Transthyretin Amyloidosis Treatment Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Type, By Therapy, By Disease Type, By Distribution Channel, By Region and Competition, 2020-2030F

Global Transthyretin Amyloidosis Treatment Market was valued at USD 4.95 Billion in 2024 and is expected to reach USD 7.45 Billion by 2030 with a CAGR of 7.26% during the forecast period. Transthyretin Amyloidosis (ATTR) is a rare and progressive genetic disorder caused by the buildup of misfolded transthyretin (TTR) proteins in various organs, leading to symptoms such as heart failure, peripheral neuropathy, and gastrointestinal issues. ATTR is divided into two types: hereditary ATTR (hATTR), which is inherited, and wild-type ATTR (wtATTR), which typically affects older adults due to spontaneous amyloid deposition.In recent years, the treatment landscape for ATTR has advanced, with a growing number of therapies designed to slow or halt disease progression. As awareness of ATTR increases and the global population ages, the number of diagnosed cases is expected to rise, particularly among individuals over 50. This heightened awareness is driving greater focus on available treatment options.

Ongoing research and clinical trials are exploring additional therapies, including oral medications and gene therapies, to improve patient outcomes and broaden treatment options. Advances in diagnostic technology have also facilitated earlier detection, which is key for effective intervention. However, the high cost of innovative therapies, especially gene silencing treatments, presents a challenge to market adoption, particularly in regions with limited healthcare budgets. Pricing strategies and reimbursement policies will be crucial factors in the market's growth. Government efforts to include rare disease treatments in insurance plans may help alleviate some of these challenges.

Despite these obstacles, the global ATTR treatment market is expected to experience significant growth, driven by the introduction of new therapies and increasing disease awareness. While cost and access remain challenges, the expanding range of effective treatments and improved diagnostic tools are likely to propel the market forward, offering hope to patients with this debilitating condition. Ongoing research into novel therapies suggests a promising future for the ATTR treatment market, with potential for better patient outcomes and wider market expansion.

Key Market Drivers

Increasing Disease Awareness

The growth of the Global Transthyretin Amyloidosis Treatment Market is intricately linked to the strategic enhancement of disease awareness. As awareness increases, there is a simultaneous uptick in early diagnosis and treatment initiation. This, in turn, fosters a higher demand for therapeutic interventions, propelling market expansion. Elevated disease awareness serves as a catalyst for patient and healthcare provider education, facilitating informed decision-making regarding available treatment options. This heightened awareness not only reduces diagnostic lag but also fuels a proactive approach towards managing transthyretin amyloidosis. In the United States, approximately 1,275 to 3,200 new cases of amyloidosis are diagnosed annually, with AL amyloidosis accounting for 78% of these cases. Familial transthyretin-associated amyloidosis (ATTR), a rarer form of systemic amyloidosis, has an unclear incidence rate, but it is estimated that 10% to 20% of cases diagnosed at specialized centers are attributed to ATTR. Among these, about 7% are hereditary, caused by mutations in the transthyretin gene (ATTRm), while around 6% are age-related, wild-type ATTR (ATTRwt). ATTRwt is more commonly observed in males and was previously referred to as senile systemic amyloidosis. Additionally, secondary (AA) amyloidosis accounts for 6% of all diagnosed amyloidosis cases each year. As awareness of ATTR grows, the recognition of its prevalence in both hereditary and age-related forms is increasing, which is critical for early diagnosis and intervention. Enhanced disease awareness is helping to identify more cases, particularly in specialized centers, and is driving demand for effective treatments. Increased understanding of the different types of amyloidosis, including both rare forms like ATTR and more common types like AL, is also contributing to better-targeted therapies and a growing focus on this condition in clinical research.

Pharmaceutical companies, recognizing this trend, can capitalize on the growing market by investing in targeted marketing campaigns and educational initiatives. Moreover, a well-informed patient population is more likely to actively seek medical assistance, leading to an augmented patient pool for treatment interventions. The resultant surge in demand not only bolsters the market's revenue but also stimulates research and development efforts to innovate novel therapeutic solutions. In essence, the symbiotic relationship between increasing disease awareness, early diagnosis, and heightened demand for treatment modalities forms the cornerstone for the sustained growth of the Global Transthyretin Amyloidosis Treatment Market.

Advancements in Diagnostic Technologies

The growth trajectory of the Global Transthyretin Amyloidosis Treatment Market is poised to ascend with the integration of cutting-edge diagnostic technologies. Advancements in diagnostic tools not only facilitate swift and accurate disease identification but also enable a more nuanced understanding of individual patient profiles. This precision in diagnosis is instrumental in tailoring targeted treatment strategies, thereby propelling the demand for specialized therapeutics. Sophisticated diagnostic technologies, such as genetic testing and biomarker identification, empower healthcare professionals to identify transthyretin amyloidosis at earlier stages, fostering timely intervention. Early detection, in turn, correlates with improved treatment outcomes and patient prognosis. This positive correlation incentivizes both healthcare providers and patients to adopt these advanced diagnostic tools, creating a conducive environment for market expansion.

In November 2024, a single infusion of CRISPR-based therapy developed by Intellia Therapeutics demonstrated promising results in stabilizing a heart disorder caused by transthyretin amyloidosis (ATTR), raising the company's expectations for success in upcoming late-stage clinical trials. Phase 1 data from 36 patients with the cardiomyopathy form of ATTR revealed that the gene-editing treatment significantly and sustainably reduced levels of the misfolded ATTR protein, which forms toxic clumps in the body. Published in The New England Journal of Medicine, the results indicated that these reductions correlated with stabilization or improvement in several key markers of cardiac disease progression. These advancements highlight the growing potential of diagnostic and therapeutic technologies in managing complex diseases like ATTR. Improved diagnostic tools are enabling earlier identification of the disease, allowing for timely interventions with innovative treatments like CRISPR gene editing. This progress not only supports more effective monitoring of disease markers but also offers new hope for patients as therapies evolve to target the root causes of conditions with greater precision and durability. Furthermore, the integration of innovative diagnostic technologies stimulates research and development initiatives, driving continuous improvement in treatment modalities. Pharmaceutical companies, in response to evolving diagnostic capabilities, are incentivized to invest in novel therapies, creating a cycle of innovation that contributes to the overall growth of the market. In essence, the synergy between diagnostic advancements and therapeutic interventions forms a pivotal driver for the Global Transthyretin Amyloidosis Treatment Market.

Patient Advocacy and Support Groups

The growth of the Global Transthyretin Amyloidosis Treatment Market is significantly influenced by the pivotal role played by patient advocacy and support groups. These groups serve as catalysts for heightened disease awareness, patient education, and community engagement. By fostering a sense of community among individuals affected by transthyretin amyloidosis, these advocacy groups create a platform for shared experiences and information exchange. Patient advocacy groups contribute to market growth by actively participating in awareness campaigns, encouraging early diagnosis, and promoting treatment options. Their influence extends to shaping healthcare policies and driving research initiatives, thereby creating an environment conducive to the development and adoption of innovative therapies. Moreover, the collaboration between patient advocacy groups and pharmaceutical companies enhances the development and accessibility of treatment options. By incorporating patient perspectives, these collaborations ensure that therapeutic solutions align with the needs and preferences of the affected community. This not only aids in the market penetration of treatments but also cultivates a supportive ecosystem that encourages patient adherence and long-term engagement with healthcare services. In essence, the symbiotic relationship between patient advocacy and the Global Transthyretin Amyloidosis Treatment Market underscores the significance of a united front in driving awareness, research, and ultimately, the overall growth of the market.

Key Market Challenges

Limited Treatment Options

The growth of the Global Transthyretin Amyloidosis Treatment Market faces a substantial hindrance due to the challenge of limited treatment options. The scarcity of diverse therapeutic interventions constrains the market's potential for expansion, as it narrows the choices available for both healthcare providers and patients. This constraint not only limits the ability to address the varied needs of individuals but also hampers the overall efficacy of managing transthyretin amyloidosis. The lack of treatment diversity poses a barrier to market growth by reducing competitive dynamics among pharmaceutical companies. A dearth of alternative therapies may result in limited innovation and slower development of new treatment modalities. Additionally, it heightens the risk of treatment resistance and suboptimal outcomes for patients, impacting their confidence in available therapeutic solutions. Moreover, from a business perspective, the constrained treatment landscape diminishes the revenue potential for pharmaceutical companies operating in this market. The competitive edge that a diverse product portfolio provides is compromised, impacting market share and profitability.

Diagnosis Challenges

The growth trajectory of the Global Transthyretin Amyloidosis Treatment Market faces impediments due to the substantial challenges associated with diagnosis. The intricate nature of transthyretin amyloidosis often leads to delayed or inaccurate diagnoses, hindering timely intervention and treatment initiation. The complexity of symptoms and the overlap with other medical conditions pose challenges for healthcare professionals, resulting in a diagnostic lag that impacts patient outcomes. Diagnostic challenges contribute to a cascade of issues for the market. Firstly, delayed diagnosis limits the patient pool seeking treatment, restricting market demand. Secondly, the lack of standardized diagnostic protocols hampers the efficiency of clinical trials and research initiatives, impeding the development of new treatment modalities. Additionally, the uncertainty associated with accurate diagnosis may lead to suboptimal treatment choices, affecting patient satisfaction and long-term engagement with therapeutic interventions. From a business perspective, the diagnostic hurdles create a barrier for pharmaceutical companies aiming to position their treatments effectively in the market. The market's potential for growth is constrained by the critical need for streamlined, accurate diagnostic tools that facilitate early and precise identification of transthyretin amyloidosis. Addressing these diagnostic challenges is pivotal for unlocking the full growth potential of the Global Transthyretin Amyloidosis Treatment Market.

Key Market Trends

Advancements in Gene Therapies

The growth trajectory of the Global Transthyretin Amyloidosis Treatment Market is poised for acceleration with the advent of groundbreaking advancements in gene therapies. Innovations in gene-based interventions hold immense promise for revolutionizing the treatment landscape of transthyretin amyloidosis. Gene therapies have the potential to address the root cause of the disease at a molecular level, providing a paradigm shift towards more targeted and efficacious treatment options. The development of gene therapies not only enhances the therapeutic arsenal but also attracts substantial investment and research interest, fostering a competitive market environment. These advancements open new avenues for pharmaceutical companies to differentiate their offerings and tap into a rapidly expanding market segment. Furthermore, gene therapies showcase the potential to provide durable and transformative outcomes for patients, creating a compelling value proposition for both healthcare providers and individuals affected by transthyretin amyloidosis. The growing focus on precision medicine and personalized treatment approaches aligns with the inherent capabilities of gene therapies, driving increased adoption and market penetration. In essence, the integration of advanced gene therapies stands as a key driver propelling the growth of the Global Transthyretin Amyloidosis Treatment Market, heralding a new era of targeted and innovative solutions for this complex and challenging medical condition.

Targeted Therapies and RNA Interference

The Global Transthyretin Amyloidosis Treatment Market is poised for dynamic growth fueled by the emergence of targeted therapies and RNA interference (RNAi) technologies. Targeted therapies, designed to specifically address the underlying mechanisms of transthyretin amyloidosis, offer a more precise and effective treatment approach. This focus on the root cause enhances therapeutic outcomes, reducing side effects and improving overall patient satisfaction. RNA interference, a revolutionary technology that enables the selective silencing of disease-causing genes, holds immense promise for transthyretin amyloidosis. By suppressing the production of aberrant transthyretin protein, RNAi therapies have the potential to halt disease progression at the molecular level. This innovative approach not only diversifies the treatment landscape but also positions the market for significant expansion. The strategic integration of targeted therapies and RNA interference aligns with the industry's trend toward personalized medicine, catering to the unique genetic and molecular profiles of individual patients. In November 2024, BridgeBio received US FDA approval for its lead drug candidate, acoramidis (branded as Attruby), for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), an inherited heart condition. An open-label extension study demonstrated that the drug remained effective over extended periods, with reductions of 34% and 48% in key endpoints at 42 months. Meanwhile, Alnylam Pharmaceuticals, which developed the RNA silencing therapy Amvuttra (vutrisiran) for ATTR-CM, reported strong Phase 3 trial results earlier this year. The company submitted the drug to the FDA for approval in October. These developments reflect key trends in the global transthyretin amyloidosis treatment market, where innovative therapies, such as gene-editing and RNA silencing treatments, are gaining traction. The approval of acoramidis and promising Phase 3 results from Alnylam's Amvuttra underscore a growing pipeline of effective treatments for ATTR-CM, signaling a shift towards more advanced and targeted therapies. As the market continues to expand, the focus on improving patient outcomes through long-term efficacy and precision medicine is expected to drive further growth in the ATTR treatment landscape. As pharmaceutical companies invest in research and development to bring these advanced therapies to market, the resulting innovation will likely drive substantial revenue growth, fostering a competitive and dynamic Global Transthyretin Amyloidosis Treatment Market. In essence, the convergence of targeted therapies and RNA interference technologies marks a transformative phase, shaping the future of transthyretin amyloidosis treatment on a global scale.

Segmental Insights

Disease Type Insights

Based on Disease Type, the Hereditary Transthyretin Amyloidosis emerged as the fastest growing segment in the global market for Transthyretin Amyloidosis Treatment during the forecast period. As awareness of hereditary transthyretin amyloidosis (hATTR) increases and diagnostic capabilities improve, the number of diagnosed cases is rising, especially for polyneuropathy and cardiomyopathy. These conditions can lead to severe complications, significantly impacting patients' quality of life. The emergence of innovative therapies, such as gene silencing treatments (e.g., patisiran and vutrisiran) and protein stabilization agents (e.g., tafamidis), has expanded treatment options for hereditary ATTR, fueling faster growth in this market segment. Additionally, growing focus on rare and genetic diseases, coupled with government incentives for orphan drug development, has driven increased investment and accelerated the development of therapies for hereditary ATTR. The combination of advanced treatment options, a more favorable regulatory environment, and increased disease awareness is expected to continue propelling this segment forward. As a result, the hereditary ATTR market is poised for significant expansion, offering new opportunities for both pharmaceutical companies and patients alike.

Type Insights

Based on Type, ATTR-CM emerged as the dominating segment in the global market for Transthyretin Amyloidosis Treatment in 2024. ATTR-CM primarily impacts the heart, often leading to heart failure, a potentially fatal condition. This can result in severe complications, such as arrhythmias, strokes, and death, making it a critical health concern that demands urgent attention. Due to its life-threatening nature, ATTR-CM has become a major focus for both healthcare professionals and pharmaceutical companies, driving significant attention and growth in the development of treatments for this condition. In recent years, advancements in diagnostic technologies, including imaging and genetic testing, have enhanced the ability to identify more cases of ATTR-CM, especially among older populations. This increased recognition, coupled with the aging global demographic, has led to a rise in diagnoses, expanding the treatment market. The urgent need for effective therapies has also prompted regulatory bodies to fast-track approval for treatments targeting ATTR-CM, recognizing its severity as a rare and critical disease. Additionally, substantial investments from both public and private sectors, along with government incentives for orphan drugs, have fostered an environment that supports the rapid development and adoption of new therapies. As a result, the life-threatening nature of ATTR-CM, coupled with its growing recognition and the focus on developing heart-targeted treatments, are the key drivers behind its market dominance.

Regional Insights

Based on Region, North America emerged as the dominant region in the Global Transthyretin Amyloidosis Treatment Market in 2024. North America benefits from access to state-of-the-art diagnostic technologies, including genetic testing and advanced imaging techniques, which enable earlier and more precise detection of ATTR. Early diagnosis is essential for effective treatment, and the region's advanced healthcare infrastructure enhances this capability, leading to higher diagnosis rates and, in turn, a larger patient population seeking treatment. The well-developed healthcare system in North America also ensures better access to specialized therapies. Moreover, reimbursement policies in the U.S. frequently cover treatments for rare diseases, including orphan drugs, making cutting-edge therapies more accessible to patients. This has attracted significant investment from pharmaceutical companies. Regulatory support from the U.S. Food and Drug Administration (FDA) and Health Canada further strengthens the market, with both agencies offering fast-track approval processes for orphan drugs. This regulatory environment has expedited the availability of several key ATTR treatments in North America. For example, in October 2023, the FDA cleared the Investigational New Drug (IND) application for Intellia Therapeutics' CRISPR-based gene-editing therapy, NTLA-2001, for ATTR amyloidosis with cardiomyopathy (ATTR-CM). Developed in collaboration with Regeneron Pharmaceuticals, NTLA-2001 is part of North America's broader commitment to advancing rare disease therapies. Together, North America's robust healthcare infrastructure, regulatory support, investment in R&D, and high prevalence of ATTR establish the region as the dominant force in the ATTR treatment market.

Key Market Players

• Pfizer Inc.
• Johnson & Johnson Services Inc.
• Ionis Pharmaceuticals Inc.
• Alnylam Pharmaceuticals Inc.
• BridgeBio Pharma Inc.
• Bristol-Myers Squibb Company.
• Acrotech Biopharma.
• AstraZeneca Plc.
• SOM Biotech.

Report Scope:

In this report, the Global Transthyretin Amyloidosis Treatment Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

Transthyretin Amyloidosis Treatment Market, By Type:

• ATTR-PN
• ATTR-CM

Transthyretin Amyloidosis Treatment Market, By Therapy:

• Targeted Therapy (Onpattro, Inotersen, Vyndaqel/Vyndamax)
• Supportive Therapy
• Pipeline Therapy

Transthyretin Amyloidosis Treatment Market, By Disease Type:

• Hereditary Transthyretin Amyloidosis (Polyneuropathy, Cardiomyopathy, Mixed Type)
• Wild Type Amyloidosis

Transthyretin Amyloidosis Treatment Market, By Distribution Channel:

• Hospital Pharmacies
• Specialty Pharmacies
• Retail Pharmacies
• Online Pharmacies

Transthyretin Amyloidosis Treatment Market, By Region:

• North America
  • United States
  • Canada
  • Mexico
• Europe
  • France
  • United Kingdom
  • Italy
  • Germany
  • Spain
• Asia Pacific
  • China
  • India
  • Japan
  • Australia
  • South Korea
• South America
  • Brazil
  • Argentina
  • Colombia
• Middle East & Africa
  • South Africa
  • Saudi Arabia
  • UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Transthyretin Amyloidosis Treatment Market.

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