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Hereditary Transthyretin Amyloidosis Market Insight, Epidemiology, and Market Forecast - 2036 Now Available Reveals Why a $3B Market Is Racing Toward a One-Time Curative Gene-Editing Therapy

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NTLA Intellia Therapeutics' gene-editing therapy Nexiguran ziclumeran (nex-z) is highlighted as a potential one-time curative treatment for hATTR, receiving Orphan Drug and Regenerative Medicine Advanced Therapy designations. The company plans to resume patient enrollment in key Phase III trials, indicating strong development progress. REGN Regeneron Pharmaceuticals is collaborating with Intellia Therapeutics on Nexiguran ziclumeran (nex-z), a gene-editing therapy for hATTR. This collaboration positions Regeneron to benefit from the potential success of this innovative curative treatment. ALNY Alnylam Pharmaceuticals is developing Nucresiran, a next-generation RNA silencer with potential launch timelines in 2028 and 2030. They also received FDA approval to expand AMVUTTRA's indication for hATTR-CM, making it the first therapy approved for both ATTR-CM and hATTR-PN. IONS Ionis Pharmaceuticals has highlighted eplontersen as a key asset for hATTR treatment, with an NDA submission expected in 2026 and a potential launch in 2027 for hATTR-CM. This indicates positive development and future market potential. PFE Pfizer announced it would discontinue VYNDAQEL (tafamidis meglumine) in the United States, though VYNDAMAX (tafamidis) remains available. This action suggests a strategic shift or reduced focus on this specific product line.

Hereditary Transthyretin Amyloidosis Market Insight, Epidemiology, and Market Forecast - 2036 Now Available Reveals Why a $3B Market Is Racing Toward a One-Time Curative Gene-Editing Therapy Dublin, Sept. 04, 2026 (GLOBE NEWSWIRE) -- "Hereditary Transthyretin Amyloidosis - Market Insight, Epidemiology, and Market Forecast - 2036" report has been added to ResearchAndMarkets.com's offering.

The report delivers an in-depth understanding of hATTR, its historical and forecasted epidemiology, and market trends in the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan.

A Market Already Worth $3 Billion, Still Transforming

According to the report, the hATTR market size was found to be approximately USD 3.00 billion in the leading markets in 2025, with a growth rate of approximately 11% CAGR projected for 2026-2036. The United States captured the largest share of the market, at approximately USD 2.00 billion in 2025, accounting for nearly 65% of the total diagnosed prevalent cases of hATTR in the 7MM, a dominance expected to increase further by 2036.

From Stabilization to Cure: The Next Phase of Treatment

The report identifies a decisive shift underway in how hATTR is treated. The current treatment landscape includes FDA-approved drugs such as vutrisiran (AMVUTTRA), patisiran (ONPATTRO), tafamidis (VYNDAMAX), and eplontersen (WAINUA) — but these therapies only stabilize or reduce abnormal TTR production; none reverse existing nerve or cardiac damage, and there is no FDA-approved curative therapy.

Hereditary Transthyretin Amyloidosis (hATTR) Market Size and Forecast in the 7MM

Numbers are subject to change with report updation, clinical information updates, etc.

The 'Hereditary Transthyretin Amyloidosis (hATTR) - Market Insights, Epidemiology and Market Forecast - 2036' report delivers an in-depth understanding of the 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.

The Hereditary Transthyretin Amyloidosis (hATTR) market report delivers a comprehensive analysis of the current treatment landscape, including standards of care, clinical practices, and evolving therapeutic algorithms. It evaluates hATTR patient burden trends, revenue & market share dynamics, peak patient share & therapy uptake analysis, and provides an in-depth market size assessment, and growth rate projections (Historical & Forecast 2022-2036) across global regions. The report highlights key unmet medical needs in Hereditary Transthyretin Amyloidosis (hATTR) and maps the competitive and clinical landscape to uncover high-value opportunities, providing a clear outlook on future market growth potential.

That gap is exactly where the report says the next wave of value is concentrated. Nexiguran ziclumeran (nex-z), a CRISPR-Cas9-based gene-editing therapy developed by Intellia Therapeutics in collaboration with Regeneron, is designed to permanently inactivate the TTR gene as a one-time treatment and has already received Orphan Drug and Regenerative Medicine Advanced Therapy designations from the FDA, plus Orphan Drug Designation from the European Commission. As per the March 2026 Intellia Therapeutics presentation, the Nex-Z program plans to resume patient enrollment in the Phase III MAGNITUDE trial in ATTR-CM and the Phase III MAGNITUDE-2 trial in ATTRv-PN, with MAGNITUDE-2 enrollment expected to complete in the second half of 2026.

Alnylam Pharmaceuticals, meanwhile, plans to launch Nucresiran, a next-generation RNA silencer, with a potential launch timeline of 2028 for polyneuropathy and 2030 for cardiomyopathy indications, according to the company's Q4 and Full Year 2025 financial results. Ionis Pharmaceuticals has highlighted eplontersen as a key asset, with an NDA submission expected in 2026 and a potential launch anticipated in 2027 for hATTR-CM.

A Market in Active Motion

The competitive landscape continues shifting in real time. In March 2025, Alnylam announced FDA approval of a supplemental New Drug Application expanding AMVUTTRA's indication to treat the cardiomyopathy of wild-type or hATTR-CM in adults making it the first and only FDA-approved therapeutic for both ATTR-CM and the polyneuropathy of hATTR-PN in adults. In August 2025, Pfizer announced it would discontinue VYNDAQEL (tafamidis meglumine) in the United States effective December 31, 2025, while VYNDAMAX (tafamidis) remains available for all eligible patients. Akcea Therapeutics discontinued commercial availability of Tegsedi (inotersen) in the U.S. on September 27, 2024, due to low market utilization.

Significant Unmet Needs Remain

The report identifies critical gaps that continue to shape opportunity in this market: no curative therapy is currently available; irreversible organ damage at diagnosis limits treatment benefit; diagnosis remains delayed and difficult due to non-specific symptoms and disease heterogeneity; and there is a clear need for therapies that effectively treat both neuropathy and cardiomyopathy together.

Why This Report Matters for Decision-Makers

This report gives pharma and biotech stakeholders a bottom-up, epidemiology-based forecast built for strategic decision-making not just market sizing. It identifies strong upcoming players to help devise strategies to get ahead of competitors, provides detailed analysis and ranking of class-wise current and emerging therapies to clarify which mechanisms will lead, and delivers KOL perspectives on accessibility, acceptability, and compliance-related challenges shaping future adoption. The analysts connected with more than 10 KOLs, with interviews conducted across more than 6 KOLs in the 7MM at centers including the University of North Carolina at Chapel Hill, the Berlin Institute of Health at Charité, and the University of Nottingham.

For business development and commercial strategy teams evaluating where to compete in a market moving from stabilization toward cure, for investors assessing which pipeline assets, RNA-silencing or gene-editing will define the next decade, and for market access teams preparing for a wave of new launches between 2026 and 2030, this report provides the data, competitive intelligence, and forecast rigor to act with confidence

Report Scope

The report evaluates hATTR patient burden trends, revenue and market share dynamics, peak patient share and therapy uptake analysis, and provides an in-depth market size assessment and growth rate projections (2022-2036) across the 7MM. It covers disease background, diagnosis, treatment algorithms, epidemiology, drug and pipeline analysis, market access and reimbursement, KOL views, unmet needs, and SWOT and conjoint analysis.

A selection of companies mentioned in this report includes, but is not limited to:

For more information about this report visit https://www.researchandmarkets.com/r/ky9q6d

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