The Global Transthyretin Amyloidosis Treatment Market is projected to grow significantly from its estimated valuation of USD 5.88 billion in 2025 to USD 13.58 billion by 2032, charting a strong CAGR of 12.70% between 2026 and 2032, according to the latest study by MarkNtel Advisors. Rising diagnostic capabilities, expanding therapeutic options, and accelerated rare-disease research funding are collectively shaping an evolving and more accessible treatment landscape for transthyretin (TTR) amyloidosis.
Advancing Outlook for TTR Amyloidosis Treatment
The global market is witnessing steady expansion as awareness levels increase, diagnostics improve, and access to RNA-based and TTR-stabilizing medicines accelerates. In major markets such as the United States, ATTR cardiomyopathy (ATTR-CM) prevalence is rising steadily, with estimates indicating up to 120,000 individuals may be affected, though many remain undiagnosed.
Commercialization efforts and treatment innovation are reshaping the competitive environment. Notably, Ionis Pharmaceuticals and AstraZeneca’s eplontersen received FDA approval in 2024 for ATTR polyneuropathy, intensifying competition against established therapies like Pfizer’s tafamidis, which generated USD 3.3 billion in global sales in 2023. In parallel, expanding retail networks—such as over 40,000 retail drug stores in the U.S.—and the rapid adoption of regulated online pharmacies are further enhancing patient access to specialty medicines.
With continuous improvements in diagnostic infrastructure and the introduction of newer RNA-interference, antisense, and stabilizer therapies, global treatment penetration is expected to increase substantially over the next decade.
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Key Regulatory Milestones
Recent high-impact approvals are bolstering treatment accessibility worldwide:
- June 2025: The European Commission approved vutrisiran (AMVUTTRA) for both wild-type and hereditary ATTR cardiomyopathy, expanding RNA-based therapeutic access across all EU states.
- February 2024: The FDA granted Fast Track status to eplontersen for ATTR-CM, underscoring unmet clinical needs and supporting accelerated review pathways for emerging therapies.
Market Drivers: Growing Rare-Disease Investment
Governments and global research institutions are intensifying investments in rare-disease programs, benefiting ATTR treatment development. The NIH invested USD 26 million toward rare-disease research, including amyloidosis, while Europe’s ERDERA Partnership committed USD 410.4 million to strengthen diagnosis and therapeutic research. Similar initiatives across Japan, Canada, and other regions continue fueling biomarker discovery, genomic research, and clinical trial expansion, creating favorable conditions for breakthrough therapies.
Market Trends: Shift Toward RNA Therapies & Digital Diagnostics
A key trend shaping the market trajectory is the transition toward next-generation RNA therapies designed for longer durability and simplified dosing. The FDA’s expanded approval of vutrisiran for ATTR-CM in March 2025 exemplifies growing confidence in RNA-interference treatments.
Digital tools are also transforming early detection. AI-assisted ECG analytics, nuclear imaging enhancements, and machine-learning–driven cardiac mapping now play pivotal roles in identifying ATTR patterns earlier, reducing misdiagnosis and enabling timely intervention.
Challenges: High Treatment Costs and Delayed Approvals
Despite progress, ATTR therapy affordability remains a major barrier. Tafamidis exceeds USD 225,000 annually per patient, while RNA therapies such as patisiran and inotersen surpass USD 450,000 per year in the U.S.—pricing levels that restrict accessibility, especially in emerging economies. Additionally, approval delays of 2–5 years in countries such as India, Mexico, and Brazil impede inclusion in insurance frameworks, limiting treatment adoption.
Segment Insights
- Drug Type: Tafamidis remains the global leader, supported by broad regulatory adoption, proven survival benefits, and convenient once-daily oral dosing.
- Distribution Channel: Hospital pharmacies dominate due to their role in managing complex diagnostics, infusion therapies, and specialized care pathways across global cardiac and neurology centers.
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Key Companies
Leading players include Acrotech Biopharma LLC, Alnylam Pharmaceuticals Inc., AstraZeneca plc, Astellas Pharma Inc., Bristol-Myers Squibb, Ionis Pharmaceuticals, Johnson & Johnson, Prothena Biosciences, Pfizer Inc., and others actively shaping the competitive landscape through R&D innovation and global expansion.
Regional Outlook: North America Leads Market Growth
North America remains the largest and fastest-advancing region driven by early FDA approvals, strong clinical capacity, and rising diagnosis rates. The U.S. and Canada continue to expand access to RNA-interference therapies and stabilizers, further strengthening the region’s leadership position.
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