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Add to CartHereditary Amyloidosis Treatment Market
By Treatment Type;
Medications and Supportive TreatmentsBy Route of Administration;
Oral, Intravenous, Subcutaneous and IntramuscularBy Disease Type;
AL Amyloidosis, ATTR Amyloidosis and Aβ AmyloidosisBy Treatment Setting;
Hospital Outpatient Clinics, Home Healthcare, Specialized Clinics and Long-Term Care FacilitiesBy 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 Treatment Type, Route of Administration, Disease Type, Treatment Setting and Geography. The structure clarifies therapeutic drivers, care-delivery challenges, and commercialization pathways across rare-disease ecosystems. It also surfaces opportunities for partnerships, patient-support models, and technology-enabled monitoring that shape access, adherence, and future pipeline momentum.
Hereditary Amyloidosis Treatment Market, Segmentation by Treatment Type
The Treatment Type lens distinguishes between disease-modifying Medications and multi-disciplinary Supportive Treatments that alleviate organ burden. Market evolution reflects advances in stabilizers, gene-silencing approaches, and expanded diagnostic screening, alongside optimized symptom management. Strategic alliances with specialty pharmacies and centers of excellence streamline titration, monitoring, and long-term outcomes tracking.
Medications
Medications address pathogenic protein production, stabilization, or deposition, anchoring the value proposition for earlier diagnosis and organ protection. Payers evaluate durability, safety, and quality-of-life impact, making real-world evidence and registries central to market access. Co-deployment with genetic counseling, biomarker monitoring, and adherence programs enhances clinical and economic outcomes.
Supportive Treatments
Supportive Treatments span multidisciplinary interventions—cardiac, neurologic, renal, and pain management—to maintain function and reduce complications. Protocols emphasize symptom control, rehabilitation, and nutrition, often coordinated through specialized clinics and home-based services. Integration with digital remote monitoring and palliative frameworks sustains patient-centered care across disease stages.
Hereditary Amyloidosis Treatment Market, Segmentation by Route of Administration
The Route of Administration dimension—Oral, Intravenous, Subcutaneous, and Intramuscular—captures trade-offs in convenience, bioavailability, monitoring needs, and site-of-care logistics. Manufacturers tailor dosing schedules and delivery formats to reduce burden on patients and systems. Supply-chain reliability, cold-chain considerations, and training support influence adoption velocity across settings.
Oral
Oral therapies prioritize ease of use and long-term management, supporting adherence through routine dosing and patient education. Programs that address drug–drug interactions, adherence reminders, and insurance navigation can further stabilize persistence. Real-world data on functional outcomes and tolerability help maintain favorable positioning on formularies.
Intravenous
Intravenous administration enables controlled delivery and close observation, often initiated in hospital outpatient or infusion centers. Scheduling, chair time, and premedication protocols are optimized to reduce infusion-related events and operational strain. Partnerships with infusion networks and home-infusion providers expand capacity while preserving safety.
Subcutaneous
Subcutaneous options balance systemic exposure with patient convenience, increasingly supported by on-body devices and nurse-led training. Reduced clinic visits, self-administration, and predictable pharmacokinetics support quality-of-life gains. Stakeholders emphasize device usability, cold-chain integrity, and data capture to document outcomes.
Intramuscular
Intramuscular delivery is considered where streamlined dosing and clinic-based oversight are preferred. Protocols focus on injection-site tolerability, technique standardization, and documentation within specialty clinics. As portfolios diversify, IM routes complement broader strategies for access and adherence.
Hereditary Amyloidosis Treatment Market, Segmentation by Disease Type
The Disease Type segmentation—AL Amyloidosis, ATTR Amyloidosis, and Aβ Amyloidosis—reflects distinct biology, diagnostic pathways, and therapeutic strategies. Growth stems from genetic testing uptake, specialist referral networks, and guideline evolution that encourages earlier intervention. Evidence generation in specific phenotypes and comorbidity profiles informs sequencing and long-term planning.
AL Amyloidosis
AL Amyloidosis management centers on plasma-cell–directed therapy to reduce light-chain production and mitigate organ damage. Multidisciplinary coordination with cardio-renal teams, toxicity monitoring, and supportive measures is vital to outcomes. Access initiatives and risk-stratified care pathways help align resources with patient complexity.
ATTR Amyloidosis
ATTR Amyloidosis strategies target transthyretin stabilization or synthesis reduction and increasingly leverage genetic screening in at-risk populations. Adoption is shaped by neurologic versus cardiac phenotype, functional status, and care-setting capacity. Longitudinal registries and outcomes-based agreements support sustainable scale-up across regions.
Aβ Amyloidosis
Aβ Amyloidosis entails specialized pathways where research, biomarker development, and referral to expert centers guide management. Care models emphasize comprehensive assessment, symptomatic control, and shared decision-making with patients and families. Collaboration among academic sites, foundations, and industry advances trial readiness and knowledge transfer.
Hereditary Amyloidosis Treatment Market, Segmentation by Treatment Setting
The Treatment Setting perspective—Hospital Outpatient Clinics, Home Healthcare, Specialized Clinics, and Long-Term Care Facilities—highlights service delivery and resource allocation. Site-of-care optimization reduces total cost while safeguarding safety and experience. Integrated data flows, telehealth, and navigation services help coordinate complex regimens over time.
Hospital Outpatient Clinics
Hospital Outpatient Clinics anchor initiation, infusion oversight, and multidisciplinary evaluation with access to advanced diagnostics. Protocolized pathways and case management reduce delays and readmissions while supporting payer requirements. Embedded pharmacists and nurses drive education, AE monitoring, and continuity at discharge.
Home Healthcare
Home Healthcare extends access through nurse visits, training for self-administration, and remote monitoring that flags deterioration early. This model improves convenience and may lessen caregiver burden when supported by clear escalation plans. Vendors invest in connected devices, cold-chain logistics, and documentation to maintain quality standards.
Specialized Clinics
Specialized Clinics concentrate expertise in rare disease, enabling rapid diagnosis, genetic counseling, and coordinated supportive care. Standardized assessments, longitudinal registries, and trial participation strengthen the evidence base. Partnerships with patient organizations and teleconsultation networks expand reach beyond urban hubs.
Long-Term Care Facilities
Long-Term Care Facilities support advanced-stage patients with multidisciplinary palliative services, symptom control, and functional maintenance. Staff training, medication reconciliation, and caregiver engagement reduce complications and transfers. Integrated plans with specialists and home-health teams preserve continuity and dignity of care.
Hereditary Amyloidosis Treatment Market, Segmentation by Geography
In this report, the Hereditary Amyloidosis Treatment Market has been segmented by Geography into five regions: North America, Europe, Asia Pacific, Middle East and Africa and Latin America.
Regions and Countries Analyzed in this Report
North America
North America benefits from established centers of excellence, advanced diagnostics, and active patient organizations that catalyze referral and support. Reimbursement frameworks, specialty pharmacy networks, and outcomes contracts shape access for innovative therapies. Expansion focuses on equitable screening, rural outreach, and integrated data to document long-term value.
Europe
Europe features coordinated rare-disease plans, cross-border registries, and guideline harmonization that streamline care pathways. HTA processes emphasize comparative effectiveness and budget impact, encouraging robust evidence packages. Collaborations among academic consortia, biotech firms, and payers foster sustainable adoption.
Asia Pacific
Asia Pacific demand grows with improving genetic testing capacity, specialist training, and public–private initiatives. Heterogeneous health systems require localized market-access strategies, patient navigation, and telehealth support. Partnerships with reference centers accelerate diagnosis, while phased rollouts align with affordability and infrastructure.
Middle East & Africa
Middle East & Africa progress reflects national rare-disease frameworks, centralized tertiary hubs, and selective funding for high-impact therapies. Priorities include clinician awareness, genetic counseling, and supply-chain reliability for temperature-sensitive products. Regional alliances and outreach programs enhance continuity across diverse care settings.
Latin America
Latin America advances through growing advocacy networks, targeted reimbursement pathways, and reference centers in major metros. Efforts emphasize early detection, adherence support, and real-world data generation to inform policy decisions. Strategic collaborations with public systems and foundations expand access while building clinical capacity.
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.
Hereditary Amyloidosis Treatment Market Competitive Landscape Analysis
Hereditary Amyloidosis Treatment Market is witnessing robust growth driven by strategic partnerships and collaborative strategies among leading pharmaceutical and biotechnology companies. Firms are leveraging advanced technological advancements in targeted therapies, gene-silencing treatments, and personalized medicine to improve patient outcomes. Leading players hold over 45% of market share, reflecting a competitive landscape with a strong future outlook.
Market Structure and Concentration
The market demonstrates a moderately concentrated structure with key manufacturers controlling more than 50% of the share. Smaller players focus on niche innovation to develop novel therapies, including RNA-based and gene-editing solutions. Mergers and strategic partnerships are facilitating market expansion, reflecting a balance between competitive strategies and collaborative initiatives enhancing overall growth.
Brand and Channel Strategies
Leading companies are implementing strong brand strategies to strengthen recognition and credibility. Multi-channel distribution accounts for over 60% of market reach, combining hospital pharmacies, specialty clinics, and direct sales channels. Strategic partnerships with healthcare providers and patient advocacy groups are driving growth, while marketing campaigns emphasize technological leadership and therapeutic efficacy.
Innovation Drivers and Technological Advancements
Continuous innovation is central to the Hereditary Amyloidosis Treatment Market, with advancements improving efficacy, safety, and patient compliance. Over 50% of top players invest in R&D for RNA-silencing drugs, gene therapies, and advanced biologics. Emerging technological advancements are shaping the future outlook and supporting market expansion.
Regional Momentum and Expansion
North America and Europe account for more than 55% of market penetration due to advanced healthcare infrastructure and strategic partnerships. Asia-Pacific is witnessing rapid expansion driven by rising awareness, improved diagnosis, and collaborations with local pharmaceutical companies. Regional mergers and innovation-led strategies are accelerating growth and strengthening the market’s long-term future outlook.
Future Outlook
The Hereditary Amyloidosis Treatment Market is expected to sustain strong growth as companies focus on technological advancements and strategic partnerships. Market concentration may rise above 60%, reflecting intensified competition and ongoing expansion. Continuous innovation and effective strategies will define the future outlook and long-term industry trajectory.
Key players in Hereditary Amyloidosis Treatment Market include,
- Alnylam Pharmaceuticals
- Pfizer
- Ionis Pharmaceuticals
- Prothena
- Akcea Therapeutics
- Eidos Therapeutics
- AstraZeneca
- BridgeBio Pharma
- Novo Nordisk
- Alexion (Neurimmune / Alexion)
- Intellia Therapeutics
- GSK (GlaxoSmithKline)
- SOM Innovation Biotech
- Baxter
- Johnson & Johnson
In this report, the profile of each market player provides following information:
- Company Overview and Product Portfolio
- Market Share Analysis
- 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 Treatment Type
- Market Snapshot, By Route of Administration
- Market Snapshot, By Disease Type
- Market Snapshot, By Treatment Setting
- 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 Treatment Type, 2021 - 2031 (USD Million)
- Medications
- Supportive Treatments
- Hereditary Amyloidosis Treatment Market, By Route of Administration, 2021 - 2031 (USD Million)
- Oral
- Intravenous
- Subcutaneous
- Intramuscular
- Hereditary Amyloidosis Treatment Market, By Disease Type, 2021 - 2031 (USD Million)
- AL Amyloidosis
- ATTR Amyloidosis
- Aβ Amyloidosis
- Hereditary Amyloidosis Treatment Market, By Treatment Setting, 2021 - 2031 (USD Million)
- Hospital Outpatient Clinics
- Home Healthcare
- Specialized Clinics
- Long-Term Care Facilities
- 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 Treatment Type, 2021 - 2031 (USD Million)
- Competitive Landscape
- Company Profiles
- Alnylam Pharmaceuticals
- Pfizer
- Ionis Pharmaceuticals
- Prothena
- Akcea Therapeutics
- Eidos Therapeutics
- AstraZeneca
- BridgeBio Pharma
- Novo Nordisk
- Alexion (Neurimmune / Alexion)
- Intellia Therapeutics
- GSK (GlaxoSmithKline)
- SOM Innovation Biotech
- Baxter
- Johnson & Johnson
- Company Profiles
- Analyst Views
- Future Outlook of the Market