Amyloidosis - Market Insight, Epidemiology, and Market Forecast - 2036

Amyloidosis Insights and Trends

• Amyloidosis is a rare, progressive, and multisystem disorder characterized by extracellular deposition of misfolded amyloid fibrils in tissues and organs, leading to progressive organ dysfunction. The most common systemic forms include immunoglobulin light chain (AL) amyloidosis and transthyretin (ATTR) amyloidosis (hereditary and wild-type).
• Disease presentation is highly heterogeneous and organ-dependent, most frequently involving the heart, kidneys, peripheral nerves, and liver. Cardiac involvement (amyloid cardiomyopathy) is a major driver of morbidity and mortality, particularly in ATTR and advanced AL disease.
• Clinical manifestations are often non-specific and multi-systemic, including heart failure with preserved ejection fraction (HFpEF), nephrotic syndrome, peripheral neuropathy, autonomic dysfunction, fatigue, and unexplained weight loss, contributing to frequent diagnostic delays.
• Amyloidosis is driven by underlying protein misfolding disorders, where unstable precursor proteins (light chains in AL or transthyretin in ATTR) aggregate into insoluble fibrils, resulting in progressive extracellular deposition and tissue damage.
• Diagnosis remains challenging and typically requires a combination of biomarkers, imaging (echocardiography, cardiac MRI, nuclear scintigraphy for ATTR), tissue biopsy with Congo red staining, and amyloid typing, often leading to underdiagnosis or late-stage detection.
• The global disease burden is increasingly recognized, particularly for ATTR amyloidosis in aging populations and AL amyloidosis due to improved awareness and diagnostic capabilities, though true prevalence remains underestimated due to historical under-recognition.
• Management is subtype-specific and multidisciplinary, focusing on reducing precursor protein production, stabilizing misfolded proteins, and managing end-organ dysfunction through coordinated cardiology, hematology, neurology, and nephrology care.
• Therapeutic advances have significantly improved outcomes, including disease-modifying agents such as tafamidis (VYNDAMAX), acoramidis (ATTRUBY/BEYONTTRA), patisiran (ONPATTRO), inotersen (TEGSEDI), and vutrisiran (AMVUTTRA) for ATTR amyloidosis, and daratumumab (DARZALEX FASPRO) and bortezomib (VELCADE) based regimens for AL amyloidosis, alongside supportive heart failure and organ-directed therapies.
• Despite progress, amyloidosis continues to represent a major unmet clinical need, driven by delayed diagnosis, limited disease awareness, irreversible organ damage at presentation, and the need for earlier intervention and broader access to advanced targeted therapies.

DelveInsight's 'Amyloidosis- Market Insights, Epidemiology and Market Forecast - 2036' report delivers an in-depth understanding of amyloidosis, historical and forecasted epidemiology, as well as the amyloidosis market trends in the United States, EU4 (Germany, Spain, Italy, and France), and the United Kingdom, and Japan.

The amyloidosis market report delivers a comprehensive analysis of the current treatment landscape, including standards of care, clinical practices, and evolving therapeutic algorithms. It evaluates amyloidosis patient burden trends, revenue & market share dynamics, peak patient share & therapy uptake analysis, and provides an in-depth market size assessment, and growth rate projections (Historical & Forecast 2022-2036) across global regions. The report highlights key unmet medical needs in amyloidosis and maps the competitive and clinical landscape to uncover high-value opportunities, providing a clear outlook on future market growth potential.

Key Factors Driving the Amyloidosis Market

Rising Amyloidosis Disease Recognition and Diagnosis

The overall prevalence and diagnosis rates of Amyloidosis are increasing due to improved disease awareness, wider availability of advanced imaging and biomarker-based diagnostics, growing use of genetic testing, and better differentiation between AL and ATTR subtypes, supporting continued market expansion.

Growing Opportunities in Targeted and Disease-Modifying Therapies

The amyloidosis market is being driven by rapid advances in targeted therapies, including RNA-silencing agents, monoclonal antibodies, plasma cell-directed therapies, and emerging gene-editing approaches aimed at reducing amyloid production, improving organ response, and potentially altering long-term disease progression.

Increasing Focus on Early Diagnosis and Multidisciplinary Disease Management

Increasing clinical focus on earlier diagnosis and multidisciplinary disease management, particularly in cardiac and neurologic amyloidosis, is expanding treatment eligibility and accelerating therapeutic adoption across both ATTR and AL amyloidosis populations.

Emerging Competitive Landscape in Amyloidosis

The competitive landscape of amyloidosis is evolving with continued development of next-generation transthyretin stabilizers, siRNA therapies, monoclonal antibodies targeting amyloid deposits, and novel plasma cell-targeted agents. Emerging pipeline assets such as nexiguran ziclumeran (nex-z), nucresiran, anselamimab, and other investigational therapies are expected to further strengthen market competition over the forecast period.

Amyloidosis Understanding and Treatment Algorithm

Amyloidosis Overview

Amyloidosis is a group of rare disorders caused by the accumulation of misfolded proteins (amyloid fibrils) in tissues and organs, leading to progressive organ dysfunction. The disease commonly affects the heart, peripheral nerves, and kidneys, with clinical manifestations varying by amyloid protein type. Major systemic forms include Hereditary Transthyretin Amyloidosis (hATTR), caused by mutations in the transthyretin (TTR) gene, and AL Amyloidosis, which results from excess monoclonal light-chain production by abnormal plasma cells.

Amyloidosis Diagnosis

Diagnosis of Amyloidosis is often delayed and challenging because of its heterogeneous clinical presentation and overlap with other cardiac, neurologic, and renal disorders. Initial evaluation includes testing for monoclonal proteins using serum and urine immunofixation and serum free light-chain assays to assess for AL Amyloidosis. Diagnosis is confirmed through tissue biopsy with Congo red staining, demonstrating apple-green birefringence under polarized light. Subtype identification may involve mass spectrometry-based amyloid typing, genetic testing for Hereditary Transthyretin Amyloidosis, and advanced imaging techniques such as echocardiography, cardiac MRI, and 99mTc-PYP scintigraphy for cardiac ATTR amyloidosis. Early diagnosis is critical to prevent irreversible organ damage.

Amyloidosis Treatment

Treatment of Amyloidosis focuses on reducing the production of amyloid-forming proteins, slowing disease progression, and managing organ-related complications. In Hereditary Transthyretin Amyloidosis, approved therapies include TTR stabilizers such as VYNDAMAX and gene-silencing agents including ONPATTRO, TEGSEDI, AMVUTTRA, and WAINUA, which reduce abnormal transthyretin production and help delay neurological and cardiac decline.

In AL Amyloidosis, treatment primarily targets the underlying plasma cell clone using anti-plasma cell regimens similar to those used in multiple myeloma. Current standard therapy includes a DARZALEX FASPRO-based regimen, while eligible patients may undergo autologous stem cell transplantation. Supportive care remains essential for managing cardiac, renal, and other organ complications, and emerging therapies such as monoclonal antibodies and CAR T-cell approaches are under clinical investigation.

Amyloidosis Unmet Needs

The section "unmet needs of amyloidosis" outlines the critical gaps between the current state of patient care, diagnosis, and the ideal & effective management of the disease. It highlights the obstacles experienced by patients, clinicians, and researchers and identifies potential solutions for future progress.

1. No curative therapies are currently available; existing treatments mainly slow disease progression rather than reverse established organ damage.

2. Delayed and challenging diagnosis due to nonspecific symptoms and clinical heterogeneity often results in advanced disease at presentation.

3. Irreversible cardiac and neurologic damage at diagnosis limits long-term treatment benefit and worsens patient outcomes.

4. There remains a need for therapies that effectively address both cardiomyopathy and neuropathy across amyloidosis subtypes, and others.....

Amyloidosis Epidemiology

Key Findings from Amyloidosis Epidemiological Analysis and Forecast

• Amyloidosis shows an increasing diagnosed prevalence globally due to improved disease awareness, advancements in diagnostic techniques, and growing recognition of systemic amyloidosis subtypes.
• AL Amyloidosis is the most common form of systemic amyloidosis and accounts for the majority of newly diagnosed cases. In the United States, approximately 1,275-3,200 new cases are reported annually, with an estimated incidence of 16.7 patients per million population in 2021.
• AL Amyloidosis primarily affects older adults, with a median age at diagnosis of 63-65 years, and incidence increases substantially with advancing age. The disease also demonstrates a slight male predominance.
• Hereditary Transthyretin Amyloidosis affects both males and females, although late-onset familial disease is more commonly observed in men. Increasing recognition of non-endemic and late-onset cases has contributed to growth in the diagnosed patient population.
• Regional variation in disease burden remains significant, with higher diagnosed prevalence reported in several developed markets due to wider access to genetic testing, cardiac imaging, and specialized amyloidosis centers.

Amyloidosis Drug Analysis & Competitive Landscape

The Amyloidosis drug chapter provides a detailed, market-focused review of the approved therapies and emerging pipeline across Phase I-III clinical trials. It covers the mechanism of action, clinical trial data, regulatory approvals, patents, collaborations, and strategic partnerships for each therapy, along with their advantages, limitations, and recent developments. This section offers critical insights into the Amyloidosis treatment landscape, supporting market assessment, competitive analysis, and growth forecasting for the Amyloidosis therapeutics market.

Approved Therapies for Amyloidosis

Vutrisiran (AMVUTTRA): Alnylam Pharmaceuticals

AMVUTTRA is a subcutaneously administered transthyretin-directed siRNA (RNAi) therapy developed by Alnylam Pharmaceuticals for the treatment of ATTR amyloidosis, including Hereditary Transthyretin Amyloidosis and wild-type ATTR amyloidosis. The therapy received FDA approval in June 2022 for adults with hATTR amyloidosis with polyneuropathy and subsequently received a positive EU opinion for adults with stage 1 or stage 2 polyneuropathy.

Daratumumab and hyaluronidase-fihj (DARZALEX FASPRO): Johnson & Johnson

DARZALEX FASPRO is the only CD38-directed antibody approved to be given subcutaneously to treat patients with multiple myeloma and now AL amyloidosis. DARZALEX FASPRO is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), which is part of Halozyme's ENHANZE drug delivery technology.

Amyloidosis Pipeline Analysis

Nexiguran ziclumeran (nex-z, NTLA-2001): Intellia Therapeutics and Regeneron

NTLA-2001 is an investigational in vivo CRISPR-based gene-editing therapy being developed by Intellia Therapeutics in collaboration with Regeneron Pharmaceuticals as a one-time treatment for ATTR amyloidosis. The therapy is designed to inactivate the TTR gene and is currently in Phase III development. NTLA-2001 has received Orphan Drug Designation (ODD), Regenerative Medicine Advanced Therapy (RMAT) designation from the US FDA, and ODD from the European Commission.

NXC-201: Immix Biopharma

NXC-201 is a sterically optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. NXC-201 is being studied for the treatment of patients with r/r AL amyloidosis, with the potential to expand into autoimmune indications. NXC-201 has been awarded ODD and regenerative medicine advanced therapy designation in the US by the FDA and ODD in Europe by the European Medicines Agency in r/r AL Amyloidosis.

Amyloidosis Key Players, Market Leaders, and Emerging Companies

• Alnylam Pharmaceuticals
• Johnson & Johnson
• Intellia Therapeutics and Regeneron
• Immix Biopharma
• AbbVie, and others

Amyloidosis Drug Updates

• In March 2026, Intellia Therapeutics announced that the US FDA had lifted the clinical hold on the Phase III MAGNITUDE trial of nexiguran ziclumeran (nex-z; NTLA-2001) for ATTR Cardiomyopathy (ATTR-CM), reinforcing regulatory momentum and enabling continued late-stage development of the investigational CRISPR-based gene-editing therapy.
• In January 2026, Immix Biopharma announced that the US FDA granted Breakthrough Therapy Designation to NXC-201 for the treatment of relapsed/refractory AL Amyloidosis, supporting expedited development of the investigational therapy based on encouraging interim Phase II results from the NEXICART-2 trial presented at the American Society of Hematology (ASH) 2025 annual meeting.
• In April 2026, Alnylam Pharmaceuticals announced plans to increase patient enrollment in the nucresiran clinical trial due to rapid recruitment momentum, while maintaining expectations to potentially launch the therapy for ATTR Cardiomyopathy (ATTR-CM) by 2030, subject to positive clinical data and regulatory approval.

Drug Class Insights

Amyloidosis Market Outlook

The amyloidosis treatment landscape has evolved markedly in recent years, shifting from largely supportive care to targeted, mechanism-based therapies across both ATTR and AL subtypes. Current strategies focus on reducing amyloid protein production, slowing disease progression, preserving organ function, and improving survival outcomes.

In ATTR amyloidosis, disease-modifying therapies such as tafamidis and RNA-silencing agents, including patisiran, inotersen, eplontersen, and vutrisiran, have transformed management by stabilizing or reducing transthyretin levels. The field is now moving toward next-generation RNA-targeting approaches and gene-editing therapies, with investigational agents such as Nexiguran Ziclumeran (nex-z) aiming for deeper and potentially durable TTR suppression.

In AL amyloidosis, treatment has progressed from conventional chemotherapy to more targeted plasma cell-directed regimens. DARZALEX FASPRO-based combinations have become a key backbone in first-line therapy, while emerging modalities such as BCMA-targeted CAR T therapies, bispecific and trispecific antibodies, and fibril-directed monoclonal antibodies are expanding treatment options and reinforcing the shift toward precision medicine.

Despite these advances, major unmet needs persist due to late diagnosis, irreversible organ damage, and the absence of curative therapies that can fully eliminate amyloid deposits. As a result, increasing focus is being placed on approaches that combine suppression of amyloid production with active amyloid clearance and organ recovery.

Overall, improving disease awareness, earlier diagnosis, expanding access to targeted therapies, and a strengthening late-stage pipeline are expected to support steady growth of the amyloidosis market across the 7MM, with the US remaining the dominant market, followed by Europe and Japan.

Drug Class/Insights into Leading Emerging and Marketed Therapies in Amyloidosis (2022-2036 Forecast)

The Amyloidosis market comprises targeted small molecules, biologics, RNA-based therapies, and emerging cell and gene therapies aimed at reducing amyloid production, stabilizing precursor proteins, suppressing abnormal plasma cells, or enhancing amyloid clearance across ATTR and AL amyloidosis.

• Small molecules: In ATTR amyloidosis, tafamidis (VYNDAMAX) acts as a TTR stabilizer, preventing transthyretin misfolding and amyloid fibril formation, particularly in ATTR cardiomyopathy, supporting its role as a key oral disease-modifying therapy.
• RNA-based therapies (gene silencing): Agents including patisiran, inotersen, eplontersen, and vutrisiran reduce hepatic TTR production through siRNA and antisense oligonucleotide mechanisms, forming the backbone of current treatment across hATTR polyneuropathy and cardiomyopathy.
• Monoclonal antibodies: In AL Amyloidosis, CD38-targeting monoclonal antibodies such as DARZALEX FASPRO represent the current standard of care, while emerging fibril-directed antibodies aim to promote immune-mediated amyloid clearance.
• Cellular immunotherapies and bispecific antibodies: Emerging AL amyloidosis therapies include BCMA-targeted CAR T-cell therapies, bispecific T-cell engagers, and trispecific antibodies, designed to improve elimination of abnormal plasma cells and deepen hematologic responses.
• Emerging gene-editing therapies: Next-generation approaches such as CRISPR-Cas9-based therapies, including nexiguran ziclumeran (nex-z), and novel siRNA candidates such as nucresiran are being developed to achieve deeper and potentially long-lasting suppression of transthyretin production.

Overall, the amyloidosis treatment landscape is evolving from supportive and protein-stabilizing approaches toward durable gene-silencing, immunotherapy, and gene-editing strategies, with targeted biologics and RNA-based therapies currently driving market growth while next-generation pipeline agents shape future therapeutic expansion.

Amyloidosis Drug Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during the forecast period (2026-2036). The analysis covers the Amyloidosis drug's uptake, performance at peak, factors affecting performance during prime years of growth, patient uptake by therapy, and anticipated sales generated by each drug.

Drug uptake in Amyloidosis is increasing steadily due to improving diagnosis rates, growing disease awareness, and expanding use of targeted therapies across ATTR and AL amyloidosis. In ATTR amyloidosis, RNA-based therapies such as vutrisiran, patisiran, inotersen, and eplontersen are witnessing strong uptake because of their robust efficacy in reducing transthyretin production and slowing disease progression, while tafamidis continues to maintain significant use in ATTR cardiomyopathy.

In AL Amyloidosis, uptake of DARZALEX FASPRO-based regimens has increased substantially following its approval as the first FDA-approved therapy specifically indicated for AL amyloidosis. Emerging therapies, including BCMA-targeted therapies, bispecific antibodies, and gene-editing approaches, are expected to further support future market growth.

Amyloidosis Therapies Price Scenario & Trends

Pricing and analogue assessment of amyloidosis therapies highlights evolving price dynamics structures. This section summarizes the cost of approved treatments, the closest and most appropriate analogue selection for emerging therapies, and the understanding of how pricing influences market access, adherence, and long-term uptake.

Further details are provided in the final report....

Industry Experts and Physician Views for Amyloidosis

To keep up with Amyloidosis market trends, we take Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on the emerging amyloidosis therapies, evolving treatment landscape, patient adherence to conventional therapies, therapy switching trends, drug adoption and uptake, accessibility challenges, and epidemiology and real-world prescription patterns in amyloidosis, including MD, PhD, Instructor, Postdoctoral Researcher, Professor, Researcher, and others.

DelveInsight's analysts connected with 10+ KOLs to gather insights at the country level. Centers such as the Columbia University Irving Medical Center, Universite Paris-Saclay, Bicetre Hospital, Mayo Clinic, National Amyloidosis Centre, etc., were contacted. Their opinion helps understand and validate current and emerging Amyloidosis therapies, highlight unmet medical needs, provide epidemiological context, and support strategic decisions for market access, therapy adoption, and pipeline prioritization in amyloidosis.

Qualitative Analysis: SWOT and Conjoint Analysis

We perform qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and conjoint analysis.

In the SWOT analysis of amyloidosis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Conjoint analysis analyzes emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

The team of analysts analyzes promising emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. In efficacy, the trial's primary and secondary outcome measures are evaluated, whereas the therapies' safety is evaluated, wherein the acceptability, tolerability, and adverse events are mainly observed. In addition, the scoring is also based on the route of administration, order of entry, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Scope of the Report:

• The report covers a segment of key events, an executive summary, a descriptive overview of amyloidosis, explaining its causes, signs and symptoms, pathogenesis, and currently available treatments.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of the diagnosis rate, and disease progression along treatment guidelines.
• Additionally, an all-inclusive account of both the current and emerging treatments, along with the elaborate profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
• A detailed review of the amyloidosis market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM amyloidosis market.

Report Insights

• Amyloidosis Patient Population Forecast
• Amyloidosis Therapeutics Market Size
• Amyloidosis Pipeline Analysis
• Amyloidosis Market Size and Trends
• Amyloidosis Market Opportunity (Current and Forecasted)

Report Key Strengths

• Epidemiology-based (Epi-based) Bottom-up Forecasting
• Artificial Intelligence (AI)-Enabled Market Research Report
• 11-Year Forecast
• Amyloidosis Market Outlook (North America, Europe, Asia-Pacific)
• Patient Burden Trends (By Geography)
• Amyloidosis Treatment Addressable Market (TAM)
• Amyloidosis Competitive Landscape
• Amyloidosis Major Companies Insights
• Amyloidosis Price Trends and Analogue Assessment
• Amyloidosis Therapies Drug Adoption/Uptake
• Amyloidosis Therapies Peak Patient Share Analysis

Report Assessment

• Amyloidosis Current Treatment Practices
• Amyloidosis Unmet Needs
• Amyloidosis Clinical Development Analysis
• Amyloidosis Emerging Drugs Product Profiles
• Amyloidosis Market Attractiveness
• Amyloidosis Qualitative Analysis (SWOT and conjoint analysis)

FAQs:

Market Insights

• What was the amyloidosis market size, the market size by therapies, the market share (%) distribution in 2025, and what would it look like by 2036? What are the contributing factors for this growth?
• What are the anticipated pricing variations among different geographies for the emerging therapies in the future?
• What can be the future treatment paradigm of amyloidosis?
• What are the disease risks, burdens, and unmet needs of amyloidosis? What will be the growth opportunities across the 7MM concerning the patient population with amyloidosis?
• Who is the major future competitor in the market, and how will the competitors affect their market share?
• What are the current options for the treatment of amyloidosis? What are the current guidelines for treating amyloidosis in the US, Europe, and Japan?

Reasons to Buy:

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the amyloidosis market.
• Bottom up forecasting builds from the affected population to product forecasts, delivering a robust, data driven approach ideal for new therapies and novel classes.
• Insights on patient burden/disease incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• To understand KOLs' perspectives on the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights into the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.
• This Artificial Intelligence (AI) enabled report summarizes and simplifies complex datasets within the report into clear, actionable insights for stakeholders, investors, and healthcare providers, enabling faster, data driven decisions.