Transthyretin Amyloidosis Treatment Market Report 2026

Transthyretin Amyloidosis Treatment Market Overview

• Transthyretin Amyloidosis Treatment market size has reached to $4.96 billion in 2025 • Expected to grow to $7.42 billion in 2030 at a compound annual growth rate (CAGR) of 8.5% • Growth Driver: The Rise In Research And Development Investments Driving The Growth Of The Market Due To Increased Innovation And Therapy Advancement • Market Trend: Innovative Orphan Drug Combinations Transforming The Landscape Of Transthyretin Amyloidosis Treatment • North America was the largest region in 2025.

What Is Covered Under Transthyretin Amyloidosis Treatment Market?

Transthyretin amyloidosis is a slowly progressive condition characterized by the buildup of abnormal protein deposits in the body's organs and tissues. Transthyretin amyloidosis treatment refers to the medical interventions and therapeutic strategies designed to manage and mitigate the effects of transthyretin amyloidosis (ATTR). The main types of transthyretin amyloidosis treatment include transthyretin amyloidosis with polyneuropathy (ATTR-PN) and transthyretin amyloidosis with cardiomyopathy (ATTR-CM). Transthyretin amyloidosis with polyneuropathy (ATTR-PN) refers to a type of amyloidosis characterized by the deposition of abnormal transthyretin protein in nerves, leading to peripheral neuropathy and other neurological symptoms. These disease types are treated with drugs such as tafamidis, patisiran, inotersen, and others, along with targeted therapy, supportive therapy, and pipeline therapies, which are distributed through channels including hospital pharmacies, specialty pharmacies, retail pharmacies, and online pharmacies.

What Is The Transthyretin Amyloidosis Treatment Market Growth Forecast?

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.

Global Transthyretin Amyloidosis Treatment Market Segmentation

1) By Type: Transthyretin Amyloidosis With Polyneuropathy (ATTR-PN), Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM) 2) By Drug: Tafamidis, Patisiran, Inotersen, Other Drugs 3) By Therapy: Targeted Therapy, Supportive Therapy, Pipeline Therapy 4) By Distribution Channel: Hospital Pharmacies, Specialty Pharmacies, Retail Pharmacies, Online Pharmacies Subsegments: 1) By Transthyretin Amyloidosis With Polyneuropathy (ATTR-PN): Early-Stage ATTR-PN, Advanced-Stage ATTR-PN 2) By Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM): Mild ATTR-CM, Moderate ATTR-CM, Severe ATTR-CM

What Is The Driver Of The Transthyretin Amyloidosis Treatment Market?

The rising investments in research and development (R&D) are expected to propel the growth of the transthyretin amyloidosis treatment market going forward. research and development investments refer to financial resources dedicated to discovering new therapies, improving existing treatments, and advancing scientific understanding across disease areas. R&D funding is increasing as governments and private sectors expand their focus on rare diseases, precision medicine, and next-generation therapeutic platforms. Rising investments in research and development accelerate the discovery and advancement of innovative therapies, improving treatment options and outcomes for transthyretin amyloidosis patients. For instance, in November 2023, according to the report published by the European Federation of Pharmaceutical Industries and Associations, a Belgium-based trade association, pharmaceutical industry research and development expenditure rose to 50,000 million in 2023, up from 47,010 million in 2022. Therefore, rising research and development investments are driving the growth of the transthyretin amyloidosis treatment industry.

Key Players In The Global Transthyretin Amyloidosis Treatment Market

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

Global Transthyretin Amyloidosis Treatment Market Trends and Insights

Major companies in the transthyretin amyloidosis treatment market focus on developing innovative and specialized treatments, such as orphan drugs, to enhance patient outcomes and expand treatment options. Eplontersen is an orphan drug specifically designed to treat transthyretin amyloidosis condition, often receiving regulatory benefits to support its development. For instance, in March 2023, Ionis Pharmaceuticals, Inc., a Canada-based biotechnology research company, received The Food and Drug Administration (FDA) approval of a new drug application for Eplontersen, an experimental orphan drug intended for the treatment of familial transthyretin-mediated amyloid, leading to progressive polyneuropathy. Eplontersen is a pharmaceutical agent developed to mitigate the synthesis of transthyretin (TTR) protein to address both the hereditary and non-hereditary variations of ATTR amyloidosis (amyloid transthyretin amyloidosis).

What Are Latest Mergers And Acquisitions In The Transthyretin Amyloidosis Treatment Market?

In April 2024, BridgeBio Pharma, a US-based biotech company, collaborated with Bayer. This partnership aimed at treating transthyretin amyloid cardiomyopathy (ATTR-CM) in Europe. Under the deal, Bayer will exclusively commercialize the drug, with BridgeBio receiving up to $310 million in payments and royalties. This collaboration enhances the potential availability of acoramidis for patients suffering from this serious condition. Bayer is a Germany-based Life Science company.

Regional Outlook

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

What Defines the Transthyretin Amyloidosis Treatment Market?

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.

How is Market Value Defined and Measured?

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.

What Key Data and Analysis Are Included in the Transthyretin Amyloidosis Treatment Market Report 2026?

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