Transthyretin Amyloidosis Treatment Market Report 2025

This report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market’s historic and forecast market growth by geography.

The transthyretin amyloidosis treatment market size has grown strongly in recent years. It will grow from $5.52 billion in 2024 to $5.95 billion in 2025 at a compound annual growth rate (CAGR) of 7.8%. The growth in the historic period can be attributed to increased disease awareness, advancements in diagnostic tools, drug development and approvals, patient advocacy and support, collaboration in research.

The transthyretin amyloidosis treatment market size is expected to see strong growth in the next few years. It will grow to $8.04 billion in 2029 at a compound annual growth rate (CAGR) of 7.8%. The growth in the forecast period can be attributed to expanded therapeutic pipeline, increased genetic screening, global efforts for rare disease awareness, personalized medicine approaches, improved access to treatment. Major trends in the forecast period include emergence of combination therapies, strategic partnerships for drug development, focus on targeting peripheral and cardiac manifestations, implementation of patient education programs, incorporation of real-world evidence in treatment guidelines.

Increased investments in research and development (R&D) are projected to be the driving force behind the growth of the transthyretin amyloidosis treatment market. R&D investments encompass financial resources allocated towards advancing scientific knowledge, developing new technologies, creating innovative products, and enhancing existing processes. Augmented funding in R&D endeavors facilitates the discovery and advancement of novel treatment methodologies for transthyretin amyloidosis. These approaches include pioneering drugs, gene therapies, and precision medicine techniques, which offer patients more effective and targeted treatment options. For instance, Alnylam Pharmaceuticals, a US-based biopharmaceutical company, elevated its research and development investment from $792,156 in 2021 to $883,015 in 2022, indicating an 11.49% growth. Thus, the escalating investment in research and development is anticipated to propel the growth of the transthyretin amyloidosis treatment market.

The expanding demographic of the aging population is poised to be a significant driver of growth in the transthyretin amyloidosis treatment market. An aging population signifies a demographic shift characterized by an increased median age due to declining fertility rates and longer life expectancies. Age is a substantial risk factor for transthyretin amyloidosis (ATTR), and as the population ages, the prevalence of cases pertaining to this condition rises. Consequently, there is a growing pool of patients necessitating treatment. For example, according to the World Health Organization's October 2022 report, every country globally is witnessing an upsurge in both the quantity and proportion of older individuals in the population. By 2030, it's projected that 1 in 6 people worldwide will be aged 60 years or older. Hence, the burgeoning demographics of the aging population are steering the growth of the transthyretin amyloidosis treatment market.

Leading companies within the transthyretin amyloidosis treatment sector are prioritizing the development of innovative and specialized therapies, notably orphan drugs, aiming to improve patient outcomes and broaden treatment options. Orphan drugs such as Eplontersen are specifically tailored to address transthyretin amyloidosis, often receiving regulatory advantages to facilitate their development. For instance, Ionis Pharmaceuticals, Inc., a Canada-based biotechnology research company, obtained Food and Drug Administration (FDA) approval for a new drug application concerning Eplontersen in March 2023. Eplontersen, an experimental orphan drug, is intended for the treatment of familial transthyretin-mediated amyloid, which leads to progressive polyneuropathy. Functioning as a pharmaceutical agent, Eplontersen is designed to decrease transthyretin (TTR) protein synthesis, targeting both hereditary and non-hereditary variations of ATTR amyloidosis (amyloid transthyretin amyloidosis). The FDA had granted Eplontersen orphan drug designation in 2022.

In April 2024, BridgeBio Pharma, a US-based biotechnology firm, entered into a collaboration with Bayer to address transthyretin amyloid cardiomyopathy (ATTR-CM) in Europe. As part of the agreement, Bayer will have exclusive rights to commercialize the drug, while BridgeBio will receive up to $310 million in payments and royalties. This partnership increases the likelihood of acoramidis becoming available for patients affected by this serious condition. Bayer is a Germany-based life science company.

Major companies operating in the transthyretin amyloidosis treatment market report are Pfizer Inc., Abbvie Inc., Sanofi S.A., Bristol Myers Squibb Company, AstraZeneca PLC, GSK PLC, Novo Nordisk A/S, Merck & Co. Inc., Astellas Pharma Inc., Alexion Pharmaceuticals Inc., Alnylam Pharmaceuticals Inc., PTC Therapeutics, Ionis Pharmaceuticals, Prothena Corporation PLC, BridgeBio Pharma Inc., Sorrento Therapeutics Inc., Intellia Therapeutics, Akcea Therapeutics Inc., Eidos Therapeutics Inc., Acrotech Biopharma Inc., Spectrum Pharmaceuticals Inc., Neurimmune AG, BELLUS Health Inc., Corino Therapeutics Inc., Celgene Corp, SOM Innovation Biotech S.A.

North America was the largest region in the transthyretin amyloidosis treatment market in 2024. The regions covered in the transthyretin amyloidosis treatment market report are Asia-Pacific, 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, Russia, South Korea, UK, USA, Canada, Italy, Spain.

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.

Transthyretin amyloidosis is a gradually progressing condition marked by the accumulation of abnormal protein deposits in the body's organs and tissues. Treatment for transthyretin amyloidosis involves various medical interventions and therapeutic strategies aimed at managing and alleviating the impacts of this condition.

The primary categories of transthyretin amyloidosis treatment include transthyretin amyloidosis with polyneuropathy (ATTR-PN) and transthyretin amyloidosis with cardiomyopathy (ATTR-CM). ATTR-PN involves the abnormal deposition of transthyretin protein in nerves, resulting in peripheral neuropathy and other neurological symptoms. Treatment options for these conditions include drugs such as tafamidis, patisiran, inotersen, as well as targeted therapy, supportive therapy, and pipeline therapies. These treatments are made available through various distribution channels, including hospital pharmacies, specialty pharmacies, retail pharmacies, and online pharmacies.

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.

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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