Transthyretin Amyloidosis Treatment - Market Share Analysis, Industry Trends & Statistics, Growth Forecasts (2024 - 2029)

The Transthyretin Amyloidosis Treatment Market size is estimated at USD 5.30 billion in 2024, and is expected to reach USD 10.90 billion by 2029, growing at a CAGR of 15.40% during the forecast period (2024-2029).

The transthyretin amyloidosis treatment market is expected to grow significantly due to the growing geriatric population and risk factors for the amyloid light chain (AL), increasing research and developments (R&D), and raising public disease awareness. Aging is associated with changes in the body's protein structure and function. Transthyretin, a transport protein primarily produced in the liver, undergoes misfolding over time. This misfolding is a critical factor in the development of amyloidosis, where abnormal protein aggregates form and accumulate in tissues, leading to organ dysfunction.

In addition, plasma cell dyscrasia, a condition characterized by abnormal plasma cells, is a known risk factor for amyloid light chain (AL) amyloidosis, a subtype of amyloidosis. The risk of developing plasma cell dyscrasias, including conditions like multiple myeloma, tends to increase as older individuals are more susceptible to abnormalities in plasma cell function and proliferation. For instance, according to the data published by the WHO in October 2023, the population of individuals aged over 65 years is estimated to outnumber those under the age of 15 years in the WHO European region by 2024. This shows a significant number of people in the aging population compared to those of a young age, which may increase the risk of amyloid light chain (AL) amyloidosis, thereby boosting market growth over the study period.

Furthermore, increasing R&D and growing disease awareness are other significant factors driving the growth of the transthyretin amyloidosis treatment market. Pharmaceutical and biotechnology firms are committing substantial resources to R&D to pioneer innovative and targeted treatments for transthyretin amyloidosis. For instance, in February 2024, AstraZeneca reported positive high-level results from the Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Similarly, in March 2023, Ionis Pharmaceuticals reported the acceptance of a New Drug Application (NDA) for eplontersen, an antisense medicine for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) by the Food and Drug Administration. These instances show the growing R&D on treating transthyretin amyloidosis, which is expected to have significant growth in the market over the forecast period.

Therefore, the growing geriatric population, risk factors for AL, and increasing R&D activities are some major factors driving the growth of the transthyretin amyloidosis treatment market. However, the high cost associated with this treatment is expected to restrain the market over the forecast period.

Transthyretin Amyloidosis Treatment Market Trends

The Hereditary Transthyretin Amyloidosis Segment is Expected to Hold a Significant Share Over the Forecast Period

Hereditary transthyretin amyloidosis (hATTR) is a rare and severe, heterogeneous multisystem condition with prevalent peripheral (both somatic and autonomic) nervous system impairment due to mutations in the transthyretin (TTR) gene. Hereditary transthyretin amyloidosis (hATTR) is expected to hold a significant market share due to its hereditary characteristics, providing a consistent and identifiable patient base for targeted therapeutic endeavors. The factors driving the growth of this segment include growing awareness of hATTR through awareness programs, growing research investments, and market player strategies like partnerships, product approvals, and launches, which increase the availability of the products.

The increasing disease awareness significantly drives the segment growth as it increases the demand for effective treatments. As healthcare professionals and the general public become more informed about the symptoms, risk factors, and diagnostic advancement related to conditions, there is a growing emphasis on proactive management. For instance, in March 2024, Alnylam Pharmaceuticals Inc. launched the Family Health History Road Trip to encourage conversations between family members about their health history with their doctor to understand their risk for developing hereditary ATTR (hATTR) amyloidosis. This awareness helps in early diagnosis and treatment of the disease using various products, thereby boosting segment growth over the study period.

Furthermore, the strategies of market players, like partnerships and product approvals, increase the availability of the products in the market, thereby boosting segment growth. For instance, in January 2024, AstraZeneca and Ionis selected Orsini Specialty Pharmacy as the exclusive specialty pharmacy partner for WAINUA (eplontersen), an FDA-approved treatment for adults living with hereditary transthyretin-mediated amyloid polyneuropathy. In addition, in August 2022, Alnylam Pharmaceuticals Inc. stated that the FDA approved Onpattro, which can be used for nerve pain caused by hereditary transthyretin amyloidosis. These strategic activities are expected to increase the availability of the products and boost the segment growth over the forecast period.

Therefore, due to the growing awareness and strategic activities of market players, the hereditary transthyretin amyloidosis segment is expected to hold a significant market share over the forecast period.

North America is Expected to Record the Largest Share in the Market Over the Forecast Period

North America is expected to have the largest share in the market due to factors such as advanced healthcare infrastructure and the increasing incidence of transthyretin amyloidosis disease in the region. Established research capabilities, growing investments for research and development, and clinical trial infrastructure are driving the region's market growth.

New investments in developing advanced treatment products for transthyretin amyloidosis (ATTR) are expected to boost the market growth. For instance, in May 2023, the National Institutes of Health (NIH) awarded the Amyloidosis Research Consortium with USD 40,000 toward advancing ATTR amyloidosis drug development. These factors are expected to propel the market growth over the forecast period.

Additionally, growing market player strategies, such as product approvals and positive recommendations from the regulatory authorities, increase the usage of treatment products in the region. For instance, in April 2024, Alnylam Canada ULC received a positive recommendation for reimbursement from the Canadian Agency for Drugs and Technologies in Health (CADTH) for AMVUTTRA (vutrisiran injection). Similarly, in October 2023, AMVUTTRA was authorized for sale in Canada to treat stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). Hence, the positive recommendations and approvals increase the usage and availability of products in the region, thereby boosting the region's market growth over the forecast period.

Transthyretin Amyloidosis Treatment Industry Overview

The transthyretin amyloidosis treatment market is moderately consolidated in nature due to the presence of limited companies operating globally as well as regionally. The competitive landscape includes an analysis of a few international as well as local companies that hold market shares and are well known, including Pfizer Inc., Ionis Pharmaceuticals, Alnylam Pharmaceuticals Inc., Intellia Therapeutics Inc., and BridgeBio Inc.

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