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.
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?
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Regional Sales of Key Therapies to Treat Amyloidosis: 2027
Drivers and Constraints
What Factors Are Driving the Market for Amyloidosis?
What Factors Are Constraining the Market for Amyloidosis?
Drug Class-Specific Trends
Forecast Sales of Key Therapies in Amyloidosis
Etiology and Pathophysiology
Etiology of Major Amyloidosis Subtypes
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
Diagnosed Prevalent Cases of Amyloidosis by Subtype
Diagnosed Prevalence Methods
Sources Used for Diagnosed Incident Cases of AL Amyloidosis
Diagnosed Incident Cases of AL Amyloidosis
Drug-Treated Cases of Amyloidosis
Major Tests and Criteria for Organ Involvement in Amyloidosis
Treatment Providers and Referral Patterns
Expert Insight on Diagnosis of Amyloidosis
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
Mechanism of Action of Key Current Drug Classes Used for Amyloidosis
Current Treatments Used for Amyloidosis
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
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
Advantages and Disadvantages of Doxycycline-TUDCA
Expert Insight on Doxycycline-TUDCA
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
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 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 on Inotersen
Expectations for Launch and Sales Opportunity of Inotersen in ATTR Amyloidosis
Expert Insight on Patisiran Versus Inotersen
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
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 on Select Early-Phase Compounds
Key Discontinuations and Failures in Amyloidosis
Development Discontinuations and Failures
Patient Registries for Amyloidosis
Prominent Amyloidosis Patient Organizations
Orphan Drug Designation
Access and Reimbursement Overview
Region-Specific Reimbursement Practices
Looking for More?
Key Abbreviations Related to Amyloidosis
Brands, Marketers, and Generic Availability of Key Therapies for Amyloidosis, by Market
Akash Saini, Ph.D.
Akash Saini, M.Sc., Ph.D., is the manager of the China In-Depth team at Clarivate. In this role, he leads the efforts to create syndicated landscape and forecast content on various therapy areas for the Chinese market.Previously, he was an assistant manager at BioXcel, where he led consulting projects based on in-silico drug discovery and identification of drug repurposing and in-licensing opportunities. Dr. Saini received his Ph.D. in biochemistry and biotechnology from the International Centre for Genetic Engineering and Biotechnology in New Delhi and conducted postdoctoral research at the University of Massachusetts Medical School. He earned his M.Sc. in biotechnology from Jawaharlal Nehru University in India.
Amy Bradshaw Kaiser, M.S.
Amy Kaiser, M.S., is an associate epidemiologist at DRG, part of Clarivate. Her focus is on infectious diseases and niche and rare diseases. She holds an M.S. in epidemiology from the University of Massachusetts in Amherst and a B.A. in international relations from Mount Holyoke College.