Transthyretin Amyloidosis Treatment Market Size & Share 2026-2036

Transthyretin Amyloidosis Treatment Market Size

The global transthyretin amyloidosis treatment market was valued at approximately USD 9.5 billion in 2025. The market is expected to grow from USD 12.1 billion in 2026 to USD 23.7 billion by 2035, expanding at a CAGR of 7.8% during the forecast period, as per the latest report published by Global Market Insights Inc.

The transthyretin amyloidosis (ATTR) treatment market is growing primarily due to the rising diagnosis rates of hereditary and wild-type ATTR amyloidosis, rising awareness of rare cardiac and neurological disorders, and the growing adoption of disease-modifying therapies targeting transthyretin protein stabilisation and gene silencing.

The market includes the management and treatment of ATTR amyloidosis, including hereditary ATTR polyneuropathy (ATTR-PN) and transthyretin amyloid cardiomyopathy (ATTR-CM).

Key players operating in the market include Pfizer, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, AstraZeneca, and BridgeBio Pharma. These companies continue to strengthen their market presence through strategic collaborations, orphan drug development programs, expansion of clinical indications, and investments in next-generation therapies.

The growing burden of ATTR amyloidosis is becoming increasingly evident through epidemiological studies and public health data. According to Orphanet, hereditary ATTR amyloidosis is estimated to affect more than 10,000 individuals globally, while wild-type ATTR amyloidosis prevalence is estimated at nearly 1 in 5,800 individuals worldwide. Also, published studies estimate that approximately 300,000–500,000 individuals worldwide may be living with transthyretin amyloid cardiomyopathy (ATTR-CM).

Moreover, improvements in diagnostic technologies have significantly enhanced early disease identification. Increasing physician awareness and the broader use of non-invasive diagnostic pathways are accelerating patient identification rates globally, thus increasing the target patient population.

Between 2022 and 2024, the market experienced strong growth driven by increasing adoption of tafamidis-based therapies and rapid expansion of RNA-targeted therapeutics. During this period, the global market increased from approximately USD 3.2 billion in 2022 to nearly USD 6.8 billion in 2024, supported by rising diagnostic rates, expanded reimbursement access for rare disease treatment, growing utilization of precision medicine approaches, and ongoing clinical advancements in ATTR cardiomyopathy and polyneuropathy management. Increased screening among elderly cardiovascular patients and advancements in genetic testing further contributed to market expansion across North America, Europe, and parts of Asia Pacific.

Transthyretin Amyloidosis Treatment Market Trends

• A major trend in the market is the transition from supportive symptom management toward targeted disease-modifying therapies. Historically, treatment primarily focused on managing heart failure and neuropathic complications. However, the commercialization of transthyretin stabilizers and RNA-targeted therapies has significantly improved disease control and survival outcomes in both hereditary ATTR polyneuropathy (ATTR-PN) and ATTR cardiomyopathy (ATTR-CM).
• In addition, increasing disease awareness and improved screening initiatives are accelerating diagnosis rates globally. The growing adoption of non-invasive diagnostic technologies is further transforming the treatment landscape.
• Gene-silencing therapies are also emerging as a dominant trend across the market. RNA interference therapies and antisense oligonucleotides that reduce hepatic transthyretin production are demonstrating strong clinical efficacy in slowing neurological impairment and cardiac disease progression.
• The market is also benefiting from favorable rare disease regulatory pathways and orphan drug incentives. Accelerated approvals, orphan drug designations, and priority review programs are encouraging pharmaceutical companies to expand rare disease pipelines and invest in ATTR-focused clinical development programs. For instance, Coramitug (formerly PRX004), a novel amyloid-depleting monoclonal antibody developed by Prothena and Novo Nordisk, received U.S. FDA Fast Track Designation in April 2026 for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM). The designation is intended to accelerate regulatory review for therapies addressing serious unmet medical needs. Such regulatory initiatives are supporting faster commercialization and expanded patient access to advanced therapies.
• Overall, the market is rapidly evolving toward earlier diagnosis, precision medicine, and long-acting disease-modifying therapies. Expanding awareness of ATTR cardiomyopathy among elderly cardiovascular patients, combined with advances in RNA therapeutics, gene editing, and non-invasive diagnostics, is expected to drive sustained market growth globally.

Transthyretin Amyloidosis Treatment Market Analysis

Based on the treatment type, the market is classified into TTR stabilizers and TTR silencers. The TTR stabilizers dominated the market with revenue of USD 6.4 billion in 2025.

• The dominance of the segment can be mainly attributed to the high rate of adoption of tafamidis drugs, especially for wild-type and hereditary cases of ATTR-CM. High revenue generated by this segment is due to the growing utilization of Pfizer’s Vyndaqel and Vyndamax, which became the first FDA-approved therapies for transthyretin amyloid cardiomyopathy (ATTR-CM) in 2019.
• This dominance is further validated by strong clinical trial results indicating the efficacy of transthyretin stabilizers in improving survival rates and lowering the incidence of hospital admissions for cardiovascular disease.
• For example, results from the ATTR-ACT trial revealed that tafamidis resulted in a significant reduction in all-cause mortality rate and cardiovascular disease-related hospital admissions among individuals with hereditary and wild-type transthyretin cardiomyopathy.
• The segment additionally benefits from the convenience of oral administration, an established long-term safety profile, and strong reimbursement support across developed healthcare systems.
• The TTR silencers segment, on the other hand, is expected to witness strong growth at a CAGR of 11.1% during the forecast period, driven by the rising adoption of advanced RNA-based therapies.
• Growing use of therapies such as patisiran and vutrisiran, along with expanding clinical research in ATTR cardiomyopathy, is further supporting segment growth.

Based on disease type, the transthyretin amyloidosis treatment market is classified into ATTR with cardiomyopathy (ATTR-CM) and ATTR with polyneuropathy (ATTR-PN). The ATTR with cardiomyopathy (ATTR-CM) segment is further segmented into wild-type ATTR-CM and hereditary ATTR-CM. The segment held a dominant market share of 80% in 2025.

• ATTR with cardiomyopathy (ATTR-CM) holds a dominant share owing to the increasing prevalence and improved diagnosis of heart disease caused by ATTR.
• Cardiomyopathy caused by ATTR is increasingly being diagnosed in patients experiencing heart failure with preserved ejection fraction (HFpEF), unexplained heart muscle thickening, and aortic stenosis, resulting in an increased pool of patients diagnosed with ATTR-CM.
• In addition, an increase in awareness among cardiologists, coupled with the adoption of better diagnostic techniques such as cardiac MRI, echocardiography, and nuclear medicine techniques, has further increased the detection of patients with cardiac symptoms.
• Furthermore, the increasing prevalence of cardiac symptoms relative to neurological symptoms of transthyretin amyloidosis is continuing to boost growth.

Based on route of administration, the transthyretin amyloidosis treatment market is classified into oral and parenteral. The oral segment held the highest market share in 2025 and is anticipated to grow at a CAGR of 6.6% between 2026 – 2035.

• Oral medications offer convenience and ease of administration, leading to better patient compliance and treatment outcomes.
• Additionally, oral medications have a more favorable safety profile compared to other treatment modalities, reducing the risk of adverse events.
• The development of novel oral therapies targeting transthyretin amyloidosis has further boosted the growth of this segment, providing patients with more treatment options, and potentially improving their quality of life.
• The parenteral route of administration, on the other hand, is expected to grow at a CAGR of 10.5% during the forecast period, driven by the increasing approvals of injectable RNA-based therapies in recent years.
• Therapies such as vutrisiran and eplontersen have expanded treatment options for hereditary ATTR amyloidosis, while several injectable gene-silencing and gene-editing therapies are currently in late-stage clinical pipelines.

Based on distribution channel, the transthyretin amyloidosis treatment market is classified into hospital pharmacies, retail drug stores, and online pharmacies. The hospital pharmacies segment is expected to be valued at USD 10.8 billion by the end of 2035.

• The hospital pharmacies segment dominated the market owing to the specialized nature of ATTR diagnosis and treatment management. Most ATTR therapies, particularly RNA-based and injectable treatments, require administration, monitoring, and follow-up care within hospital or specialty care settings.
• In addition, patients with ATTR cardiomyopathy and polyneuropathy often require multidisciplinary management involving cardiologists, neurologists, and genetic specialists, further increasing dependence on hospital-based dispensing channels.
• The availability of advanced diagnostic facilities, reimbursement support, and access to high-cost orphan drugs through hospital pharmacies also continues to support the segment’s leading market share.

North America Transthyretin Amyloidosis Treatment Market

The North America market dominated the global market with a market share of 60.1% in 2025.

• North America holds a dominant share of the market due to high awareness of rare cardiac disorders, strong adoption of advanced therapies, and the presence of leading pharmaceutical companies developing ATTR-targeted treatments.
• The region benefits from advanced diagnostic infrastructure, including widespread use of cardiac MRI, echocardiography, nuclear scintigraphy, and genetic testing, which has significantly improved diagnosis rates of ATTR cardiomyopathy (ATTR-CM).
• In addition, favorable reimbursement and increasing access to high-cost orphan drugs such as tafamidis and RNA-based therapies continue to support market growth across the region.

The U.S. transthyretin amyloidosis treatment market was valued at USD 1.8 billion and USD 2.5 billion in 2022 and 2023, respectively. The market size reached USD 5.3 billion in 2025, growing from USD 3.9 billion in 2024.

• The U.S. market is driven by increasing diagnoses of ATTR cardiomyopathy among elderly patients with heart failure and unexplained cardiac hypertrophy.
• According to the American Heart Association, approximately 6.7 million adults in the U.S. are living with heart failure, and ATTR-CM is increasingly being identified as an important underlying cause among older adults.
• In addition, NIH-published studies estimate that wild-type ATTR amyloidosis may affect 10–25% of individuals above 80 years of age based on autopsy findings, supporting the growing addressable patient pool.
• The country also benefits from early approvals and strong commercial uptake of therapies such as tafamidis, patisiran, vutrisiran, and eplontersen.
• Moreover, the presence of major biotechnology companies and ongoing ATTR-focused clinical trials continues to accelerate innovation and commercialization of advanced therapies in the U.S. market.

Europe Transthyretin Amyloidosis Treatment Market

Europe market accounted for USD 2 billion in 2025 and is anticipated to show lucrative growth over the forecast period.

• The market growth in Europe is supported by increasing awareness of cardiac amyloidosis, expanding elderly populations, and improved access to rare disease diagnostics.
• According to the European Commission, more than 21% of the European population is aged above 65 years, increasing the risk of age-associated conditions such as wild-type ATTR cardiomyopathy.
• The region also benefits from strong rare disease policies, favorable orphan drug regulations, and active academic research programs focused on amyloidosis treatment and diagnosis.

Germany dominates the Europe market, showcasing strong growth potential.

• The country’s leadership is supported by advanced cardiovascular care infrastructure, strong reimbursement coverage, and early adoption of innovative ATTR therapies.
• Germany has a well-established network of specialty cardiac and neurology centers that facilitate early diagnosis and multidisciplinary disease management.
• In addition, increasing physician awareness regarding ATTR-CM in elderly heart failure patients is contributing to higher screening and treatment rates across the country.

Asia Pacific Transthyretin Amyloidosis Treatment Market

The Asia Pacific market is anticipated to grow at the highest CAGR of 12.6% during the analysis timeframe.

• The Asia Pacific region is expected to witness the fastest growth in the market due to improving awareness of rare diseases, rising healthcare expenditure, and expanding access to advanced diagnostic technologies.
• Large aging populations in countries such as Japan and China are increasing the prevalence of ATTR cardiomyopathy, particularly among elderly patients with heart failure and aortic stenosis.

China market is estimated to grow with a significant CAGR in the Asia Pacific market.

• The market is supported by a large aging population and increasing diagnosis of cardiovascular diseases associated with ATTR cardiomyopathy.
• Rapid improvements in healthcare infrastructure and expansion of tertiary care hospitals are enhancing access to advanced cardiac imaging and rare disease diagnostics.
• In addition, increasing physician awareness and government initiatives supporting rare disease management are expected to improve diagnosis and treatment rates across the country.

Latin America Transthyretin Amyloidosis Treatment Market

Brazil leads the Latin American market, exhibiting remarkable growth during the analysis period.

• The market growth is driven by improving access to specialized healthcare services and increasing awareness of hereditary ATTR amyloidosis in the region.
• Expansion of diagnostic capabilities and rising collaborations with global pharmaceutical companies are supporting earlier disease diagnosis and adoption of therapy.

Middle East and Africa Transthyretin Amyloidosis Treatment Market

Saudi Arabia market is expected to experience substantial growth in the Middle East and Africa market.

• The market growth is supported by increasing investment in advanced healthcare infrastructure and rising awareness of rare cardiovascular diseases.
• In addition, improving access to genetic testing, specialty cardiac care, and rare disease diagnostics is expected to support earlier diagnosis and treatment adoption in the country.
• However, the market remains at a very early stage due to limited disease awareness, underdiagnosis, and restricted access to specialized ATTR therapies across several healthcare settings.

Transthyretin Amyloidosis Treatment Market Share

The market is moderately concentrated, with a few leading pharmaceutical and biotechnology companies accounting for a significant share of total market revenue. Major players such as Pfizer Inc., Alnylam Pharmaceuticals, Ionis Pharmaceuticals, AstraZeneca, and BridgeBio Pharma collectively account for approximately 92% of the global market, driven by strong commercial adoption of transthyretin stabilizers and RNA-targeted therapies. These companies maintain their market position through innovative treatment platforms, orphan drug exclusivity, robust clinical pipelines, and expanding global commercialization strategies.

Companies are actively focusing on strategic collaborations, licensing agreements, and pipeline expansion initiatives to strengthen their presence in both ATTR cardiomyopathy and ATTR polyneuropathy segments. In addition, continued investment in next-generation therapies with improved efficacy, convenient dosing schedules, and broader clinical indications is intensifying market competition.

Transthyretin Amyloidosis Treatment Market Companies

Prominent players operating in the market are as mentioned below:

• Alnylam Pharmaceuticals
• AstraZeneca
• BridgeBio Pharma
• Ionis Pharmaceuticals
• Intellia Therapeutics
• Neurimmune
• Prothena Corporation
• Pfizer
• Pfizer

Pfizer is a leading player in the market through its tafamidis products, Vyndaqel and Vyndamax. In 2019, the U.S. FDA approved these therapies for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), making them the first approved drugs for this indication. The company continues to maintain a strong market presence supported by growing global adoption and expanding diagnosis of ATTR-CM.

• Alnylam Pharmaceuticals

Alnylam Pharmaceuticals is a key innovator in the market with its RNA interference (RNAi)-based therapies targeting transthyretin production. The company received FDA approval for Onpattro in 2018 as the first RNAi therapeutic for hereditary ATTR polyneuropathy, followed by approval of Amvuttra in 2022. These approvals significantly strengthened Alnylam’s position in the ATTR treatment landscape and accelerated adoption of gene-silencing therapies globally.

Transthyretin Amyloidosis Treatment Industry News:

• In November 2024, Alnylam Pharmaceuticals announced that the U.S. FDA accepted the supplemental New Drug Application (sNDA) for vutrisiran for the treatment of transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The application was supported by positive clinical trial results, with the FDA assigning a PDUFA target action date of March 23, 2025.
• In October 2024, Wainzua (eplontersen) received a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR). The recommendation was supported by Phase III clinical trial data showing sustained improvements in neuropathy symptoms and patient quality of life compared to placebo.
• In January 2022, AstraZeneca received Orphan Drug Designation (ODD) in the U.S. by the Food and Drug Administration (FDA) for Eplontersen drug for the treatment of transthyretin-mediated amyloidosis. This helped the company to expand its product portfolio.
• In June 2022, Alnylam Pharmaceuticals, Inc. received FDA authorization for AMVUTTRA (vutrisiran) for the treatment of Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis. This helped the company to market its products in the U.S. and expand its customer base.

The transthyretin amyloidosis treatment market research report includes an in-depth coverage of the industry with estimates & forecast in terms of revenue in USD Million from 2022 - 2035 for the following segments:

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Market, By Treatment Type

• TTR stabilizers
• TTR silencers

Market, By Disease Type

• ATTR with cardiomyopathy (ATTR-CM)     
  • Wild-type ATTR-CM
  • Hereditary ATTR-CM
• ATTR with polyneuropathy (ATTR-PN)       

Market, By Route of Administration

• Oral
• Parenteral

Market, By Distribution Channel

• Hospital pharmacies
• Retail drug stores
• Online pharmacies

The above information is provided for the following regions and countries:

• North America
  • U.S.
  • Canada
• Europe
  • Germany
  • UK
  • France
  • Spain
  • Italy
  • Netherlands
• Asia Pacific
  • Japan
  • China
  • India
  • Australia
  • South Korea
• Latin America
  • Brazil
  • Mexico
  • Argentina
• Middle East and Africa
  • South Africa
  • Saudi Arabia
  • UAE