Transthyretin Amyloidosis Treatment Market Report 2026

The transthyretin amyloidosis treatment market size has grown strongly in recent years. It will grow from $4.96 billion in 2025 to $5.36 billion in 2026 at a compound annual growth rate (CAGR) of 8.1%. The growth in the historic period can be attributed to increased recognition of hereditary amyloidosis, expansion of genetic testing availability, introduction of disease-modifying drugs, improved specialist referral networks, growing rare disease registries.

The transthyretin amyloidosis treatment market size is expected to see strong growth in the next few years. It will grow to $7.42 billion in 2030 at a compound annual growth rate (CAGR) of 8.5%. The growth in the forecast period can be attributed to advancement of next-generation attr therapies, increasing investments in pipeline and combination treatments, broader access to specialty pharmacies, rising demand for personalized therapy selection, continued expansion of clinical trial activity. Major trends in the forecast period include increasing development of targeted attr therapies, rising adoption of gene silencing drugs, growing focus on multisystem disease management, expansion of pipeline therapies for attr variants, improved patient stratification and monitoring.

Rising investments in research and development (R&D) are expected to drive the growth of the transthyretin amyloidosis treatment market in the coming years. R&D investments refer to financial resources allocated to discovering new therapies, improving existing treatments, and advancing scientific knowledge across various disease areas. Funding for R&D is increasing as governments and private organizations focus more on rare diseases, precision medicine, and next-generation therapeutic platforms. Higher R&D investments accelerate the development of innovative therapies, enhancing treatment options and outcomes for transthyretin amyloidosis patients. For example, in November 2023, according to a report by the European Federation of Pharmaceutical Industries and Associations, a Belgium-based trade association, pharmaceutical industry R&D expenditure rose to $50.0 billion in 2023, up from $47.01 billion in 2022. Therefore, rising research and development investments are supporting the growth of the transthyretin amyloidosis treatment market.

Key companies in the transthyretin amyloidosis treatment market are focusing on developing innovative and specialized therapies, including orphan drugs, to improve patient outcomes and broaden treatment options. Eplontersen is an orphan drug specifically designed to treat transthyretin amyloidosis and often receives regulatory incentives to support its development. For example, in March 2023, Ionis Pharmaceuticals, Inc., a Canada-based biotechnology company, received FDA approval for a new drug application for Eplontersen, an experimental orphan drug for treating familial transthyretin-mediated amyloidosis, which can lead to progressive polyneuropathy. Eplontersen works by reducing the synthesis of transthyretin (TTR) protein, targeting both hereditary and non-hereditary forms of ATTR amyloidosis.

In April 2024, BridgeBio Pharma, a US-based biotechnology company, partnered with Bayer to treat transthyretin amyloid cardiomyopathy (ATTR-CM) in Europe. Under this agreement, Bayer will exclusively commercialize the therapy, while BridgeBio will receive up to $310 million in payments and royalties. This collaboration aims to increase the availability of acoramidis for patients with this serious condition. Bayer is a Germany-based life sciences company.

Major companies operating in the transthyretin amyloidosis treatment market are Pfizer Inc, Alnylam Pharmaceuticals Inc, Ionis Pharmaceuticals, Prothena Corporation plc, Eidos Therapeutics Inc, BridgeBio Pharma Inc, Neurimmune AG, AstraZeneca plc, Intellia Therapeutics Inc, Novo Nordisk A/S, YolTech Therapeutics Co Ltd, Life Molecular Imaging GmbH, Attralus Inc, Regeneron Pharmaceuticals, Silence Therapeutics, Proclara Biosciences, Corino Therapeutics, SOM Biotech, Oncopeptides AB, Takeda Pharmaceutical Company Limited, Horizon Therapeutics, Roche Holding AG, SOM Innovation Biotech SA.

North America was the largest region in the transthyretin amyloidosis treatment market in 2025. The regions covered in the transthyretin amyloidosis treatment market report are Asia-Pacific, South East Asia, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the transthyretin amyloidosis treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Taiwan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Tariffs are influencing the transthyretin amyloidosis treatment market by raising costs of imported biologics, gene-silencing compounds, formulation excipients, and specialized packaging materials. Markets in North America and Europe experience pricing pressure due to dependence on global pharmaceutical manufacturing networks, while Asia-Pacific faces increased costs for trial supplies and distribution. These tariffs contribute to higher therapy prices and delayed market entry in some regions. At the same time, they are promoting regional drug production, localized clinical research, and more resilient supply chains for rare disease therapeutics.

The transthyretin amyloidosis treatment market research report is one of a series of new reports that provides transthyretin amyloidosis treatment market statistics, including transthyretin amyloidosis treatment industry global market size, regional shares, competitors with a transthyretin amyloidosis treatment market share, detailed transthyretin amyloidosis treatment market segments, market trends and opportunities, and any further data you may need to thrive in the transthyretin amyloidosis treatment industry. This transthyretin amyloidosis treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

Transthyretin amyloidosis is a slowly progressive disorder marked by the accumulation of abnormal protein deposits in various organs and tissues of the body. Treatment for transthyretin amyloidosis involves medical interventions and therapeutic strategies aimed at managing and alleviating the effects of the condition (ATTR).

The main types of transthyretin amyloidosis treatment include transthyretin amyloidosis with polyneuropathy (ATTR-PN) and transthyretin amyloidosis with cardiomyopathy (ATTR-CM). ATTR-PN is a form of amyloidosis in which abnormal transthyretin proteins accumulate in the nerves, causing peripheral neuropathy and other neurological symptoms. These conditions are managed using medications such as tafamidis, patisiran, inotersen, and others, along with targeted therapies, supportive care, and pipeline therapies. Treatments are distributed through hospital pharmacies, specialty pharmacies, retail pharmacies, and online pharmacies.

The transthyretin amyloidosis treatment market consists of revenues earned by entities by providing biopsy, echocardiography, genetic testing, and imaging studies. The market value includes the value of related goods sold by the service provider or included within the service offering. The transthyretin amyloidosis treatment market also includes sales of tolcapone, and diflunisal. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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