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Transthyretin Amyloidosis Treatment Industry Poised for Rapid Growth, Forecast to Touch $7.42 Billion by 2030 at 8.5% CAGR

March 3, 2026March 3, 2026 by Marketing Posting

Uncover key drivers, emerging technologies, and competitive movements shaping the transthyretin amyloidosis treatment market from 2026–2035 with trusted insights from The Business Research Company

What level of CAGR-driven expansion is anticipated in the Transthyretin Amyloidosis Treatment Market between 2026 and 2030?

The transthyretin amyloidosis treatment market size has experienced robust expansion in recent years. This market is projected to expand from $4.96 billion in 2025 to $5.36 billion in 2026, demonstrating a compound annual growth rate (CAGR) of 8.1%. Historically, this growth has been driven by factors such as enhanced awareness of hereditary amyloidosis, wider access to genetic testing, the launch of disease-modifying therapies, better specialist referral systems, and the proliferation of rare disease registries.

The transthyretin amyloidosis treatment market is set for substantial expansion over the upcoming years. It is projected to reach $7.42 billion by 2030, demonstrating a compound annual growth rate (CAGR) of 8.5%. This anticipated growth during the forecast period stems from various factors, including the progress in next-generation attr therapies, increasing investments in pipeline and combination treatments, improved access to specialty pharmacies, a rising demand for personalized therapy selection, and the ongoing expansion of clinical trial activities. Key trends expected in this period encompass the escalating development of targeted attr therapies, a growing adoption of gene silencing drugs, an increased focus on multisystem disease management, the expansion of pipeline therapies for attr variants, and enhanced patient stratification and monitoring.

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What Drivers Are Affecting Demand-Supply Dynamics In The Transthyretin Amyloidosis Treatment Market?

The escalating financial commitments towards research and development (R&D) are projected to boost the transthyretin amyloidosis treatment market moving forward. These R&D investments signify financial resources dedicated to discovering new therapies, refining existing treatments, and advancing scientific understanding across diverse disease areas. Funding for R&D is on the rise as governments and private sectors intensify their focus on rare diseases, precision medicine, and next-generation therapeutic platforms. Such increased investments in R&D accelerate the discovery and progression of innovative therapies, consequently improving treatment options and outcomes for transthyretin amyloidosis patients. For instance, in November 2023, according to a report published by the European Federation of Pharmaceutical Industries and Associations, a Belgium-based trade association, pharmaceutical industry research and development expenditure increased to 50,000 million in 2023, up from 47,010 million in 2022. Therefore, these growing research and development investments are propelling the growth of the transthyretin amyloidosis treatment market.

What Segments Are Included Within The Transthyretin Amyloidosis Treatment Market?

The transthyretin amyloidosis treatment market covered in this report is segmented –

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

Which Trends Are Influencing The Development Of The Transthyretin Amyloidosis Treatment Market?

Leading companies in the transthyretin amyloidosis treatment market are concentrating on developing innovative and specialized therapies, such as orphan drugs, to improve patient outcomes and broaden available treatment options. Eplontersen is an orphan drug specifically created to manage transthyretin amyloidosis, often benefiting from regulatory support for its development. For example, in March 2023, Ionis Pharmaceuticals, Inc., a biotechnology research company based in Canada, received The Food and Drug Administration (FDA) approval for a new drug application regarding Eplontersen. This experimental orphan drug is intended for treating familial transthyretin-mediated amyloid, which results in progressive polyneuropathy. Eplontersen functions as a pharmaceutical agent designed to reduce the synthesis of transthyretin (TTR) protein, thereby addressing both hereditary and non-hereditary forms of ATTR amyloidosis (amyloid transthyretin amyloidosis).

Who Are The Primary Competitors 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

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Which Regions Are Projected To Dominate The Transthyretin Amyloidosis Treatment Market In The Coming Years?

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.

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