Hereditary Transthyretin Amyloidosis Market Report 2026

The hereditary transthyretin amyloidosis market size has grown strongly in recent years. It will grow from $1.49 billion in 2025 to $1.62 billion in 2026 at a compound annual growth rate (CAGR) of 9%. The growth in the historic period can be attributed to increasing clinical recognition of attr amyloidosis, availability of tafamidis therapy, expansion of specialized neurology and cardiology centers, improved genetic testing for ttr mutations, rising patient registry programs.

The hereditary transthyretin amyloidosis market size is expected to see strong growth in the next few years. It will grow to $2.28 billion in 2030 at a compound annual growth rate (CAGR) of 8.9%. The growth in the forecast period can be attributed to expansion of next-generation gene therapies, increasing investment in rare disease research, growing adoption of precision medicine approaches, rising development of crispr-based treatments, improved disease outcome monitoring technologies. Major trends in the forecast period include increasing adoption of gene silencing therapies, rising use of rnAI-based treatments, growing focus on early cardiac and neurological diagnosis, expansion of oral therapeutic options, enhanced long-term disease management strategies.

The expanding geriatric population is expected to drive the growth of the hereditary transthyretin amyloidosis market in the coming years. The geriatric population includes individuals aged 65 years and older, who often require specialized medical care and support as a result of aging-related health conditions. This population segment is increasing due to rising life expectancy supported by improvements in healthcare systems and living standards. Hereditary transthyretin amyloidosis (hATTR) has a significant impact on older adults, as it leads to progressive neuropathy and cardiomyopathy, contributing to higher morbidity among aging individuals. For example, in January 2024, according to the Population Reference Bureau, a US-based nonprofit organization, the population in the United States aged 65 and above is projected to increase from 58 million in 2022 to 82 million by 2050, representing a 47% rise. As a result, the growing geriatric population is contributing to the expansion of the hereditary transthyretin amyloidosis market.

Key companies operating in the hereditary transthyretin amyloidosis market are advancing innovative treatment options, including RNA interference (RNAi) therapeutics, to target and reduce the production of transthyretin (TTR) protein. RNAi therapeutics represent a class of treatments that utilize RNA interference technology to silence specific genes, thereby lowering the production of proteins responsible for disease progression. For instance, in October 2024, Alnylam Pharmaceuticals, Inc., a US-based biopharmaceutical company, submitted a supplemental new drug application to the US Food and Drug Administration for Vutrisiran (AMVUTTRA) for the treatment of transthyretin amyloidosis with cardiomyopathy. This submission highlights the company’s innovative RNAi-based approach aimed at reducing TTR protein levels, a central driver of the disease. The subcutaneous mode of administration of Vutrisiran provides a more convenient and patient-friendly alternative to conventional intravenous therapies, positioning it as a potentially impactful treatment option for patients with this condition.

In July 2023, Ionis Pharmaceuticals, Inc., a US-based biopharmaceutical company focused on RNA-targeted drug discovery and development, entered into a partnership with AstraZeneca plc to co-develop and commercialize eplontersen for the treatment of transthyretin amyloidosis in the United States. The collaboration also aims to broaden global access to the therapy by utilizing AstraZeneca’s established commercial infrastructure, particularly across Latin America and other international markets. AstraZeneca plc is a UK-based biopharmaceutical company engaged in the discovery, development, and commercialization of prescription medicines.

Major companies operating in the hereditary transthyretin amyloidosis market are Alnylam Pharmaceuticals Inc., Ionis Pharmaceuticals Inc., BridgeBio Pharma Inc., Pfizer Inc., Eli Lilly and Company, Intellia Therapeutics Inc., Regeneron Pharmaceuticals Inc., AstraZeneca plc, Novartis AG, Roche Holding AG, Sanofi S.A., Takeda Pharmaceutical Company Limited, GlaxoSmithKline plc, Bristol Myers Squibb, Merck and Co Inc., Johnson and Johnson, AbbVie Inc., Biogen Inc., Vertex Pharmaceuticals Incorporated, Amgen Inc.

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

Tariffs are influencing the hereditary transthyretin amyloidosis market by increasing costs of imported biologics, gene-silencing drug components, advanced diagnostic tools, and specialized pharmaceutical manufacturing inputs. North America and Europe are most affected due to reliance on high-value imported therapies, while Asia-Pacific faces increased costs for rare disease drug access. These tariffs are raising treatment prices and impacting reimbursement strategies. However, they are also supporting regional biopharmaceutical manufacturing, local research collaborations, and long-term investments in advanced rare disease therapy development.

The hereditary transthyretin amyloidosis market research report is one of a series of new reports that provides hereditary transthyretin amyloidosis market statistics, including hereditary transthyretin amyloidosis industry global market size, regional shares, competitors with a hereditary transthyretin amyloidosis market share, detailed hereditary transthyretin amyloidosis market segments, market trends and opportunities, and any further data you may need to thrive in the hereditary transthyretin amyloidosis industry. This hereditary transthyretin amyloidosis 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.

Hereditary transthyretin amyloidosis is a rare inherited condition resulting from mutations in the TTR gene, which cause transthyretin protein to misfold and accumulate as amyloid deposits in various organs. The disease primarily affects the nervous system, heart, and other tissues, leading to progressive neuropathy and cardiomyopathy.

The main treatment administration types for hereditary transthyretin amyloidosis include oral, subcutaneous injection, and other methods. Oral administration, delivered through tablets, capsules, or liquid formulations, provides a convenient and patient-friendly option for treating hATTR amyloidosis, thereby enhancing accessibility and treatment adherence. The therapeutic drug classes used in the management of hATTR amyloidosis include RNA interference (RNAi) agents, such as patisiran, and small interfering RNAs (siRNAs), including vutrisiran. These therapies are distributed via hospital and retail pharmacies and are utilized across settings such as hospitals, clinics, and other healthcare facilities.

The hereditary transthyretin amyloidosis market consists of revenues earned by entities by providing services such as diagnostic services, treatment services, and genetic counseling services. The market value includes the value of related goods sold by the service provider or included within the service offering. The hereditary transthyretin amyloidosis market also includes sales of genetic testing kits, imaging technologies, biomarkers, and assays. 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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