Amyotrophic lateral sclerosis (ALS) is a degenerative motor neuron disorder that leads to progressive muscle wasting and weakness. The typical survival time is three to five years from onset. The FDA’s 2017 approval of Mitsubishi Tanabe Pharma’s Radicava resulted in the second approved treatment option for ALS, yet pressing unmet need remains in this arena. The ALS pipeline comprises diverse programs designed to be neuroprotective, to enhance respiratory function, or to restore lost neurons in the spinal cord; however, clinical success in ALS has historically been a formidable challenge. Nonetheless, clinical and commercial opportunity exists for developers that can overcome the scientific and clinical hurdles blocking the path to regulatory approval for the treatment of this devastating disease.

QUESTIONS ANSWERED

  • How has Mitsubishi Tanabe’s Radicava been incorporated into the treatment of U.S. ALS patients? What are the key advantages and disadvantages of the product?
  • How will the size of the drug-treated ALS population change through 2029?
  • What are the key unmet treatment needs in ALS?
  • Which emerging therapies have ALS experts most intrigued or optimistic? How would new therapies influence the management of ALS patients?
  • How are emerging drugs being evaluated, and which are likely to launch by 2029? What commercial impact will they have on the ALS market?

PRODUCT DESCRIPTION

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

Scope:

  • Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom
  • Primary research: Six country-specific interviews with ALS neurologists
  • Epidemiology: Diagnosed prevalence, diagnosed prevalence by disease type (familial or sporadic ALS), diagnosed prevalence by comorbid frontotemporal dementia
  • Emerging therapies: Phase III: 6; Phase II/III: 5; Phase II & I/II: 23; coverage of select preclinical and Phase I products
  • Market forecast and alternative market scenarios: Drug-level sales and patient share of select ALS therapies in 2019 and 2029
  • Key companies: Mitsubishi Tanabe Pharma, Biogen, Ionis, Orphazyme, Orion Pharma, AB Science, Seneca Biopharma, BrainStorm Cell Therapeutics, Alexion Pharmaceuticals, and Halozyme Therapeutics
  • Key drugs: Radicava (edaravone), BIIB-067, arimoclomol, levosimendan, masitinib, riluzole, NSI-566, mesenchymal stem cells expanded and induced to secrete neurotrophic factors (MSC-NTF cells), and Ultomiris

Table of contents

  • Amyotrophic Lateral Sclerosis - Landscape & Forecast - Disease Landscape & Forecast 2020
    • COVID-19
      • Key Findings
        • Amyotrophic Lateral Sclerosis Key Findings - September 2020
      • Commercial Outlook
        • Key Findings
          • Regional Sales of Key Therapies to Treat Amyotrophic Lateral Sclerosis: 2019 and 2029
          • Amyotrophic Lateral Sclerosis SWOT Analysis
        • COVID-19: Areas of Potential Forecast Impact
          • Drivers and Constraints
            • What Factors Are Driving Sales in Amyotrophic Lateral Sclerosis?
            • What Factors Are Constraining Sales in Amyotrophic Lateral Sclerosis?
          • Drug-Class-Specific Trends
            • Free Radical Scavenger
            • Antisense Oligonucleotides
            • Stem Cell Therapy
            • Molecular Chaperone Activators
        • Forecast
          • Sales of Key Therapies in Amyotrophic Lateral Sclerosis
        • Etiology and Pathophysiology
          • Etiology
            • Etiology of Amyotrophic Lateral Sclerosis
            • Select Genes Associated with Amyotrophic Lateral Sclerosis
            • Select Genes Associated with Amyotrophic Lateral Sclerosis
          • Pathophysiology
            • Pathophysiology of Amyotrophic Lateral Sclerosis
            • Molecular Mechanisms of Motor Neuron Degeneration in Amyotrophic Lateral Sclerosis
            • Pathophysiology of Amyotrophic Lateral Sclerosis
          • Disease Presentation
            • Signs and Symptoms of Amyotrophic Lateral Sclerosis
            • Expert Insight: Disease Presentation
          • Overlap of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
            • Physician Opinion on the Overlap of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
          • Disease Burden
            • Expert Insight: Disease Burden
          • Prognostic Factors
            • Expert Insight: Prognostic Factors
          • Key Pathways and Drug Targets
          • Epidemiology
            • Introduction
              • Key Findings
            • Epidemiology Populations
              • Disease Definition
              • Methods
              • Sources Used for Diagnosed Prevalent Cases of Amyotrophic Lateral Sclerosis
              • Number of Diagnosed and Drug-Treated Prevalent Cases of Amyotrophic Lateral Sclerosis in the Major Pharmaceutical Markets: 2019-2029
              • Disease Definition
              • Methods
              • Sources Used for Subtypes of Amyotrophic Lateral Sclerosis
              • Number of Diagnosed Prevalent Cases of Amyotrophic Lateral Sclerosis by Subtype: 2019-2029
              • Number of Drug-Treated Prevalent Cases of Amyotrophic Lateral Sclerosis: 2019-2029
          • Current Treatment
            • Key Findings
              • Diagnosis
                • Overview
                • Requirements for the Diagnosis of Amyotrophic Lateral Sclerosis
                • Conditions Considered as Part of a Differential Diagnosis of Amyotrophic Lateral Sclerosis
                • Diagnostic Criteria for Amyotrophic Lateral Sclerosis
                • Treatment Providers and Referral Patterns for Amyotrophic Lateral Sclerosis
                • Expert Insight: Diagnosis of Amyotrophic Lateral Sclerosis
                • Expert Insight: Referral Patterns for Amyotrophic Lateral Sclerosis
              • Treatment Goals
                • Key Endpoints Used in Clinical Trials for Amyotrophic Lateral Sclerosis
                • Key Endpoints Used in Clinical Trials for Amyotrophic Lateral Sclerosis
                • Staging Systems of Amyotrophic Lateral Sclerosis
                • Expert Insight: Clinical Endpoints
              • Key Current Therapies
                • Mechanism of Action of Key Current Drug Classes Used for Amyotrophic Lateral Sclerosis
                • Current Treatments Used for Amyotrophic Lateral Sclerosis
                • Market Events Impacting the Use of Key Current Therapies in Amyotrophic Lateral Sclerosis
                • Efficacy Outcomes of Riluzole Trials in Amyotrophic Lateral Sclerosis
                • Expert Insight: Use of Riluzole in Amyotrophic Lateral Sclerosis
                • Efficacy Outcomes from Nuedexta Trials in Amyotrophic Lateral Sclerosis
                • Expert Insight on the Use of Nuedexta in Amyotrophic Lateral Sclerosis
                • Select Clinical Trial Results for Edaravone in the Treatment of Amyotrophic Lateral Sclerosis
                • Expert Insight: Edaravone
                • Symptomatic Treatment of Amyotrophic Lateral Sclerosis
                • Symptomatic Treatment of Amyotrophic Lateral Sclerosis
                • Expert Insight: Symptomatic Treatment in Amyotrophic Lateral Sclerosis
              • Medical Practice
                • Overview
                • Amyotrophic Lateral Sclerosis Treatment Guidelines by Market
                • Expert Insight: Nonpharmacological Interventions for Amyotrophic Lateral Sclerosis
                • Multidisciplinary Centers for Amyotrophic Lateral Sclerosis
                • Multidisciplinary Team Members
                • Expert Insight: Multidisciplinary Approach to Treating Amyotrophic Lateral Sclerosis
                • Multidisciplinary Care Model
                • Region-Specific Treatment
            • Unmet Need Overview
              • Key Findings
                • Expert Insight
              • Attainment of Unmet Needs
                • Current Attainment of Unmet Needs in Amyotrophic Lateral Sclerosis
                • Future Attainment of Unmet Needs in Amyotrophic Lateral Sclerosis
                • Top Unmet Needs in Amyotrophic Lateral Sclerosis: Current and Future Attainment
                • Expert Insight: Unmet Needs in Amyotrophic Lateral Sclerosis
            • Emerging Therapies
              • Key Findings
                • Pipeline Overview
                • Pipeline Trends for Amyotrophic Lateral Sclerosis
              • Key Emerging Therapies
                • Key Therapies in Development for Amyotrophic Lateral Sclerosis
                • Estimated Launch Dates of Key Emerging Therapies for the Treatment of Amyotrophic Lateral Sclerosis
                • MSC-NTF Cells Profile
                • Key Ongoing Clinical Trials Investigating MSC-NTF Cells for the Treatment of Amyotrophic Lateral Sclerosis
                • Expert Insight: MSC-NTF Cells
                • Expectations for Launch and Sales Opportunity for MSC-NTF Cells in Amyotrophic Lateral Sclerosis
                • NSI-566 Profile
                • Expectations for Launch and Sales Opportunity for NSI-566 in Amyotrophic Lateral Sclerosis
                • Tofersen Profile
                • Key Ongoing Clinical Trials Investigating Tofersen for the Treatment of Amyotrophic Lateral Sclerosis
                • Expert Insight: Tofersen
                • Expectations for Launch and Sales Opportunity for Tofersen in Amyotrophic Lateral Sclerosis
                • Key Results from Select Clinical Trials Investigating Arimoclomol for the Treatment of Amyotrophic Lateral Sclerosis
                • Key Ongoing Clinical Trials Investigating Arimoclomol for the Treatment of Amyotrophic Lateral Sclerosis
                • Expert Insight: Arimoclomol
                • Expectations for Launch and Sales Opportunity for Arimoclomol in Amyotrophic Lateral Sclerosis
                • Select Clinical Trial Results for Masitinib in the Treatment of Amyotrophic Lateral Sclerosis
                • Expectations for Launch and Sales Opportunity for Masitinib in Amyotrophic Lateral Sclerosis
                • Reldesemtiv Profile
                • Expectations for Launch and Sales Opportunity for Reldesemtiv in Amyotrophic Lateral Sclerosis
                • Levosimendan Profile
                • Key Ongoing Clinical Trials Investigating Levosimendan for the Treatment of Amyotrophic Lateral Sclerosis
                • Expectations for Launch and Sales Opportunity for Levosimendan in Amyotrophic Lateral Sclerosis
                • Key Ongoing Clinical Trials Investigating Ultomiris for the Treatment of Amyotrophic Lateral Sclerosis
                • Expectations for Launch and Sales Opportunity for Ultomiris in Amyotrophic Lateral Sclerosis
                • Key Ongoing Clinical Trials Investigating Zilucoplan for the Treatment of Amyotrophic Lateral Sclerosis
                • Expectations for Launch and Sales Opportunity for Zilucoplan in Amyotrophic Lateral Sclerosis
                • Key Ongoing Clinical Trials Investigating Verdiperstat for the Treatment of Amyotrophic Lateral Sclerosis
                • Expectations for Launch and Sales Opportunity for Verdiperstat in Amyotrophic Lateral Sclerosis
                • Key Ongoing Clinical Trials Investigating CNM Au 8 for the Treatment of Amyotrophic Lateral Sclerosis
                • Expectations for Launch and Sales Opportunity for CNM Au 8 in Amyotrophic Lateral Sclerosis
              • Early-Phase Pipeline Analysis
                • Select Compounds in Early-Phase Development for Amyotrophic Lateral Sclerosis
              • Key Discontinuations and Failures in Amyotrophic Lateral Sclerosis
                • Patient Registries
                  • Patient Registries for Amyotrophic Lateral Sclerosis
                • Orphan-Drug Designation
                • Access and Reimbursement Overview
                  • Reimbursement of Current Amyotrophic Lateral Sclerosis Therapies
                  • Expert Insight: Reimbursement of Current Amyotrophic Lateral Sclerosis Therapies
                  • Region-Specific Reimbursement Practices
                    • General Reimbursement Environment: United States
                    • General Reimbursement Environment: EU5
                • Appendix
                  • Key Abbreviations Related to Amyotrophic Lateral Sclerosis
                  • Brands, Marketers, and Generic Availability of Key Therapies for Amyotrophic Lateral Sclerosis by Market
                  • Amyotrophic Lateral Sclerosis Bibliography

              Author(s): Natasha Bardhan, M.Pharm; Swarali Tadwalkar

              Natasha Bardhan is a Research Associate for the Infectious, Niche, & Rare Diseases (INRD) team at Decision Resources.

              Natasha Bardhan holds a bachelor’s degree in Pharmacy from DIPSAR, New Delhi and has completed her master’s in Pharmaceutics from Amity University, Noida. Prior to joining DRG, she worked as a Senior Associate at WNS (Healthcare) for two and a half years. She has worked on multiple client projects involving patents, sales and forecasting, pipeline analysis, keywords research and product/company profiling.

              Swarali joined Decision Resources Group (DRG) in 2016 and with the Epidemiology team develops epidemiological populations forecasts for different infectious and non-communicable diseases with her particular interests in the oncology space.

              Prior to joining DRG, she has been extensively involved in primary and secondary healthcare research. Her experience involves projects in digital health, health policy and management, and health economics and outcomes research (HEOR). She has also coordinated various non-governmental public health projects focusing in hepatitis and human papilloma virus treatment access. Swarali holds a Masters in Public Health (Epidemiology) degree from the University of South Florida, Tampa.