran (nex-z), also known as NTLA-2001, is an investigational in vivo CRISPR gene-editing therapy being developed by Intellia Therapeutics as a potential one-time treatment for transthyretin (ATTR) amyloidosis. The therapy is engineered to silence the TTR gene, responsible for producing the transthyretin (TTR) protein. Development and commercialization are being led by Intellia in partnership with Regeneron, with the program currently advancing through Phase III clinical trials.
Neurimmune/Alexion Pharmaceuticals' ALXN2220 (formerly NI006) is a human monoclonal antibody that selectively binds with high affinity to the pathogenic amyloid form of transthyretin, without affecting its normal physiological state. It is designed to target both wild-type ATTR and mutant forms associated with hereditary ATTR cardiomyopathy (ATTR-CM) and ATTR polyneuropathy.
The anticipated launch of these emerging therapies are poised to transform the hereditary transthyretin amyloidosis market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the hereditary transthyretin amyloidosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.
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Recent Developments in the Hereditary Transthyretin Amyloidosis Market
- In August 2025, Prothena Corporation announced that Novo Nordisk communicated during their second-quarter 2025 results that they expect to advance coramitug, a potential first-in-class amyloid depleter antibody, into a Phase III program for ATTR amyloidosis with cardiomyopathy (ATTR-CM) in 2025.
- In June 2025, Alnylam Pharmaceuticals announced that the European Commission (EC) had approved an additional indication for AMVUTTRA (vutrisiran), an orphan RNAi therapeutic, for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
- In May 2025, Intellia Therapeutics announced positive two-year follow-up data from the ongoing Phase I trial of investigational nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).
What is Hereditary Transthyretin Amyloidosis?
Hereditary transthyretin-mediated (hATTR) amyloidosis is a rare genetic disorder that worsens over time, resulting from mutations in the transthyretin (TTR) gene. These mutations cause abnormal folding of the transthyretin protein, which then deposits as amyloid fibrils in different organs and tissues. The buildup most often impacts the peripheral nerves, heart, and, in some cases, the gastrointestinal system. Symptoms can vary but typically include progressive peripheral neuropathy (numbness, tingling, pain), autonomic dysfunction (such as dizziness and digestive problems), and cardiomyopathy (leading to fatigue, shortness of breath, and heart failure). Without treatment, hATTR amyloidosis causes severe organ damage and can be life-threatening.
Hereditary Transthyretin Amyloidosis Epidemiology Segmentation
The hereditary transthyretin amyloidosis epidemiology section provides insights into the historical and current hereditary transthyretin amyloidosis patient pool and forecasted trends for the leading markets. Around 45% of hATTR cases in the US were specific to familial amyloid polyneuropathy (FAP). Hereditary ATTR affects males and females, with no significant gender prevalence; however, a parent-of-origin effect in carriers is hypothesized, as maternal inheritance of the mutation appears to be associated with a higher risk of disease. In contrast, families with late-onset disease exhibit a male predominance.
The hereditary transthyretin amyloidosis market report proffers epidemiological analysis for the study period 2020-2034 in the leading markets, segmented into:
- Total Prevalent Cases of hATTR
- Total Diagnosed Prevalent Cases of hATTR
- Type-specific Cases of hATTR
- Stage-specific cases of hATTR Distribution of FAC patients by NYHA Criteria
- New York Heart Association (NYHA) Classification of Familial Amyloid Cardiomyopathy (FAC)
Hereditary Transthyretin Amyloidosis Market Report Metrics | Details |
Study Period | 2020-2034 |
Hereditary Transthyretin Amyloidosis Market Report Coverage | 7MM [The United States, the EU4 (Germany, France, Italy, and Spain) the United Kingdom, and Japan]. |
Hereditary Transthyretin Amyloidosis Epidemiology Segmentation | Total Prevalent Cases of hATTR, Total Diagnosed Prevalent Cases of hATTR, Type-specific Cases of hATTR, Stage-specific cases of hATTR Distribution of FAC patients by NYHA Criteria, and New York Heart Association (NYHA) Classification of Familial Amyloid Cardiomyopathy (FAC) |
Key Hereditary Transthyretin Amyloidosis Companies | Intellia Therapeutics, Neurimmune, Alexion Pharmaceuticals, Novo Nordisk, Alnylam Pharmaceuticals, AstraZeneca, Ionis Pharmaceuticals, and others |
Key Hereditary Transthyretin Amyloidosis Therapies | Nexiguran Ziclumeran, ALXN2220 (formerly NI006), Coramitug, AMVUTTRA, WAINUA, ONPATTRO, TEGSEDI, and others |
Scope of the Hereditary Transthyretin Amyloidosis Market Report
- Therapeutic Assessment: Hereditary Transthyretin Amyloidosis current marketed and emerging therapies
- Hereditary Transthyretin Amyloidosis Market Dynamics: Key Market Forecast Assumptions of Emerging Hereditary Transthyretin Amyloidosis Drugs and Market Outlook
- Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
- Unmet Needs, KOL's views, Analyst's views, Hereditary Transthyretin Amyloidosis Market Access and Reimbursement
Download the report to understand which factors are driving hereditary transthyretin amyloidosis therapeutics market trends @ Hereditary Transthyretin Amyloidosis Market Trends
Table of Contents
1 | Hereditary Transthyretin Amyloidosis Market Key Insights |
2 | Hereditary Transthyretin Amyloidosis Market Report Introduction |
3 | Executive Summary |
4 | Key Events |
4.1 | Key Transactions and Collaborations |
5 | Epidemiology and Market Forecast Methodology of hATTR |
6 | hATTR Market Overview at a Glance |
6.1 | Hereditary Transthyretin Amyloidosis Market Landscape Analysis (By Phase, RoA, and Molecule type) |
6.2 | Emerging Landscape Analysis (By Phase, RoA, and Molecule type) |
6.3 | Market Share (%) Distribution of hATTR by Therapies in 2024 |
6.4 | Market Share (%) Distribution of hATTR by Therapies in 2034 |
7 | Disease Background and Overview |
7.1 | Hereditary Transthyretin Amyloidosis Introduction |
7.2 | Hereditary Transthyretin Amyloidosis Signs and Symptoms |
7.3 | Hereditary Transthyretin Amyloidosis Types |
7.4 | Hereditary Transthyretin Amyloidosis Classification |
7.5 | Hereditary Transthyretin Amyloidosis Causes |
7.6 | Hereditary Transthyretin Amyloidosis Stages |
7.7 | Hereditary Transthyretin Amyloidosis Diagnosis |
8 | Hereditary Transthyretin Amyloidosis Treatment |
9 | Epidemiology and Patient Population of hATTR |
9.1 | Key Findings |
9.2 | Assumptions and Rationale |
9.3 | Total Prevalent Cases of hATTR in the US |
9.4 | The United States |
9.4.1 | Total Prevalent Cases of Hereditary Transthyretin Amyloidosis (hATTR) |
9.4.2 | Total Diagnosed Prevalent Cases of Hereditary Transthyretin Amyloidosis (hATTR) |
9.4.3 | Type-specific Cases of Hereditary Transthyretin Amyloidosis (hATTR) |
9.4.4 | Stage-specific Cases of Hereditary Transthyretin Amyloidosis (hATTR) |
9.4.5 | New York Heart Association (NYHA) Classification of Familial Amyloid Cardiomyopathy (FAC) |
9.5 | EU4 and the UK |
9.6 | Japan |
10 | Patient Journey of hATTR |
11 | Marketed Therapies of hATTR |
11.1 | Key Competitors |
11.2 | AMVUTTRA (vutrisiran): Alnylam Pharmaceuticals |
11.2.1 | Product Description |
11.2.2 | Regulatory Milestone |
11.2.3 | Other Development Activities |
11.2.4 | Summary of Pivotal Trials |
11.2.5 | Clinical Trials Information |
11.2.6 | Analyst Views |
11.3 | WAINUA (eplontersen): AstraZeneca/Ionis Pharmaceuticals |
List to be continued in the report… | |
12 | Emerging Therapy of hATTR |
12.1 | Key Competitors |
12.2 | Nexiguran Ziclumeran: Intellia Therapeutics |
12.2.1 | Product Description |
12.2.2 | Other Development Activities |
12.2.3 | Clinical Development |
12.2.3.1 | Clinical Trials Information |
12.2.4 | Safety and Efficacy |
12.2.5 | Analyst Views |
List to be continued in the report… | |
13 | hATTR Market: Seven Major Market Analysis |
13.1 | Key Findings |
13.2 | Hereditary Transthyretin Amyloidosis Market Outlook |
13.3 | Key Hereditary Transthyretin Amyloidosis Market Forecast Assumptions |
13.4 | Conjoint Analysis |
13.5 | Launch Year and Therapy Uptakes |
13.6 | Total Market Size of hATTR in the 7MM |
13.7 | Market Size of hATTR by Therapies in the 7MM |
13.8 | The United States Hereditary Transthyretin Amyloidosis Market Size |
13.8.1 | Total Market Size of hATTR in the United States |
13.8.2 | Market Size of hATTR by Therapies in the United States |
13.9 | EU4 and the UK Hereditary Transthyretin Amyloidosis Market Size |
13.10 | Japan Hereditary Transthyretin Amyloidosis Market Size |
14 | Unmet Needs of hATTR |
15 | SWOT Analysis of hATTR |
16 | KOL Views of hATTR |
17 | Hereditary Transthyretin Amyloidosis Market Access and Reimbursement |
18 | Bibliography |
19 | Hereditary Transthyretin Amyloidosis Market Report Methodology |
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