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 2030?
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 2030? 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), drug treated prevalence
Amyotrophic Lateral Sclerosis Key Findings - August 2021
Commercial Outlook
Key Findings
Regional Sales of Key Therapies to Treat Amyotrophic Lateral Sclerosis: 2020 and 2030
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 Scavengers
Antisense Oligonucleotides
Stem Cell Therapy
Histone Deacetylase Inhibitors
Complement C5 Inhibitors
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: 2020-2030
Disease Definition
Methods
Sources Used for Subtypes of Amyotrophic Lateral Sclerosis
Number of Diagnosed Prevalent Cases of Amyotrophic Lateral Sclerosis by Subtype: 2020-2030
Number of Drug-Treated Prevalent Cases of Amyotrophic Lateral Sclerosis: 2020-2030
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
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
Drug Pipeline
Pipeline
Regulatory Milestones
Indication Comparison
Emerging Therapies
Key Findings
Pipeline Overview
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
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
ION363
Key Ongoing Clinical Trials Investigating ION363 for the Treatment of Amyotrophic Lateral Sclerosis
Expectations for Launch and Sales Opportunity for ION363 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
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
Key Ongoing Clinical Trials Investigating Masitinib for the Treatment of Amyotrophic Lateral Sclerosis
Expectations for Launch and Sales Opportunity for Masitinib in Amyotrophic Lateral Sclerosis
AMX0035 Profile
Key Ongoing Clinical Trials Investigating AMX0035 for the Treatment of Amyotrophic Lateral Sclerosis
Expert Insights: AMX0035
Expectations for Launch and Sales Opportunity for AMX0035 in Amyotrophic Lateral Sclerosis
Reldesemtiv Profile
Expectations for Launch and Sales Opportunity for Reldesemtiv in Amyotrophic Lateral Sclerosis
Key Ongoing Clinical Trials Investigating Pridopidine for the Treatment of Amyotrophic Lateral Sclerosis
Expectations for Launch and Sales Opportunity for Pridopidine 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-Au8 for the Treatment of Amyotrophic Lateral Sclerosis
Expectations for Launch and Sales Opportunity for CNM-Au8 in Amyotrophic Lateral Sclerosis
Ibudilast Profile
Key Ongoing Clinical Trials Investigating Ibudilast for the Treatment of Amyotrophic Lateral Sclerosis
Expectations for Launch and Sales Opportunity for Ibudilast 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
Tapish Dogra
Tapish Dogra, MTech, is an analyst on the Infectious, Niche & Rare Diseases team at Clarivate. In this role he works on a range of indications as well as numerous niche and rare diseases. Tapish brings five years of experience in market assessment, competitive intelligence, business intelligence, and procurement intelligence, and has worked on various therapeutic areas to provide strategic recommendations to pharma companies. He holds a MTech degree in Biotechnology from Jaypee University of Information Technology, Himachal Pradesh.
Swarali Tadwalkar
Swarali Tadwalkar, M.P.H., is a principal epidemiologist at Clarivate. Previously, she was involved in primary and secondary healthcare research, including projects in digital health, health policy and management, and health economics and outcomes research (HEOR). Ms. Tadwalkar also coordinated various nongovernmental public health projects focusing on access to treatment for hepatitis and human papilloma virus. She received her M.P.H. from the University of South Florida in Tampa.