Amyloidosis | Landscape & Forecast | Disease Landscape & Forecast

Publish date: August 2018

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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

  • Disease Landscape & Forecast
    • Commercial Outlook
      • Key Findings
      • 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
    • Etiology and Pathophysiology
      • Disease Overview
      • Etiology
      • Pathophysiology
      • Symptoms
      • Key Pathways and Drug Targets
    • Epidemiology Overview
      • Introduction
        • Key Findings
      • Epidemiology Populations
        • Diagnosed Prevalent Cases of Amyloidosis by Subtype
        • Diagnosed Incident Cases of AL Amyloidosis
        • Drug-Treatable Cases of Amyloidosis
    • Current Treatment
      • Key Findings
      • Diagnosis
        • Treatment Providers and Referral Patterns
      • Treatment Goals
        • Key Physician Insights on Clinical End Points
      • Key Current Therapies
        • Overview
        • Proteasome Inhibitors
        • Immunomodulatory Agents
        • Autologous Stem-Cell Transplantation
        • Interleukin-6 Inhibitors
        • Transthyretin Stabilizers
        • Liver Transplantation
        • Amyloid Degraders
      • Medical Practice
        • Overview
        • Supportive Management
        • Treatment Guidelines
        • Region-Specific Treatment
    • Unmet Need Overview
    • Emerging Therapies
      • Key Findings
      • Key Emerging Therapies
        • Notable Developments Among Key Emerging Therapies for Amyloidosis
        • RNA-Inhibiting Therapies
        • Proteasome Inhibitors
        • CD38 Inhibitors
      • Early-Phase Pipeline Analysis
        • Notable Developments in the Early-Phase Pipeline for Amyloidosis
        • Expert Insight
      • Key Discontinuations and Failures in Amyloidosis
      • Patient Registries
        • Patient Organizations
      • Orphan Drug Designation
        • Orphan Drug Provisions: United States
        • Orphan Drug Provisions: Europe
    • Access and Reimbursement Overview
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
        • Looking for More?
    • Methodology
      • Drug-Specific Assumptions
      • Patient Populations
      • Bottom-Up Forecast Assumptions
        • General Sources of Data
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Out-Year Forecasting
        • Emerging Therapy Prices
    • Appendix
      • Experts Interviewed
      • Bibliography

Author(s): Akash Saini, PhD; Amy Bradshaw Kaiser, MS

Akash Saini, is a lead analyst with the Infectious, Niche, and Rare Disease team at Decision Resources Group. Prior to joining Decision Resources Group, Saini was a postdoctoral fellow at the University of Massachusetts Medical School, where he studied mitochondrial dysfunction in amyotrophic lateral sclerosis (ALS). He earned a in Biochemistry and Biotechnology from the International Centre for Genetic Engineering and Biotechnology, New Delhi, where he also studied the underlying disease mechanism in ALS, and an in Biotechnology from Jawaharlal Nehru University, New Delhi, where he studied the biophysical characteristics of amyloid formation.

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.