Transthyretin Amyloidosis Treatment Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, 2021-2031

The Global Transthyretin Amyloidosis Treatment Market is projected to expand significantly, rising from USD 7.78 Billion in 2025 to USD 17.82 Billion by 2031, reflecting a compound annual growth rate of 14.81%. This sector includes pharmacological treatments aimed at stabilizing the transthyretin protein or inhibiting its production in the liver to stop amyloid fibrils from accumulating in the heart and nerves. Growth is largely fueled by an aging global population and the widespread use of non-invasive nuclear scintigraphy, which has vastly improved diagnostic accuracy over older biopsy techniques. For instance, the European Society of Cardiology noted in 2025 that approximately 19% of elderly patients with unexplained hypertrophic cardiomyopathy were diagnosed with transthyretin amyloid cardiomyopathy, highlighting how targeted screening is revealing a larger patient base.

However, the market faces a major impediment due to the high costs associated with disease-modifying therapies, which create significant reimbursement obstacles and limit access within budget-constrained healthcare systems. These financial limitations frequently lead to delays in starting treatment or outright denial of insurance coverage, effectively curbing revenue growth even as the number of diagnosed cases continues to rise worldwide.

Market Drivers

The primary engine for growth in the Global Transthyretin Amyloidosis Treatment Market is the introduction and increasing utilization of innovative disease-modifying therapies. Medical professionals are more frequently prescribing targeted treatments that either stabilize the transthyretin tetramer or suppress its hepatic synthesis, thereby modifying the disease's natural progression. This trend is highlighted by the strong commercial performance of existing standards of care and the rapid uptake of new products. For example, Pfizer Inc. reported in its Third Quarter 2024 Earnings that the Vyndaqel product family reached global revenues of USD 1.45 billion, representing 63 percent operational growth due to high demand. Additionally, AstraZeneca's Year to date and Q3 2024 Results in November 2024 indicated that its newly launched Wainua treatment generated USD 23 million in quarterly revenue, showing strong initial adoption in the polyneuropathy sector.

This commercial momentum is bolstered by a strong clinical pipeline and increased research and development investment aimed at broadening labeled indications and enhancing survival rates. Pharmaceutical companies are actively developing next-generation RNA interference and antisense oligonucleotide therapies to treat the larger cardiomyopathy patient group, which has historically been challenging to manage.

A significant breakthrough occurred with the release of data confirming the effectiveness of gene silencers for cardiac issues; according to Alnylam Pharmaceuticals' June 2024 HELIOS-B Phase 3 Study Results, vutrisiran showed a statistically significant 28 percent reduction in the composite risk of all-cause mortality and recurrent cardiovascular events in patients with ATTR amyloidosis and cardiomyopathy. These advancements are essential for regulatory approvals that are expected to substantially expand the market size in the near future.

Market Challenges

A major barrier to market expansion is the exorbitant cost of disease-modifying therapies for transthyretin amyloidosis, which disrupts the link between improved diagnostic capabilities and revenue generation. Although screening programs are successfully identifying more patients, the steep prices of these lifelong medications force public and private insurers to implement strict reimbursement protocols. These financial hurdles often appear as complicated prior authorization processes or coverage refusals, preventing the growing pool of diagnosed patients from accessing treatment. As a result, manufacturers encounter a situation where clinical demand is increasing, yet actual product adoption is artificially constrained by economic gatekeeping.

The disconnect caused by financial toxicity directly affects patient access to care. Data from the Amyloidosis Research Consortium in 2024 revealed that one in five patients with transthyretin amyloidosis reported delaying treatment initiation specifically because of costs or insurance coverage difficulties. With a significant segment of the patient population compelled to postpone or forego therapy, affordability issues are directly shrinking the practical market size and hindering the sector's commercial trajectory.

Market Trends

The market is being fundamentally transformed by the advancement of In Vivo CRISPR-Cas9 gene editing therapeutics, which are shifting the treatment paradigm from chronic management to potential one-time curative solutions. Unlike current gene silencers that require ongoing administration, these next-generation agents use lipid nanoparticles to permanently disable the TTR gene in hepatocytes, effectively stopping the production of disease-causing proteins. The potential for a single dose to halt disease progression is supported by recent durability data; for instance, Intellia Therapeutics announced in its November 2024 Phase 1 Clinical Data that the investigational agent nexiguran ziclumeran achieved a deep and consistent mean serum TTR reduction of 90 percent at month 12 across all patients, irrespective of baseline levels.

Concurrently, the commercialization of next-generation high-affinity TTR stabilizers is setting a new benchmark for efficacy by achieving near-complete tetramer stabilization compared to earlier options. These novel small molecules are designed to maximize blood binding occupancy, thereby significantly lowering the release of toxic monomers responsible for amyloid deposition in the heart. This evolution toward superior stabilization was confirmed by recent regulatory achievements; according to BridgeBio Pharma's November 2024 announcement regarding the FDA approval for Attruby, data from the pivotal Phase 3 ATTRibute-CM trial demonstrated that acoramidis resulted in a 42 percent reduction in the composite risk of all-cause mortality and recurrent cardiovascular-related hospitalizations at month 30 compared to placebo.

Key Players Profiled in the Transthyretin Amyloidosis Treatment Market

• Alnylam Pharmaceuticals
• Bridgebio Pharma
• Pfizer
• Ionis Pharmaceuticals
• Astrazeneca
• Alexion Pharmaceutical
• Proliferan
• Enobio Pharma
• Moderna
• Intellia Therapeutics
• Regeneron Pharmaceuticals

Report Scope

In this report, the Global Transthyretin Amyloidosis Treatment Market has been segmented into the following categories:

Transthyretin Amyloidosis Treatment Market, by Type:

• ATTR-PN
• ATTR-CM

Transthyretin Amyloidosis Treatment Market, by Therapy:

• Targeted Therapy (Onpattro, Inotersen, Vyndaqel/Vyndamax)
• Supportive Therapy
• Pipeline Therapy

Transthyretin Amyloidosis Treatment Market, by Disease Type:

• Hereditary Transthyretin Amyloidosis (Polyneuropathy, Cardiomyopathy, Mixed Type)
• Wild Type Amyloidosis

Transthyretin Amyloidosis Treatment Market, by Distribution Channel:

• Hospital Pharmacies
• Specialty Pharmacies
• Retail Pharmacies
• Online Pharmacies

Transthyretin Amyloidosis Treatment Market, by Region:

• North America
• Europe
• Asia-Pacific
• South America
• Middle East & Africa

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Transthyretin Amyloidosis Treatment Market.

Available Customization

The analyst offers customization according to your specific needs. The following customization options are available for the report:

• Detailed analysis and profiling of additional market players (up to five).

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