Healthcare Services

Global Transthyretin Amyloidosis Treatment Market Analysis for 2026–2030 with Strategic Forecast Insights for Businesses

April 7, 2026 by Marketing Posting

The Business Research Company’s 2026 market reports include new capabilities such as market attractiveness scoring and analysis, total addressable market analysis, company scoring matrix, interactive excel data dashboard, improved supply chain analysis, upcoming startups in the market, and overview of key products, aimed at improving the depth, usability, and strategic value of the insights delivered.

What Market Size Growth Is Projected For The Transthyretin Amyloidosis Treatment Market During The Forecast Period 2026–2030?

The transthyretin amyloidosis treatment market has seen substantial growth in recent years. It is anticipated to increase from $4.96 billion in 2025 to $5.36 billion in 2026, achieving a compound annual growth rate (CAGR) of 8.1%. This historical expansion can be attributed to increased recognition of hereditary amyloidosis, the wider availability of genetic testing, the introduction of disease-modifying drugs, enhancements in specialist referral networks, and the rise of rare disease registries.

The transthyretin amyloidosis treatment market is projected to experience substantial expansion over the upcoming years. This market is set to reach $7.42 billion by 2030, demonstrating a compound annual growth rate (CAGR) of 8.5%. This anticipated growth is largely driven by factors such as the progress in next-generation attr therapies, greater investments into pipeline and combination treatments, wider availability through specialty pharmacies, increasing preference for personalized therapy options, and the ongoing broadening of clinical trial initiatives. Key developments expected during this period involve the growing creation of targeted attr therapies, a surge in the use of gene silencing medications, an increased emphasis on managing multisystem disease, the extension of pipeline therapies for attr variants, and enhanced methods for patient stratification and monitoring.

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Which Major Factors Are Driving The Expansion Of The Transthyretin Amyloidosis Treatment Market?

The expansion of the transthyretin amyloidosis treatment market is anticipated to be driven by increasing investments in research and development (R&D). These R&D investments represent financial resources primarily channeled into uncovering novel therapies, refining current treatments, and furthering scientific knowledge across various disease domains. The growth in R&D funding stems from a heightened focus by both governmental bodies and private entities on conditions such as rare diseases, precision medicine approaches, and emerging therapeutic platforms. Greater allocation to research and development facilitates the quicker discovery and progress of groundbreaking treatments, thereby enhancing available options and patient outcomes for transthyretin amyloidosis. A notable example occurred in November 2023, when, as per a report from the European Federation of Pharmaceutical Industries and Associations, a Belgium-based trade group, pharmaceutical sector R&D spending climbed to 50,000 million in 2023 from 47,010 million in 2022. Consequently, the rising financial commitment to research and development is serving as a primary catalyst for the growth of the transthyretin amyloidosis treatment market.

What Market Segments Are Evaluated 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 Impacting The Growth Of The Transthyretin Amyloidosis Treatment Market?

Leading companies within the transthyretin amyloidosis treatment market are concentrating on developing innovative and specialized therapies, such as orphan drugs, to enhance patient outcomes and broaden available treatment options. Eplontersen is an orphan drug specifically formulated to treat the transthyretin amyloidosis condition, frequently receiving regulatory advantages to support its development. For instance, in March 2023, Ionis Pharmaceuticals, Inc., a Canada-based biotechnology research company, obtained The Food and Drug Administration (FDA) approval for a new drug application concerning Eplontersen, an experimental orphan drug intended for the management of familial transthyretin-mediated amyloid, which leads to progressive polyneuropathy. Eplontersen serves as a pharmaceutical agent designed to mitigate the synthesis of transthyretin (TTR) protein, addressing both the hereditary and non-hereditary forms of ATTR amyloidosis (amyloid transthyretin amyloidosis).

Who Are The Top Market Participants Influencing The 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 Region Currently Holds The Largest Share Of The Transthyretin Amyloidosis Treatment Market?

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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