Verified Insights
Precision-driven research you can trust. We uphold rigorous data validation processes to ensure every report is reliable and based on credible sources.
+91 9425150513 (Asia) support@24lifesciences.com
+91 9425150513 (Asia) support@24lifesciences.com
Download Sample Pdf
MARKET INSIGHTS
The global Transthyretin Amyloidosis (ATTR) treatment market was valued at approximately USD 1.5 billion in 2024. The market is projected to experience significant expansion, reaching an estimated USD 3.2 billion by 2032, exhibiting a robust compound annual growth rate (CAGR) of over 10% during the forecast period.
Transthyretin amyloidosis is a progressive and potentially fatal disease characterized by the buildup of abnormal amyloid fibrils composed of misfolded transthyretin protein in various tissues and organs. This condition manifests primarily in two forms: hereditary ATTR amyloidosis (hATTR), caused by a genetic mutation, and wild-type ATTR amyloidosis (wtATTR), which occurs sporadically in older adults, often affecting the heart. Current treatments focus on stabilizing the TTR protein, silencing its production, or clearing the amyloid deposits. These therapeutic classes include TTR stabilizers (e.g., tafamidis, diflunisal), gene silencers (e.g., patisiran, inotersen), and a newly approved amyloid fibril remover.
The market is experiencing rapid growth due to several factors, including increasing disease awareness and diagnosis rates, a growing aging population susceptible to wild-type ATTR, and the successful launch of novel, effective therapies. Recent approvals, such as the August 2023 FDA approval of BridgeBio Pharma's acoramidis, highlight the active and promising pipeline. Furthermore, the rising prevalence of heart failure with preserved ejection fraction (HFpEF), a significant portion of which is now attributed to wtATTR amyloidosis, is a major driver. Key players like Pfizer Inc., Alnylam Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. are heavily invested in this space, continuously advancing treatment paradigms.
Increasing Disease Awareness and Improved Diagnostic Capabilities
Enhanced recognition of ATTR amyloidosis, encompassing both hereditary (ATTRv) and wild-type (ATTRwt) forms, among clinicians is a primary driver. Improved diagnostic tools, including targeted imaging with bone scintigraphy and precise biomarker assays, have significantly reduced the historically lengthy diagnostic odyssey. This leads to earlier patient identification and expands the addressable patient population for treatment.
Approval and Adoption of Targeted Therapies
The regulatory approval of disease-modifying therapies has transformed the treatment landscape. The introduction and widespread adoption of TTR stabilizers like tafamidis and diflunisal, along with RNA-targeted therapies such as patisiran and inotersen, provide clinically proven options to slow disease progression. These therapies have established a robust and growing revenue stream, driving market expansion. The proven efficacy in improving quality of life and functional capacity for patients with cardiomyopathy and polyneuropathy is a key factor.
➤ The global market for ATTR amyloidosis treatments is projected to experience significant growth, with some estimates suggesting it may exceed $7 billion by 2030, fueled by new drug launches and geographic expansion.
Furthermore, the continued investment in clinical trials for next-generation therapies, including CRISPR-based gene-editing treatments, underscores a strong pipeline that sustains market momentum and investor confidence.
MARKET CHALLENGES
High Cost of Therapy and Reimbursement Hurdles
The annual cost of branded ATTR amyloidosis treatments is exceptionally high, often exceeding hundreds of thousands of dollars per patient. This creates substantial barriers to patient access, particularly in cost-sensitive healthcare systems. Navigating complex and often restrictive reimbursement policies from public and private payers remains a significant challenge for manufacturers and patients alike, potentially limiting market penetration.
Other Challenges
Diagnostic Delays and Misdiagnosis
Despite improvements, ATTR amyloidosis is still frequently misdiagnosed as more common conditions like hypertrophic cardiomyopathy or chronic inflammatory demyelinating polyneuropathy. This delay in accurate diagnosis shortens the window for effective therapeutic intervention and continues to limit the treated patient population.
Treatment Administration and Monitoring
Some advanced therapies require specialized administration, such as regular intravenous infusions, which can burden healthcare infrastructure and patients. Additionally, certain treatments necessitate rigorous monitoring for potential side effects, adding complexity and cost to long-term management.
Small and Fragmented Patient Population
ATTR amyloidosis is a rare disease, with an estimated prevalence that, while growing due to better diagnosis, remains limited globally. The market is inherently constrained by this small patient base. Furthermore, the disease manifests in distinct phenotypes (cardiomyopathy vs. polyneuropathy) and genotypes, leading to a fragmented market that requires targeted drug development and commercial strategies, increasing the cost per patient.
Stringent Regulatory Pathways for Rare Diseases
While orphan drug designation provides incentives, the regulatory pathway for rare disease treatments is rigorous. Demonstrating significant efficacy in small patient populations poses statistical challenges. Post-marketing requirements and the need for long-term safety data add to the development burden, potentially slowing the entry of new products and restraining market growth.
Expansion into Untapped Geographic Markets
Current treatment penetration is highest in North America, Europe, and Japan. Significant opportunities exist in expanding access to emerging markets across Asia-Pacific and Latin America, where improving healthcare infrastructure and rising disease awareness are creating new demand. Tailoring market access and pricing strategies for these regions is a key growth avenue.
Development of Combination Therapies and Curative Approaches
There is a substantial opportunity for developing regimens that combine TTR stabilizers with TTR silencers to potentially enhance efficacy. Furthermore, the advancement of one-time gene-editing therapies represents a paradigm shift towards a potential functional cure. These innovative approaches could command premium pricing and capture significant market share if successfully developed.
Focus on Asymptomatic Carrier Populations
Treating individuals who carry disease-causing TTR mutations but have not yet developed symptoms (penetrant carriers) is a major future opportunity. Clinical trials are exploring whether pre-emptive treatment can prevent or delay disease onset, which would substantially expand the treatable population and create a new, proactive treatment paradigm.
Segment Analysis:| Segment Category | Sub-Segments | Key Insights |
| By Type |
|
ATTR-CM represents a significant portion of the market focus due to the high disease burden and associated mortality, driving extensive research and development efforts for treatments that can stabilize cardiac function and improve patient survival. The neurologically-focused ATTR-PN segment, while smaller in patient population, commands attention for its debilitating impact on quality of life, leading to a high value therapeutic area with several innovative drugs in development aimed at halting disease progression and managing symptoms. |
| By Application |
|
Specialty Pharmacies are the dominant channel for dispensing transthyretin amyloidosis treatments, as they are uniquely equipped to manage the complexities of these high-cost, injectable, and often specialty-tier medications, providing essential patient support services, adherence monitoring, and reimbursement assistance. While Hospital Pharmacies are crucial for initial diagnosis and inpatient treatment initiation, the trend is shifting towards specialty and online pharmacies for long-term management, offering greater convenience and specialized care coordination for patients with this chronic condition. |
| By End User |
|
Hospitals serve as the primary end-user segment, acting as the central hub for diagnosis, complex multidisciplinary care, and administration of therapies for both ATTR-CM and ATTR-PN patients, often involving cardiology and neurology departments. The growing role of Specialty Clinics is notable, particularly amyloidosis centers of excellence, which provide highly specialized, coordinated care that is essential for managing this rare disease, often leading to better patient outcomes and serving as important sites for clinical trial recruitment and advanced treatment access. |
| By Drug Class |
|
Gene Silencers represent the most innovative and rapidly evolving segment, with RNA-targeted therapies showing high efficacy in reducing the production of the mutant transthyretin protein, fundamentally altering the disease course and generating significant market interest. Stabilizers continue to hold a strong position as foundational therapies that can slow disease progression by preventing the misfolding of the TTR protein, while treatments for symptom management remain critical for addressing the multifaceted clinical manifestations of the disease and improving patients' daily living. |
| By Route of Administration |
|
Subcutaneous administration is gaining prominence as a leading segment due to patient preference for at-home or clinic-based administration that offers greater convenience and improved quality of life compared to frequent intravenous infusions. Although Intravenous formulations are well-established for certain therapies, the development pipeline shows a clear trend towards subcutaneous options, while Oral medications provide a crucial option for patients seeking the utmost convenience, though their therapeutic targets may differ from the more potent injectable disease-modifying agents. |
Innovation and Strategic Alliances Drive Market Dynamics
The competitive landscape of the Transthyretin Amyloidosis (ATTR) treatment market is characterized by the dominance of a few key players who have pioneered novel therapeutic approaches and secured significant market share. The market has been revolutionized by the introduction of RNA-targeting therapies, which directly address the underlying cause of the disease by reducing the production of the misfolded transthyretin protein. Alnylam Pharmaceuticals, Inc. is a clear leader with its groundbreaking drug, Onpattro® (patisiran), the first-ever RNAi therapeutic approved for the treatment of polyneuropathy of hereditary ATTR amyloidosis. Pfizer Inc. also holds a substantial position through its stabilizer drug, Vyndaqel®/Vyndamax® (tafamidis), which has become a cornerstone treatment for ATTR cardiomyopathy. Ionis Pharmaceuticals, Inc., through its partner Akcea Therapeutics (now part of Ionis), developed Tegsedi® (inotersen), another significant RNA-targeted therapy. These companies drive the market through continuous clinical development, global commercialization efforts, and strategic expansion of treatment indications.
Beyond the leaders, the market features a dynamic mix of established pharmaceutical giants and specialized biotechnology firms carving out significant niches. Companies like BridgeBio Pharma, Inc. are advancing next-generation therapies, such as acoramidis, aiming to improve upon existing standards of care. Prothena is developing PRX004, an anti-amyloid monoclonal antibody, representing a different mechanistic approach. Eidos Therapeutics, a BridgeBio affiliate, was instrumental in the development pathway for ATTR-CM treatments. Furthermore, large diversified players like Johnson & Johnson, Bristol-Myers Squibb Company, and AstraZeneca contribute through their extensive R&D pipelines and global reach, often exploring combination therapies or novel targets. Smaller, research-focused entities like SOM Biotech utilize advanced computational methods for drug repurposing, adding to the diversity of the development landscape. The market is also witnessing increased M&A activity and collaborations, as companies seek to bolster their ATTR portfolios and leverage complementary expertise.
List of Key Transthyretin Amyloidosis Treatment Companies ProfiledPfizer Inc.
BridgeBio Pharma, Inc.
Bristol-Myers Squibb Company
Acrotech Biopharma
AstraZeneca
Astellas Pharma, Inc.
SOM Biotech
Eidos Therapeutics, Inc. (a BridgeBio company)
Intellia Therapeutics, Inc.
Akcea Therapeutics (part of Ionis Pharmaceuticals)
The global Transthyretin Amyloidosis (ATTR) Treatment market is on a significant growth trajectory, valued at a substantial figure in 2024 and projected to expand to a multi-million dollar market by 2032. This robust compound annual growth rate is primarily driven by extensive research and development in novel therapies. The existence of a strong pipeline of drugs, including gene silencers and stabilizers, promises to address the underlying causes of both hereditary and wild-type ATTR amyloidosis. The development of these targeted treatments reflects a broader trend in pharmaceuticals towards precision medicine for rare diseases, moving beyond symptomatic management to disease-modifying interventions.
Other TrendsSegmentation by Disease Type and Distribution Channel
The market is distinctly segmented by disease type into ATTR polyneuropathy (ATTR-PN) and ATTR cardiomyopathy (ATTR-CM), each presenting unique clinical challenges and driving specific therapeutic demand. Regarding distribution, specialty pharmacies currently hold a dominant market share, a trend attributed to the complex handling and patient support services required for these high-cost, specialty medications. However, online pharmacies are projected to be the fastest-growing segment, reflecting a broader shift towards digital health platforms and improved patient access, particularly in remote areas.
Regional Dynamics and Competitive LandscapeNorth America leads the global market, a position supported by high disease awareness, advanced healthcare infrastructure, and favorable reimbursement policies. Europe follows as a significant market, with Asia-Pacific showing the highest growth potential due to improving diagnostic capabilities and increasing healthcare expenditure. The competitive landscape is characterized by key players such as Pfizer, Alnylam Pharmaceuticals, and Ionis Pharmaceuticals. The market is witnessing strategic collaborations and acquisitions aimed at expanding product portfolios and geographic reach, intensifying competition and driving further innovation in treatment options.
Regional Analysis: Transthyretin Amyloidosis Treatment MarketEurope
Europe represents a major and highly developed market for ATTR amyloidosis treatments, characterized by strong national healthcare systems and coordinated regulatory oversight through the European Medicines Agency (EMA). Market growth is driven by increasing diagnostic rates, particularly for wild-type ATTR cardiomyopathy, which is being recognized more frequently in aging populations. Country-specific healthcare budgets and health technology assessment (HTA) processes, such as those by NICE in the UK and the G-BA in Germany, play a pivotal role in determining reimbursement and market access. While this can lead to variations in adoption speed between countries, the overall trend is toward greater acceptance of new therapies. Collaborative networks of amyloidosis centers across the continent ensure that expertise is shared, promoting standardized care and supporting market penetration.
Asia-Pacific
The Asia-Pacific region is identified as a high-growth market for ATTR amyloidosis treatments, albeit from a smaller base. Growth is primarily fueled by improving healthcare infrastructure, rising medical awareness, and increasing investments from multinational pharmaceutical companies seeking new growth opportunities. Japan is the most mature market in the region, with a well-established system for approving and reimbursing orphan drugs. In larger emerging economies, challenges remain, including lower disease awareness among physicians, diagnostic delays, and budget constraints within public healthcare systems. However, the vast and aging population presents a significant potential patient pool, particularly for wild-type ATTR, driving long-term market potential. Local clinical trials and partnerships are increasingly common, aiming to tailor strategies to diverse healthcare landscapes.
South America
The ATTR treatment market in South America is emerging and currently characterized by nascent development. Access to modern therapeutics is often limited to major urban centers and private healthcare sectors in countries like Brazil and Argentina. A significant barrier is the high cost of novel treatments relative to public healthcare budgets, leading to restricted reimbursement and accessibility challenges. Disease awareness is growing but remains lower than in North America and Europe, contributing to underdiagnosis. Despite these hurdles, there is a gradual increase in clinical trial activity and efforts by patient advocacy groups to improve diagnosis and care, indicating the beginnings of market evolution and future growth potential.
Middle East & Africa
The Middle East and Africa region represents a highly variable and developing market for ATTR amyloidosis treatments. The Gulf Cooperation Council (GCC) countries, with their higher healthcare expenditure, show better access to advanced diagnostics and treatments, often importing therapies available in Western markets. In contrast, access across most of Africa is extremely limited, constrained by infrastructure challenges, low awareness, and significant cost barriers. The market is largely opportunistic and concentrated in private healthcare settings in urban areas. Efforts are focused on improving genetic counseling and basic diagnostic capabilities, with market growth expected to be slow and uneven across the vast region.
This market research report offers a holistic overview of global and regional markets for the forecast period 2025–2032. It presents accurate and actionable insights based on a blend of primary and secondary research.
✅ Market Overview
Global and regional market size (historical & forecast)
Growth trends and value/volume projections
✅ Segmentation Analysis
By product type or category
By application or usage area
By end-user industry
By distribution channel (if applicable)
✅ Regional Insights
North America, Europe, Asia-Pacific, Latin America, Middle East & Africa
Country-level data for key markets
✅ Competitive Landscape
Company profiles and market share analysis
Key strategies: M&A, partnerships, expansions
Product portfolio and pricing strategies
✅ Technology & Innovation
Emerging technologies and R&D trends
Automation, digitalization, sustainability initiatives
Impact of AI, IoT, or other disruptors (where applicable)
✅ Market Dynamics
Key drivers supporting market growth
Restraints and potential risk factors
Supply chain trends and challenges
✅ Opportunities & Recommendations
High-growth segments
Investment hotspots
Strategic suggestions for stakeholders
✅ Stakeholder Insights
This report is designed to support strategic decision-making for a wide range of stakeholders, including:
Pharmaceutical and biotech companies
Medical device and diagnostics manufacturers
Healthcare providers and hospital systems
Contract research and manufacturing organizations
Investors, consultants, and policy makers
-> Global Transthyretin Amyloidosis Treatment market was valued at USD 1.5 billion in 2024 and is expected to reach USD 3.2 billion by 2032.
-> Key players include Pfizer Inc., Alnylam Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., and BridgeBio Pharma, Inc., among others.
-> The market is expected to grow at a CAGR of over 10% during 2024-2032.
-> Key growth drivers include increasing disease awareness, aging population, and novel therapy approvals.
-> Major treatments include TTR stabilizers, gene silencers, and amyloid fibril removers.
Our Clients
“The data provided by 24LifeScience was clear, well-organized, and useful for internal strategy planning. It helped us understand the competitive landscape more effectively.”
“We used one of their market overview reports for early-stage feasibility work. It gave us a helpful snapshot of current trends and key players in our therapeutic area.”
“I appreciated the team’s responsiveness and willingness to adjust the scope based on our feedback. The final report was aligned with our expectations and timelines.”
“Their custom report on clinical trial trends was a helpful reference as we explored new indications."
“As someone working on early product planning, I found their therapeutic area briefs quite useful. The information was presented in a way that made it easy to extract key takeaways.”
“We didn’t need anything overly complex—just solid, dependable data. 24LifeScience delivered exactly that, without unnecessary fluff.”
“Their reports gave us a good foundation to start our own market assessment. While we supplemented it with other data, this was a great starting point.”
“I’ve used a few of their reports for academic and grant writing purposes. They’re generally well-cited and reliable for understanding market scope.”
At 24LifeScience, we combine domain expertise with dependable research delivery. What truly differentiates us isn't just what we do — it's how we do it. Our clients trust us because we offer consistency, security, value, and most importantly, insight that drives action.
Precision-driven research you can trust. We uphold rigorous data validation processes to ensure every report is reliable and based on credible sources.
We uphold rigorous data validation processes to ensure every report is reliable, up-to-date, and based on credible sources.
24LifeScience powers research for top firms in 20+ nations.Chosen by leading life sciences companies worldwide.
We offer competitive pricing models that align with your project scope — no hidden charges, no lock-in. Tailored pricing for every scale and need.
8–10+ years of life sciences expertise turned into strategic insights.We don’t just summarize data we contextualize it.
Whether it's a ready-made report or a custom project, we deliver within the promised timeline With real-time updates