(Albany, US), November 6, 2026 – DelveInsight’s ‘Hereditary Transthyretin Amyloidosis (hATTR) Market Insights, Epidemiology and Market Forecast – 2036’ report delivers an in-depth understanding of hereditary transthyretin amyloidosis (hATTR), historical and forecasted epidemiology, as well as the hATTR market trends in the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan.
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Key Takeaways from the Hereditary Transthyretin Amyloidosis Market Report
In March 2026, Intellia Therapeutics outlined a planned resumption of patient enrollment in the Phase III MAGNITUDE trial in ATTR-CM and the Phase III MAGNITUDE-2 trial in ATTRv-PN, with enrollment in MAGNITUDE-2 expected to complete in the second half of 2026.
In February 2026, Alnylam Pharmaceuticals’ Q4 and Full Year 2025 results indicated plans to launch nucresiran, with a potential launch timeline of 2028 for polyneuropathy and 2030 for cardiomyopathy indications.
The hATTR market size is projected to reach approximately USD 3,000 million in 2025 and is expected to grow at a robust CAGR of ~11% during 2026–2036.
According to DelveInsight’s estimates, the United States accounted for the largest hATTR market size, reaching approximately USD 2,000 million in 2025.
The transthyretin amyloidosis market is shifting toward targeted therapies, especially RNA-based and gene-editing treatments, driven by improved efficacy, safety, and broader use across earlier disease stages.
There is no FDA-approved curative therapy for hATTR. Current treatments focus on stabilising or reducing abnormal TTR production, and they do not reverse existing nerve or cardiac damage, which highlights a key unmet need.
Regional dynamics in the amyloidosis therapeutics market show the US leading in revenue, while Japan and EU markets demonstrate steady growth, reflecting increasing diagnosis rates and rising adoption of novel mechanisms.
The leading hATTR companies include Alnylam Pharmaceuticals, Ionis Pharmaceuticals, AstraZeneca, Pfizer, Intellia Therapeutics, Regeneron, and others.
The current treatment landscape includes US FDA-approved drugs such as vutrisiran (AMVUTTRA), patisiran (ONPATTRO), inotersen (TEGSEDI), tafamidis (VYNDAMAX), and eplontersen (WAINUA), among others.
The anticipated launch of promising candidates like nexiguran ziclumeran (nex-z), nucresiran, and others during the forecast period (2026–2036) is expected to bring a significant shift in the market dynamics of hATTR.
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Hereditary Transthyretin Amyloidosis Overview
Hereditary transthyretin amyloidosis (hATTR) is a rare, inherited disorder caused by mutations in the transthyretin (TTR) gene, leading to misfolded protein deposits (amyloid) in tissues. The mutation makes the TTR protein unstable, so it folds the wrong way and builds up in different organs and tissues as amyloid. The disease primarily affects the peripheral nerves (hATTR-PN) and the heart (hATTR-CM), resulting in progressive neuropathy and cardiomyopathy. hATTR-PN leads to progressive motor decline, with fatal outcomes within about ten years if untreated, while transthyretin amyloidosis–related cardiomyopathy may result in death within about five years if untreated.
Diagnosis is often challenging due to clinical heterogeneity and overlap with other neuropathies and cardiomyopathies. It is confirmed through genetic testing to identify pathogenic TTR gene mutations, along with tissue biopsy demonstrating amyloid deposits using Congo red staining with apple-green birefringence under polarised light. Common biopsy sites include the abdominal fat pad, salivary gland, gastric mucosa, or affected nerve tissue. In cases of cardiac involvement, echocardiography, cardiac MRI, and nuclear scintigraphy help detect transthyretin cardiac amyloid deposition.
Hereditary Transthyretin Amyloidosis Epidemiology Segmentation in the 7MM
Total Prevalent Cases of hATTR
Total Diagnosed Prevalent Cases of hATTR
Type-specific Diagnosed Prevalent Cases of hATTR
Stage-specific Diagnosed Prevalent Cases of FAP
Distribution of FAC Patients by New York Heart Association (NYHA) Criteria
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Hereditary Transthyretin Amyloidosis Epidemiology Key Findings
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.
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, while late-onset familial cases tend to show a higher prevalence among males.
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, underscoring the burden of Japan amyloidosis hATTR.
In 2025, France recorded the highest diagnosed prevalence of hATTR among other EU4 countries and the United Kingdom.
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).
In the USA, the 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.
The hATTR mutation has a 50% inheritance risk, but variable penetrance means symptoms may not appear. Close monitoring is therefore essential, as asymptomatic carriers can still pass the disease to their children.
Approved Therapies for Hereditary Transthyretin Amyloidosis
Management focuses on disease-modifying therapies (TTR stabilisers and gene-silencing agents), along with symptomatic care and, in select cases, organ transplantation to slow progression and improve outcomes. Approved therapies include TTR stabilisers (tafamidis) and gene-silencing agents (patisiran, inotersen, vutrisiran, eplontersen) that reduce abnormal TTR levels. These treatments are most effective when initiated early and primarily aim to delay neurological and cardiac decline rather than reverse existing damage.
Vutrisiran (AMVUTTRA): Alnylam Pharmaceuticals
Vutrisiran (AMVUTTRA) is a subcutaneously administered transthyretin-directed small interfering ribonucleic acid (siRNA) therapeutic, also called an RNAi therapeutic, developed by Alnylam Pharmaceuticals for the treatment of ATTR amyloidosis, including hATTR amyloidosis and wild-type ATTR (wtATTR) amyloidosis. It was approved in June 2022 in the USA for the treatment of the polyneuropathy of hATTR amyloidosis in adults and received a positive opinion in the EU in July 2022 for adult patients with stage 1 or stage 2 polyneuropathy. Vutrisiran is also under regulatory review in Japan for the treatment of the polyneuropathy of hATTR amyloidosis in adults.
Eplontersen (WAINUA/WAINZUA): Ionis Pharmaceuticals and AstraZeneca
WAINUA/WAINZUA (eplontersen) is a ligand-conjugated antisense oligonucleotide (LICA) medicine designed to reduce the production of transthyretin (TTR) protein. It has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Eplontersen is an antisense oligonucleotide-GalNAc conjugate that causes degradation of mutant and wild-type TTR mRNA, resulting in a reduction of serum TTR protein and TTR protein deposits in tissues.
Hereditary Transthyretin Amyloidosis Pipeline Analysis
Nexiguran ziclumeran (nex-z): Intellia Therapeutics and Regeneron
Nexiguran ziclumeran (nex-z), also known as NTLA-2001, is an investigational in vivo CRISPR-based gene editing therapy being developed by Intellia Therapeutics as a one-time treatment for ATTR amyloidosis. It is designed to inactivate the TTR gene, which encodes the TTR protein. Intellia leads development and commercialisation as part of a collaboration with Regeneron, and the drug is currently active in Phase III. Nex-z has received Orphan Drug (ODD) and Regenerative Medicine Advanced Therapy (RMAT) designations from the US FDA and has also been granted ODD by the European Commission.
Nucresiran: Alnylam Pharmaceuticals
Nucresiran is an investigational RNAi therapy being developed by Alnylam Pharmaceuticals for the treatment of transthyretin amyloidosis. It aims to reduce hepatic production of transthyretin protein and is currently in Phase III clinical development for ATTR-CM and hATTR-PN.
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Hereditary Transthyretin Amyloidosis Market Outlook
Although there is currently no curative therapy for hATTR, early diagnosis and timely intervention play a critical role in improving patient outcomes and slowing disease progression. TTR stabilisers such as tafamidis prevent TTR protein misfolding and subsequent amyloid fibril formation, while gene-silencing therapies, including patisiran, inotersen, eplontersen, and vutrisiran, reduce hepatic production of both mutant and wild-type TTR protein and slow neuropathy progression. While no approved therapies specifically target amyloid fibril clearance, this remains an important emerging area of research.
The emerging pipeline is increasingly focused on next-generation disease-modifying and potentially curative approaches, including CRISPR-based gene-editing therapies and novel RNA-targeting platforms designed to deliver deeper and more durable TTR suppression. Liver transplantation is now rarely used due to the availability of effective RNA-based therapies, and comprehensive symptomatic management remains essential. Overall, the launch of first-in-class therapies, improved diagnostic approaches, and increasing disease awareness are expected to drive steady growth in the 7MM hATTR market from 2022–2036.
Hereditary Transthyretin Amyloidosis Market Drivers
Rising prevalence and improved diagnosis of hereditary transthyretin amyloidosis (hATTR).
Growing awareness and early disease screening.
Increasing adoption of gene-silencing therapies.
Advancements in targeted treatments and drug development.
Rising investment in rare disease research and innovative therapies.
Hereditary Transthyretin Amyloidosis Market Barriers
High treatment costs and limited patient affordability.
Delayed diagnosis due to nonspecific symptoms.
Limited awareness and genetic testing accessibility.
Complex treatment administration and patient monitoring.
Strict regulatory requirements and reimbursement challenges.
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Scope of the Hereditary Transthyretin Amyloidosis Market Report
Coverage: 7MM (US, Germany, France, Italy, Spain, the UK, and Japan)
Study Period: 2022–2036
Historical Year: 2022–2025
Forecast Period: 2026–2036
Base Year: 2026
hATTR Market CAGR: ~11% (2026–2036)
hATTR Companies: Alnylam Pharmaceuticals, Ionis Pharmaceuticals, AstraZeneca, Pfizer, Intellia Therapeutics, Regeneron, and others
hATTR Therapies: Vutrisiran (AMVUTTRA), Patisiran (ONPATTRO), Inotersen (TEGSEDI), Eplontersen (WAINUA), Tafamidis (VYNDAMAX), Nexiguran ziclumeran (nex-z), Nucresiran, and others
hATTR Market Segmentation: By Region/Geographies and Drugs/Therapies
Analysis: KOL Views, SWOT Analysis, Reimbursement, Conjoint Analysis, Unmet Need, Market Drivers and Barriers
Table of Contents
Key Insights
Report Introduction
Executive Summary
Key Events
Epidemiology and Market Forecast Methodology
Hereditary Transthyretin Amyloidosis (hATTR) Market Overview at a Glance
Disease Background and Overview of Hereditary Transthyretin Amyloidosis (hATTR)
Treatment Guidelines of Hereditary Transthyretin Amyloidosis (hATTR)
Epidemiology and Patient Population of Hereditary Transthyretin Amyloidosis (hATTR)
Patient Journey of Hereditary Transthyretin Amyloidosis (hATTR)
Marketed Therapies
Emerging Therapies
Hereditary Transthyretin Amyloidosis (hATTR): 7MM Analysis
Unmet Needs of Hereditary Transthyretin Amyloidosis (hATTR)
SWOT Analysis of Hereditary Transthyretin Amyloidosis (hATTR)
KOL Views of Hereditary Transthyretin Amyloidosis (hATTR)
Market Access and Reimbursement of Hereditary Transthyretin Amyloidosis (hATTR)
Appendix
DelveInsight Capabilities
Disclaimer
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