Amyloidosis is caused by the deposition of insoluble amyloid fibrils formed by the accumulation of misfolded proteins into affected organs. Amyloid immunoglobulin light chain (AL), amyloid A (AA), and amyloid transthyretin (ATTR) amyloidosis are the three major subtypes of the disease. The treatment of AL amyloidosis is derived from the chemotherapy regimens used to treat multiple myeloma, while the treatment of AA amyloidosis requires the management of the underlying inflammation that leads to the disease, through off-label use of IL-6/IL-1 inhibitors. ATTR amyloidosis is managed using TTR tetramer stabilizers tafamidis (Pfizer’s Vyndaqel) and diflunisal (generics), the doxycycline/TUDCA (generics) combination, and symptomatic treatment of cardiomyopathy. Tafamidis, approved in Europe for polyneuropathy caused by hereditary ATTR amyloidosis(ATTR-FAP), is the only approved drug for any amyloidosis subtype. Although there is a dearth of approved drugs for amyloidosis, several pharmacotherapies are in late-stage development for the disease. RNA-inhibiting therapies—Akcea Therapeutics and Ionis Pharmaceuticals’ inotersen and Alnylam’s Pharmaceuticals patisiran—are preregistered for hereditary ATTR amyloidosis in the United States; and while patisiran is also preregistered in Europe, inotersen has been approved in the European Union for ATTR-FAP. Tafamidis, approved in Europe for ATTR-FAP, is in Phase III development for ATTR-associated cardiomyopathy. Moreover, Takeda Oncology’s oral proteasome inhibitor, ixazomib (Ninlaro), and Janssen Biotech and Genmab’s daratumumab (Darzalex), an anti-CD38 monoclonal antibody, are in late-phase development for AL amyloidosis. Despite available treatment options and various therapies in late-phase development, there will remain a high unmet need for additional and effective therapies for amyloidosis through 2027.

Questions Answered

  • How will the sizes of the AL, AA and ATTR amyloidosis populations across the United States and the EU5 change through 2027?
  • What are key current therapies in the amyloidosis market?
  • What are the key drug targets emerging from basic and clinical research in amyloidosis? Which emerging therapies do amyloidosis experts consider most promising? How would new therapies influence the management of amyloidosis patients?
  • How are emerging amyloidosis therapies being evaluated by the amyloidosis experts across the United States and the EU5, and which are likely to launch by 2027? What commercial impact will they have on the amyloidosis market?

Product Description

Niche & Rare Disease Landscape & Forecast: Comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

Table of contents

  • Amyloidosis - Landscape & Forecast - Disease Landscape & Forecast
    • Commercial Outlook
      • Key Findings
        • Regional Sales of Key Therapies to Treat Amyloidosis: 2027
        • SWOT Analysis
      • Drivers and Constraints
        • What Factors Are Driving the Market for Amyloidosis?
        • What Factors Are Constraining the Market for Amyloidosis?
      • Drug Class-Specific Trends
        • RNA-Inhibiting Therapies
        • Transthyretin Stabilizers
        • Proteasome Inhibitors
        • CD38 Inhibitors
    • Forecast
      • Forecast Sales of Key Therapies in Amyloidosis
    • Etiology and Pathophysiology
      • Disease Overview
        • Etiology
          • Etiology of Major Amyloidosis Subtypes
        • Pathophysiology
          • Symptoms
            • Symptoms and Signs of Major Subtypes of Amyloidosis
          • Key Pathways and Drug Targets
            • Key Pathways and Drug Targets for AL Amyloidosis
            • Key Pathways and Drug Targets for AA Amyloidosis
            • Key Pathways and Drug Targets for ATTR Amyloidosis
        • Epidemiology Overview
          • Introduction
            • Key Findings
          • Epidemiology Populations
            • Disease Definition
            • Methods
            • Diagnosed Prevalent Cases of Amyloidosis by Subtype
            • Disease Definition
            • Diagnosed Prevalence Methods
            • Sources Used for Diagnosed Incident Cases of AL Amyloidosis
            • Diagnosed Incident Cases of AL Amyloidosis
            • Drug-Treated Cases of Amyloidosis
        • Current Treatment
          • Key Findings
            • Diagnosis
              • Major Tests and Criteria for Organ Involvement in Amyloidosis
              • Diagnostic Algorithm
              • Treatment Providers and Referral Patterns
              • Expert Insight on Diagnosis of Amyloidosis
            • Treatment Goals
              • Key End Points Used in Clinical Trials for Amyloid Light-Chain Amyloidosis
              • Key End Points Used in Clinical Trials for Amyloid A Amyloidosis
              • Key End Points Used in Clinical Trials for Amyloid Transthyretin Amyloidosis
              • Key Physician Insights on Clinical End Points
              • Expert Insight on Clinical End Points
            • Key Current Therapies
              • Overview
              • Mechanism of Action of Key Current Drug Classes Used for Amyloidosis
              • Current Treatments Used for Amyloidosis
              • Bortezomib
              • Clinical Trial Outcomes for Bortezomib
              • Advantages and Disadvantages of Bortezomib
              • Expert Insight on Bortezomib
              • Clinical Trial Outcomes for Thalidomide
              • Advantages and Disadvantages of Thalidomide
              • Clinical Trial Outcomes for Lenalidomide
              • Advantages and Disadvantages of Lenalidomide
              • Clinical Trial Outcomes for Pomalidomide
              • Advantages and Disadvantages of Pomalidomide
              • Clinical Trial Outcomes for Autologous Stem-Cell Transplantation
              • Expert Insight on Autologous Stem-Cell Transplantation
              • Tocilizumab
              • Advantages and Disadvantages of Tocilizumab
              • Clinical Trial Outcomes for Tafamidis
              • Select Ongoing Trials of Tafamidis
              • Advantages and Disadvantages of Tafamidis
              • Expert Insight on Tafamidis
              • Advantages and Disadvantages of Diflunisal
              • Expert Insight on Diflunisal
              • Advantages and Disadvantages of Liver Transplantation
              • Expert Insight on Liver Transplantation
              • Doxycycline-TUDCA
              • Advantages and Disadvantages of Doxycycline-TUDCA
              • Expert Insight on Doxycycline-TUDCA
            • Medical Practice
              • Overview
              • Symptomatic Treatment of Amyloidosis
              • Treatment Guidelines, by Market
              • Generalized Treatment Decision Tree for Amyloidosis
          • Unmet Need Overview
            • Top Unmet Needs in Amyloidosis: Current and Future Attainment
          • Emerging Therapies
            • Key Findings
              • Pipeline Trends for Amyloidosis
            • Key Emerging Therapies
              • Notable Developments Among Key Emerging Therapies for Amyloidosis
              • Key Therapies in Development for Amyloidosis
              • Estimated Launch Dates of Key Emerging Therapies for the Treatment of Amyloidosis
              • Expert Insight on RNA-Inhibiting Therapies
              • Key Results from Select Clinical Trials Investigating Patisiran for the Treatment of ATTR Amyloidosis
              • Key Ongoing Clinical Trials of Patisiran for the Treatment of ATTR Amyloidosis
              • Expert Insight
              • Expert Insight on Patisiran
              • Expectations for Launch and Sales Opportunity of Patisiran in ATTR Amyloidosis
              • Key Results from Select Clinical Trials Investigating Inotersen for the Treatment of ATTR Amyloidosis
              • Expert Insight
              • Expert Insight on Inotersen
              • Expectations for Launch and Sales Opportunity of Inotersen in ATTR Amyloidosis
              • Expert Insight on Patisiran Versus Inotersen
              • Ixazomib Profile
              • Key Ongoing Clinical Trials of Ixazomib for the Treatment of Refractory or Relapsed AL Amyloidosis
              • Expert Insight on Ixazomib
              • Expectations for Launch and Sales Opportunity of Ixazomib in Amyloidosis
              • Daratumumab Profile
              • Key Ongoing Clinical Trials of Daratumumab for the Treatment of AL Amyloidosis
              • Expert Insight on Daratumumab
              • Expectations for Launch and Sales Opportunity of Daratumumab in AL Amyloidosis
            • Early-Phase Pipeline Analysis
              • Notable Developments in the Early-Phase Pipeline for Amyloidosis
              • Select Early-Phase Compounds in Development for Amyloidosis
              • Expert Insight
              • Expert Insight on Select Early-Phase Compounds
            • Key Discontinuations and Failures in Amyloidosis
              • Development Discontinuations and Failures
            • Patient Registries
              • Patient Registries for Amyloidosis
              • Prominent Amyloidosis Patient Organizations
            • Orphan Drug Designation
            • Access and Reimbursement Overview
              • Region-Specific Reimbursement Practices
                • Looking for More?
            • Methodology
              • Drug-Specific Assumptions
              • Patient Populations
              • Bottom-Up Forecast Assumptions
                • General Sources of Data
                • Drug-Treatment Rate Assumptions in Amyloidosis
                • Patient-Share Assumptions in Amyloidosis
                • General Statements About Pricing
                • Sources for Drug Prices
                • 2027 Exchange Rates
                • Price per Treated Day: 2027
                • Dosing, Days of Therapy, and Compliance
                • Out-Year Forecasting
                • Emerging Therapy Prices
            • Appendix
              • Key Abbreviations Related to Amyloidosis
              • Brands, Marketers, and Generic Availability of Key Therapies for Amyloidosis, by Market
              • Experts Interviewed
              • Bibliography

          Author(s): Akash Saini, Ph.D.; Amy Bradshaw Kaiser, MS

          Akash is a Principal Analyst in the China In-Depth team at Decision Resources Group. Since 2016, he has specialized in a range of indications including rare disease indications such as DMD, ALS, ITP, IPF, JIA, and retinitis pigmentosa. He has authored Disease Landscape & Forecast reports, Access & Reimbursement reports, and Treatment Algorithm reports based on primary market research and real-world evidence. Prior to joining DRG, Akash was a post-doctoral fellow at the University of Massachusetts Medical School. He has a Ph.D. from the International Centre for Genetic Engineering and Biotechnology, New Delhi.

          Amy Kaiser joined Decision Resources Group as an associate epidemiologist in 2017. Her focus is on the epidemiology of infectious diseases and niche and rare diseases.

          She holds an MS in Epidemiology from the University of Massachusetts, Amherst and a BA in International Relations from Mount Holyoke College. Prior to joining Decision Resources Group, she worked as a human health research associate at a environmental consulting firm where her epidemiology research focused on occupational and environmental exposures and associated outcomes.