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Add to CartHereditary Amyloidosis Treatment Market
By Disease Type;
ATTR Amyloidosis and Non-TTR AmyloidosisBy Treatment Type;
Supportive Treatment and Source TreatmentBy Route Of Administration;
Oral, Intravenous, and OthersBy End User;
Hospitals, Specialty Clinics and Inpatient PharmaciesBy Geography;
North America, Europe, Asia Pacific, Middle East & Africa, and Latin America - Report Timeline (2021 - 2031)Hereditary Amyloidosis Treatment Market Overview
Hereditary Amyloidosis Treatment Market (USD Million)
Hereditary Amyloidosis Treatment Market was valued at USD 220.91 million in the year 2024. The size of this market is expected to increase to USD 403.83 million by the year 2031, while growing at a Compounded Annual Growth Rate (CAGR) of 9.0%.
Hereditary Amyloidosis Treatment Market
*Market size in USD million
CAGR 9.0 %
| Study Period | 2025 - 2031 |
|---|---|
| Base Year | 2024 |
| CAGR (%) | 9.0 % |
| Market Size (2024) | USD 220.91 Million |
| Market Size (2031) | USD 403.83 Million |
| Market Concentration | Medium |
| Report Pages | 317 |
Major Players
- Pfizer Inc.
- Ionis Pharmaceuticals, Inc.
- Alnylam Pharmaceuticals, Inc.
- Prothena Corporation plc
- Akcea Therapeutics, Inc.
- Eidos Therapeutics, Inc.
Market Concentration
Consolidated - Market dominated by 1 - 5 major players
Hereditary Amyloidosis Treatment Market
Fragmented - Highly competitive market without dominant players
The Hereditary Amyloidosis Treatment Market is experiencing strong momentum as advanced therapies are introduced to address the rare and complex nature of the disease. With over 60% of treatments now leveraging RNA-targeted approaches, innovation is reshaping patient outcomes. The rise of personalized medicine is unlocking new opportunities, backed by strategic partnerships that promote long-term therapeutic development. These efforts signal rapid growth and expansion across clinical pipelines.
Innovative Trends Driving Growth
Continuous technological advancements are reshaping the landscape of hereditary amyloidosis care, with gene-silencing therapies contributing to more than 55% of the clinical focus. These innovations are enabling collaborations between biotech firms and research institutes to accelerate discovery and validation. The increasing emphasis on targeted therapy enhances treatment effectiveness and offers new strategies for chronic disease management.
Strategic Collaborations and Partnerships
About 58% of pharmaceutical companies involved in the hereditary amyloidosis space are entering merger and partnership agreements to broaden their access to innovative platforms. These collaborations are essential for reducing development timelines and improving drug delivery efficiency. The market is also seeing cross-sector engagement between pharma and digital health, enhancing diagnostic precision and real-time treatment monitoring.
Focus on Future Market Potential
With over 65% of new entrants emphasizing early-stage innovation, the Hereditary Amyloidosis Treatment Market is well-positioned for sustained growth. This focus on early diagnosis tools, combined with increased funding, is transforming the way amyloidosis is identified and managed. The sector is expected to witness greater expansion as digital tools and biomarkers become central to long-term care strategies.
Hereditary Amyloidosis Treatment Market Recent Developments
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In June 2022, The U.S. Food and Drug Administration approved Alnylam Pharmaceuticals' AMVUTTRA™, a novel therapy designed to treat hereditary transthyretin (hATTR) amyloidosis with polyneuropathy. This groundbreaking treatment provided new hope for patients suffering from this progressive and debilitating condition.
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In March 2023, Ionis Pharmaceuticals, based in Carlsbad, California, announced that the US Food and Drug Administration (FDA) accepted for review a New Drug Application (NDA) for eplontersen, an investigational antisense medicine for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
Hereditary Amyloidosis Treatment Market Segment Analysis
In this report, the Hereditary Amyloidosis Treatment Market has been segmented by Disease Type, Treatment Type, Route of Administration, End User, and Geography.
Hereditary Amyloidosis Treatment Market, Segmentation by Disease Type
The Hereditary Amyloidosis Treatment Market has been segmented by Disease Type into ATTR Amyloidosis and Non-TTR Amyloidosis.
ATTR Amyloidosis
ATTR amyloidosis is the most prevalent form of hereditary amyloidosis and is associated with mutations in the transthyretin gene. The growing awareness of its genetic basis has led to an increase in early diagnosis and targeted therapies. Pharmaceutical companies are actively developing gene-silencing and stabilizing agents for this sub-segment. The expansion of newborn screening programs is expected to drive market growth further.
Non-TTR Amyloidosis
Non-TTR amyloidosis encompasses a broader range of hereditary protein misfolding disorders beyond transthyretin. This segment includes rare types such as AApoAI, AGel, and ALys amyloidosis. Limited therapeutic options and delayed diagnosis have hindered rapid growth, but increased investment in genetic research and orphan drug development is creating new opportunities in this niche.
Hereditary Amyloidosis Treatment Market, Segmentation by Treatment Type
The Hereditary Amyloidosis Treatment Market has been segmented by Treatment Type into Supportive Treatment and Source Treatment.
Supportive Treatment
Supportive treatment focuses on managing symptoms and preventing disease progression through the use of diuretics, anti-arrhythmic drugs, and nutritional support. This approach remains dominant in regions with limited access to advanced therapies. Healthcare providers rely on multi-disciplinary care plans to improve quality of life and extend patient survival. Its cost-effectiveness sustains adoption in public healthcare settings.
Source Treatment
Source treatment targets the underlying cause of amyloidosis by reducing or eliminating the production of misfolded proteins. Recent advances in gene silencing therapies, liver transplantation, and TTR stabilizers have revolutionized this sub-segment. High clinical efficacy and FDA approvals for targeted drugs like patisiran and inotersen are propelling market momentum. The growing trend of personalized medicine is a major driver here.
Hereditary Amyloidosis Treatment Market, Segmentation by Route of Administration
The Hereditary Amyloidosis Treatment Market has been segmented by Route of Administration into Oral, Intravenous, and Others.
Oral
Oral administration is preferred for its convenience, adherence, and outpatient suitability. Drugs like tafamidis and diflunisal have become cornerstones of oral therapy for ATTR amyloidosis. Pharmaceutical firms are innovating extended-release formulations to enhance bioavailability. Patient-centric care models and home treatment protocols are further boosting this route.
Intravenous
Intravenous therapy is essential for delivering gene-silencing agents, monoclonal antibodies, and chemotherapy in severe amyloidosis cases. It allows for rapid systemic absorption and is typically administered in hospital or infusion centers. Advancements in IV biologics and increased infusion clinic availability are supporting growth. This route remains vital for acute and advanced-stage treatments.
Others
Other routes include subcutaneous injections and experimental delivery methods such as RNA-based therapies and implantables. Research institutions and biotech firms are exploring novel delivery mechanisms to reduce systemic toxicity. Although still in early stages, these alternatives aim to improve efficacy and patient experience. Their future potential depends on ongoing clinical success and regulatory support.
Hereditary Amyloidosis Treatment Market, Segmentation by End User
The Hereditary Amyloidosis Treatment Market has been segmented by End User into Hospitals, Specialty Clinics, and Inpatient Pharmacies.
Hospitals
Hospitals dominate the end-user landscape due to their capacity for advanced diagnostic tools, multidisciplinary care teams, and access to high-cost biologics. These institutions are the primary treatment centers for severe or newly diagnosed cases. Centralized procurement of orphan drugs also favors this setting. Continued investment in rare disease infrastructure supports long-term demand.
Specialty Clinics
Specialty clinics focus on treating rare and genetic disorders, making them critical in early intervention, monitoring, and personalized care. Their expertise in hereditary amyloidosis enables quicker diagnosis and tailored therapy plans. As outpatient centers, they help reduce healthcare costs and hospital burden. Growing patient awareness and improved referral systems are increasing clinic visits.
Inpatient Pharmacies
Inpatient pharmacies play a crucial role in drug preparation, dosage accuracy, and inventory management for complex treatments. They work closely with hospital units to ensure safe delivery of orphan drugs and biologics. The increase in hospital-based infusion therapies and regulatory compliance needs is elevating their importance. Digital pharmacy systems are further streamlining operations.
Hereditary Amyloidosis Treatment Market, Segmentation by Geography
In this report, the Hereditary Amyloidosis Treatment Market has been segmented by Geography into North America, Europe, Asia Pacific, Middle East & Africa, and Latin America.
Regions and Countries Analyzed in this Report
Hereditary Amyloidosis Treatment Market Share (%), by Geographical Region
North America
North America leads the market with approximately 39% share due to strong regulatory frameworks, advanced diagnostic infrastructure, and high orphan drug uptake. The U.S. in particular benefits from FDA fast-track approvals and widespread insurance coverage. Patient advocacy groups and precision medicine initiatives further stimulate growth in this region.
Europe
Europe holds around 28% of the market, supported by national rare disease programs, research funding, and coordinated care models. Countries like Germany, France, and the UK have established amyloidosis centers of excellence. EMA designations for orphan drugs ensure faster access. Cross-border health collaborations enhance clinical trial and treatment availability.
Asia Pacific
Asia Pacific accounts for nearly 20% of global share, with rapid expansion in genetic testing, academic research, and specialty pharma presence. Japan leads in patient registry initiatives, while China and India are investing in rare disease diagnostics. Government policies promoting local biotech innovations are improving accessibility across emerging markets.
Middle East & Africa
This region holds approximately 7% share and is witnessing gradual improvements in diagnosis rates, referral systems, and private healthcare participation. Gulf nations are spearheading rare disease registries and funding niche therapies. However, limited reimbursement and awareness challenge wider adoption. International partnerships are key to long-term development.
Latin America
Latin America comprises about 6% of the market, led by Brazil, Mexico, and Argentina. Access to orphan drugs is improving through public-private collaboration, NGO involvement, and telemedicine. Regulatory harmonization and patient education are enhancing early intervention. Clinical trials and academic research are emerging steadily in this region.
Market Trends
This report provides an in depth analysis of various factors that impact the dynamics of Global Hereditary Amyloidosis Treatment Market. These factors include; Market Drivers, Restraints and Opportunities Analysis.
Drivers, Restraints and Opportunity Analysis
Drivers:
- Increasing Awareness and Diagnosis Rates
- Advances in Research and Development
- Growing Pipeline of Therapeutic Candidates
- Supportive Regulatory Environment
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Rising Healthcare Expenditure- Rising healthcare expenditure represents a significant driver shaping the Global Hereditary Amyloidosis Treatment Market, as increased investment in healthcare infrastructure and services enables broader access to diagnosis, treatment, and supportive care for individuals affected by hereditary amyloidosis. As healthcare expenditure grows globally, governments, insurers, and healthcare organizations allocate greater resources towards addressing rare and complex diseases, including hereditary amyloidosis. This heightened investment facilitates the development and adoption of innovative therapies, supports research and development initiatives, and enhances patient access to specialized healthcare services, ultimately driving market growth and improving patient outcomes.
Rising healthcare expenditure fosters collaborations between industry stakeholders, healthcare providers, and regulatory agencies, leading to the development of targeted interventions and treatment modalities tailored to the unique needs of patients with hereditary amyloidosis. With increased funding available for research, clinical trials, and patient support programs, market players can accelerate the pace of innovation and bring novel therapies to market more efficiently. Additionally, investments in healthcare infrastructure and training programs enhance healthcare delivery capabilities, enabling healthcare professionals to provide high-quality, multidisciplinary care to individuals affected by hereditary amyloidosis. As healthcare expenditure continues to rise, the Global Hereditary Amyloidosis Treatment Market is poised to benefit from expanded access to care, improved treatment outcomes, and a more favorable regulatory environment conducive to innovation and growth.
Restraints:
- Limited Understanding of Disease Mechanisms
- High Cost of Treatment
- Lack of Specific Biomarkers for Diagnosis
- Challenges in Patient Recruitment for Clinical Trials
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Adverse Events Associated with Therapeutic Interventions- Adverse events associated with therapeutic interventions represent a significant challenge in the Global Hereditary Amyloidosis Treatment Market, posing risks to patient safety and potentially limiting the widespread adoption of certain treatment modalities. While pharmacological therapies and surgical interventions aim to alleviate symptoms and slow disease progression, they may also be accompanied by adverse effects ranging from mild discomfort to severe complications. These adverse events can arise due to the pharmacological properties of medications, interactions with underlying health conditions, or individual patient factors, highlighting the importance of vigilant monitoring and personalized treatment approaches in managing hereditary amyloidosis.
The occurrence of adverse events underscores the need for comprehensive risk assessment, patient education, and close monitoring throughout the treatment process. Healthcare providers play a crucial role in identifying and managing adverse events, promptly addressing concerns, and adjusting treatment regimens as needed to optimize patient safety and tolerability. Additionally, ongoing pharmacovigilance efforts and post-market surveillance initiatives are essential for detecting and reporting adverse events associated with therapeutic interventions, enabling continuous evaluation of treatment safety profiles and informing regulatory decision-making. By addressing adverse events proactively and implementing risk mitigation strategies, stakeholders in the Global Hereditary Amyloidosis Treatment Market can enhance patient outcomes, build trust in treatment modalities, and foster a supportive and patient-centered approach to care.
Opportunities:
- Expansion of Target Patient Population
- Emerging Markets with Untapped Potential
- Development of Disease-Modifying Therapies
- Collaboration and Partnerships in Research
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Integration of Precision Medicine Approaches- The integration of precision medicine approaches represents a significant advancement in the Global Hereditary Amyloidosis Treatment Market, offering personalized and targeted therapies tailored to the specific genetic mutations and clinical profiles of individuals affected by hereditary amyloidosis. Precision medicine leverages genomic sequencing, biomarker analysis, and advanced diagnostic techniques to identify genetic mutations associated with hereditary amyloidosis, enabling healthcare providers to customize treatment strategies based on individual patient characteristics. By understanding the underlying molecular mechanisms driving disease progression, precision medicine approaches aim to optimize therapeutic outcomes, minimize adverse effects, and improve overall patient prognosis.
Precision medicine holds promise for accelerating the development of novel therapies and advancing research efforts in hereditary amyloidosis. By identifying biomarkers and molecular targets associated with disease pathogenesis, researchers can identify potential drug candidates, design targeted interventions, and conduct clinical trials with greater precision and efficiency. Additionally, precision medicine approaches facilitate the identification of at-risk individuals and asymptomatic carriers of hereditary amyloidosis mutations, enabling early intervention and preventive measures to mitigate disease progression and improve long-term outcomes. As precision medicine continues to evolve and gain prominence in healthcare, its integration into the Global Hereditary Amyloidosis Treatment Market offers new avenues for personalized care, innovative therapeutics, and improved management of this complex and rare genetic disorder.
Competitive Landscape Analysis
Key players in Global Hereditary Amyloidosis Treatment Market include,
- Pfizer Inc.
- Ionis Pharmaceuticals, Inc.
- Alnylam Pharmaceuticals, Inc.
- Prothena Corporation plc
- Akcea Therapeutics, Inc.
- Eidos Therapeutics, Inc.
In this report, the profile of each market player provides following information:
- Company Overview and Product Portfolio
- Key Developments
- Financial Overview
- Strategies
- Company SWOT Analysis
- Introduction
- Research Objectives and Assumptions
- Research Methodology
- Abbreviations
- Market Definition & Study Scope
- Executive Summary
- Market Snapshot, By Disease Type
- Market Snapshot, By Treatment Type
- Market Snapshot, By Route Of Administration
- Market Snapshot, By End User
- Market Snapshot, By Region
- Hereditary Amyloidosis Treatment Market Dynamics
- Drivers, Restraints and Opportunities
- Drivers
- Increasing Awareness and Diagnosis Rates
- Advances in Research and Development
- Growing Pipeline of Therapeutic Candidates
- Supportive Regulatory Environment
- Rising Healthcare Expenditure
- Restraints
- Limited Understanding of Disease Mechanisms
- High Cost of Treatment
- Lack of Specific Biomarkers for Diagnosis
- Challenges in Patient Recruitment for Clinical Trials
- Adverse Events Associated with Therapeutic Interventions
- Opportunities
- Expansion of Target Patient Population
- Emerging Markets with Untapped Potential
- Development of Disease-Modifying Therapies
- Collaboration and Partnerships in Research
- Integration of Precision Medicine Approaches
- Drivers
- PEST Analysis
- Political Analysis
- Economic Analysis
- Social Analysis
- Technological Analysis
- Porter's Analysis
- Bargaining Power of Suppliers
- Bargaining Power of Buyers
- Threat of Substitutes
- Threat of New Entrants
- Competitive Rivalry
- Drivers, Restraints and Opportunities
- Market Segmentation
- Hereditary Amyloidosis Treatment Market, By Disease Type, 2021 - 2031 (USD Million)
- ATTR amyloidosis
- Non-TTR amyloidosis
- Hereditary Amyloidosis Treatment Market, By Treatment Type, 2021 - 2031 (USD Million)
- Supportive treatment
- Source treatment
- Hereditary Amyloidosis Treatment Market, By Route Of Administration, 2021 - 2031 (USD Million)
- Oral
- Intravenous
- Others
- Hereditary Amyloidosis Treatment Market, By End User, 2021 - 2031 (USD Million)
- Hospitals
- Specialty Clinics
- Inpatient Pharmacies
- Hereditary Amyloidosis Treatment Market, By Geography, 2021 - 2031 (USD Million)
- North America
- United States
- Canada
- Europe
- Germany
- United Kingdom
- France
- Italy
- Spain
- Nordic
- Benelux
- Rest of Europe
- Asia Pacific
- Japan
- China
- India
- Australia & New Zealand
- South Korea
- ASEAN (Association of South East Asian Countries)
- Rest of Asia Pacific
- Middle East & Africa
- GCC
- Israel
- South Africa
- Rest of Middle East & Africa
- Latin America
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- North America
- Hereditary Amyloidosis Treatment Market, By Disease Type, 2021 - 2031 (USD Million)
- Competitive Landscape
- Company Profiles
- Pfizer Inc.
- Ionis Pharmaceuticals, Inc.
- Alnylam Pharmaceuticals, Inc.
- Prothena Corporation plc
- Akcea Therapeutics, Inc.
- Eidos Therapeutics, Inc.
- Company Profiles
- Analyst Views
- Future Outlook of the Market