Friedreich’s ataxia (FA) is a progressive, autosomal recessive, neurodegenerative disease characterized by slowly advancing ataxia (loss of bodily control). No treatments are currently approved for FA; thus, patients rely on a combination of off-label therapies targeted to specific symptoms, over-the-counter antioxidants, and nonpharmacological interventions to manage their disease. Despite the high unmet need for effective treatments, the late-stage FA pipeline is sparse; the most advanced agents are only in Phase II. However, optimism remains high among thoughtleaders as technological breakthroughs in molecular biology and gene-editing techniques are spurring early-stage development in FA. Given the lack of current therapies for FA, significant commercial opportunity remains for any agent that can alleviate the disease’s symptoms or modify its course.

QUESTIONS ANSWERED

  • How large is the diagnosed prevalent FA population in the United States and EU5? How will the population change through 2027?
  • What is the current treatment landscape for FA patients, and how will it change in the next ten years?
  • What clinical needs remain unfulfilled?
  • Which emerging therapies do neurologists consider most promising, and which are likely to launch by 2027? What will be the commercial impact of these new FA therapies?

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

  • Friedreich's Ataxia - Landscape & Forecast - Disease Landscape & Forecast
    • Commercial Outlook
      • Key Findings
        • Regional Sales of Key Therapies to Treat Friedreich's Ataxia: 2017 and 2027
        • Friedreich's Ataxia SWOT Analysis
      • Drivers and Constraints
        • What Factors Are Driving Sales in Friedreich's Ataxia?
        • What Factors Are Constraining Sales in Friedreich's Ataxia?
      • Nrf2 Activator-Specific Trends
        • Nrf2 Activators
    • Forecast
      • Sales of Key Therapies in Friedreich's Ataxia
    • Etiology and Pathophysiology
      • Disease Overview
        • Etiology
          • Etiology and Pathophysiology of Friedreich's Ataxia
        • Disease Pathophysiology
          • Clinical Presentation and Symptomology
            • Clinical Presentation and Key Symptoms of Friedreich's Ataxia
            • Frequency of Symptoms Associated with Friedreich's Ataxia
          • Key Pathways and Drug Targets
          • Epidemiology Overview
            • Key Findings
              • Epidemiology Populations
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalence of Friedreich's Ataxia
                • Number of Diagnosed Prevalent Cases of Friedreich's Ataxia: 2017-2027
                • Drug-Treatable Diagnosed Prevalent Cases of Friedreich's Ataxia: 2017-2027
                • Disease Definition
                • Methods
                • Sources Used for Friedreich's Ataxia with Comorbid Cardiomyopathy
                • Number of Diagnosed Prevalent Cases of Friedreich's Ataxia with Comorbid Cardiomyopathy: 2017-2027
                • Disease Definition
                • Methods
                • Sources Used for Friedreich's Ataxia with Comorbid Diabetes Mellitus
                • Number of Diagnosed Prevalent Cases of Friedreich's Ataxia with Comorbid Diabetes: 2017-2027
            • Current Treatment Overview
              • Key Findings
                • Diagnosis
                  • Treatment Providers and Referral Patterns
                  • Expert Insight: Diagnosis of Friedreich's Ataxia
                • Treatment Goals
                  • Key End Points Used in Clinical Trials of Therapies to Treat Friedreich's Ataxia
                • Key Current Therapies
                  • Overview
                  • Recommended Pharmacological and Nonpharmacological Interventions for Noncardiac Symptoms Associated with Friedreich's Ataxia
                  • Recommended Pharmacological Interventions for Cardiac Symptoms Associated with Friedreich's Ataxia
                • Medical Practice
                  • Overview
                  • Treatment Guidelines
                  • Recommended Assessments and Considerations for Patients with Friedreich's Ataxia
              • Unmet Need Overview
                • Current and Future Attainment of Unmet Needs in Friedreich's Ataxia
                • Top Unmet Needs in Friedreich's Ataxia—Current and Future Attainment
                • Expert Insight: Unmet Need in Friedreich's Ataxia
              • Emerging Therapies
                • Key Findings
                  • Pipeline Trends in Friedreich's Ataxia
                • Key Emerging Therapies
                  • Notable Developments Among Key Emerging Therapies for Friedreich's Ataxia
                  • Key Therapies in Development for Friedreich's Ataxia
                  • Estimated Launch Dates of Key Emerging Therapies for the Treatment of Friedreich's Ataxia
                  • Omaveloxolone Profile
                  • Analysis of the Clinical Development Program for Omaveloxolone
                  • Expert Insight: Omaveloxolone
                  • Expectations for Launch and Sales Opportunity of Omaveloxolone in Friedreich's Ataxia
                  • TAK-831 Profile
                  • Analysis of the Clinical Development Program for TAK-831
                  • Expert Insight: TAK-831
                  • Expectations for Launch and Sales Opportunity of TAK-831 in Friedreich's Ataxia
                • Early-Phase Pipeline Analysis
                  • Notable Developments in the Early-Phase Pipeline for Friedreich's Ataxia
                  • Select Phase II Compounds in Development for Friedreich's Ataxia
                  • Select Compounds in Early-Phase Development for Friedreich's Ataxia
                • Key Discontinuations and Failures in Friedreich's Ataxia
                  • Expert Insight: Key Discontinuations and Failures
                • Patient Registries
                  • Patient Registries for Friedreich's Ataxia
                  • Prominent Friedreich's Ataxia Patient Organizations
                • Orphan Drug Designation
                • Access & Reimbursement Overview
                  • Region-Specific Reimbursement Practices
                    • General Reimbursement Environment: United States
                    • General Reimbursement Environment: EU5
                  • Looking for More?
                • Methodology
                  • Bottom-Up Forecasting Overview
                    • Patient Populations
                    • Drug-Specific Assumptions
                  • Bottom-Up Forecast Assumptions
                    • General Sources of Data
                    • Drug-Treatable Rate Assumptions in Friedreich's Ataxia
                    • Patient-Share Assumptions in Friedreich's Ataxia
                    • General Statements About Pricing
                    • Sources for Drug Prices
                    • Price per Treated Day: 2027
                    • Dosing, Days of Therapy, and Compliance
                    • Out-Year Forecasting
                    • Emerging Therapy Prices
                  • Primary Market Research
                    • Experts Interviewed
                • Appendix
                  • Key Abbreviations Related to Friedreich's Ataxia
                  • Friedreich's Ataxia Bibliography

              Author(s): Laura Darnieder; Amy Bradshaw Kaiser, MS

              Laura Darnieder is a Business Insights Analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, where she is focused on providing market forecasts within the niche biopharmaceutical space.

              Prior to joining DRG, Laura earned a Ph.D. in Neuroscience from Tufts University Sackler School of Graduate Biomedical Sciences. Her research focused on the role of stress neuropeptides and inhibitory signaling in alcohol dependence and drug addiction. She also holds a B.S., summa cum laude, in Psychobiology and Linguistics from Binghamton University (SUNY).

              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.