OpportunityAnalyzer: Amyotrophic Lateral Sclerosis (ALS) - Opportunity Analysis and Forecasts to 2018

Published: June 2014
No. of Pages: 134

Amyotrophic lateral sclerosis (ALS) is a rare, but fatal, motor neuron disease (MND) characterized by axonal degeneration and progressive loss of motor neurons. ALS affects both the upper (brain to spinal cord) and lower (brain stem to muscle tissue) motor neurons, gradually reducing a patient's ability to initiate and control muscle movement. Muscle atrophy, starting in the periphery (arms and legs), ultimately leads to complete paralysis after several years of progressive worsening of muscle strength. Respiratory failure is the most common cause of death, as smooth muscle function becomes affected when the disease is in its final stages. The progressively degenerative course of the condition is a great burden for both patients and caregivers, as well as for society. ALS usually affects people age =40 years, and has a worldwide incidence of about two cases per 100,000 population, and a prevalence ranging between four and seven cases per 100,000.


Key Questions Answered

  • How does the patent expiration of Rilutek effect the ALS market?
  • The pipeline consists of novel but unproven mechanisms of action in ALS. Which of these therapies will attain high sales revenues during 2013-2018, and in which markets?
  • How large an impact will Methycobal and Edaravone have on the ALS market in Japan? What do key opinion leaders think of these treatment approaches?
  • What effects will advancing diagnostic and disease modeling capabilities have on drug development?
  • What regulatory efforts are likely to affect drug development in the markets researched? Which is the largest growth market globally?

Key Findings

  • Country-specific orphan drug and rare disease policy heavily influences development opportunities in the 7MM.
  • Discovery of potential symptomatic and disease-modifying therapies in early stage clinical development is challenged by the absence of ALS biomarkers and disease staging that can demonstrate a patient's response to treatment.
  • Pharmaceutical products for ALS will face growing competition from gene and stem cell-based therapies.
  • Advances in animal modeling are expected to have a significant impact on ALS drug development.


  • Overview of ALS, including etiology and pathophysiology, general symptoms, quality of life, country-specific treatment recommendations and epidemiologic data.
  • Annualized ALS market revenue, annual cost of treatment and usage patterns from 2013 and forecast for five years to 2018.
  • Key topics covered include unmet needs analysis, research and development strategies, pipeline assessment, and pipeline valuation analysis for the ALS market.
  • Pipeline valuation analysis: clinical and commercial benchmarking of AB Science's AB-1010 (masitinib), Cytokinetics' CK-2017357 (tirasemtiv), Mitsubishi Tanabe's Radicut (edaravone), Eisai's Methycobal (mecobalamin), and Orphazyme ApS' arimoclomol.
  • Assessment of the current and future market competition in the global ALS drug market. Insightful review of the key industry and governmental drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
  • Develop business strategies by understanding the trends shaping and driving the ALS market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global ALS market in the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Track drug sales in the global ALS market from 2013-2018.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

OpportunityAnalyzer: Amyotrophic Lateral Sclerosis (ALS) - Opportunity Analysis and Forecasts to 2018

Table of Contents

1 Table of Contents 8
1.1 List of Tables 12
1.2 List of Figures 14

2 Introduction 15
2.1 Catalyst 15
2.2 Upcoming Related Reports 15

3 Disease Overview 16
3.1 Etiology and Pathophysiology 17
3.2 Symptoms 23
3.2.1 Prognosis and Diagnosis 24
3.2.2 Quality of Life 27

4 Epidemiology 28
4.1 Risk Factors and Comorbidities 28
4.2 Global Trends 30
4.2.1 US 30
4.2.2 5EU 31
4.2.3 Japan 33
4.3 Forecast Methodology 33
4.3.1 Sources Used 34
4.3.2 Sources Not Used 37
4.3.3 Forecast Assumptions and Methods, Incident Cases 38
4.4 Epidemiological Forecast for ALS (2013-2023) 41
4.4.1 Diagnosed Prevalent Cases of ALS 41
4.4.2 Age-Specific Incident Cases of ALS 42
4.4.3 Sex-Specific Diagnosed Prevalent Cases of ALS 44
4.4.4 Age-Standardized Diagnosed Prevalence of ALS 46
4.5 Discussion 48
4.5.1 Epidemiological Forecast Insight 48
4.5.2 Limitations of the Analysis 48
4.5.3 Strengths of the Analysis 49

5 Current Treatment Options 50
5.1 Overview 50
5.2 Product Profiles - Major Brands 53
5.2.1 Rilutek (riluzole) 53

6 Unmet Needs Assessment and Opportunity Analysis 57
6.1 Overview 57
6.2 Unmet Needs Analysis 59
6.2.1 Therapies That Can Slow or Halt Disease Progression 59
6.2.2 Appetite-Stimulating Agents 59
6.2.3 Preservation of Muscle Strength/Mobility 60
6.2.4 Biomarkers for Diagnosis/Staging 60
6.2.5 Treatments for Sialorrhea 61
6.2.6 Protection of Cognitive Function 62
6.2.7 Relief of Spasticity, Cramps, and Pain 63
6.2.8 Extending Patient Survival 63
6.2.9 Increasing Patient Function 63

7 Research and Development Strategies 65
7.1 Overview 65
7.1.1 Anti-Glutamate Agents 65
7.1.2 Antioxidants and Mitochondrial Preservation 67
7.1.3 Growth Factors 67
7.1.4 Anti-Apoptosis 68
7.1.5 Immunosuppression 69
7.1.6 Protein Aggregation 70
7.2 Clinical Trial Design 71

8 Pipeline Assessment 73
8.1 Overview 73
8.2 Drugs in Late-Stage Clinical Development 73
8.2.1 AB-1010 (masitinib) 74
8.2.2 Radicut (edaravone) 77
8.2.3 Methycobal (mecobalamin) 80
8.2.4 Arimoclomol 82
8.2.5 CK-2017357 (CK-357, tirasemtiv) 84
8.3 Innovative Early-Stage Approaches 88

9 Pipeline Valuation Analysis 90
9.1 Clinical Benchmarks of Key Pipeline Drugs 91
9.2 Commercial Benchmarks of Key Pipeline Drugs 93
9.3 Competitive Assessment 94
9.4 Top-Line Five-Year Forecast 96
9.4.1 US 98
9.4.2 5EU 99
9.4.3 Japan 100

10 Appendix 103
10.1 Bibliography 103
10.2 Abbreviations 118
10.3 Methodology 122
10.4 Forecast Methodology 122
10.4.1 Diagnosed and Treated ALS Patients 124
10.4.2 Launch and Patent Expiry Dates 124
10.4.3 General Pricing Assumptions 125
10.4.4 Individual Drug Assumptions 126
10.4.5 Generic Erosion 128
10.4.6 Pricing of Pipeline Agents 129
10.5 Physicians and Specialists Included in This Study 130
10.6 About the Authors 131
10.6.1 Author 131
10.6.2 Epidemiologist 132
10.6.3 Global Head of Healthcare 132
10.7 About GlobalData 133
10.8 Disclaimer 133

List of Tables

Table 1: Common MNDs 19
Table 2: Genes Related to FALS 22
Table 3: Main Presentations of ALS 26
Table 4: WFN Anatomic Levels of ALS 26
Table 5: Risk Factors and Comorbidities for ALS 29
Table 6: Incidence (Cases per 100,000 Population) of ALS in the US, as Reported in Epidemiological Studies 30
Table 7: Prevalence (Cases per 100,000 Population) of ALS in the US, as Reported in Epidemiological Studies 31
Table 8: Incidence (Cases per 100,000 Population) of ALS in the European Markets, as Reported in Epidemiological Studies 32
Table 9: Prevalence (Cases per 100,000 Population) of ALS in the European Markets, as Reported in Epidemiological Studies 32
Table 10: El Escorial Diagnostic Criteria for ALS 33
Table 11:7MM, Sources of Diagnosed Prevalence Data Used to Forecast the Diagnosed Prevalent Cases of ALS 34
Table 12: 7MM, Diagnosed Prevalent Cases of ALS, Age ?40 Years, Both Sexes, N, 2013-2023 41
Table 13: 7MM, Diagnosed Prevalent Cases of ALS, by Age, Both Sexes, N, (Row %), 2013 43
Table 14: 7MM, Diagnosed Prevalent Cases of ALS, by Sex, Age ?40 Years, N (Row %), 2013 45
Table 15: Management of ALS Symptoms 51
Table 16: Overview of Only FDA- Approved ALS Treatment 52
Table 17: Product Profile - Rilutek 54
Table 18: Rilutek SWOT Analysis 56
Table 19: Overall Unmet Needs - Current Level of Attainment 58
Table 20: ALS - Late Stage Pipeline, 2014 74
Table 21: AB-1010 Product Profile 75
Table 22: AB-1010 SWOT Analysis 77
Table 23: Product Profile - Radicut 79
Table 24: Methycobal Product Profile 81
Table 25: Arimoclomol Product Profile 83
Table 26: Arimoclomol SWOT Analysis 84
Table 27: CK-2017357 Product Profile 86
Table 28: CK-2017357 SWOT Analysis 88
Table 29: Key Mid- and Early-Stage Pipeline Drugs in ALS 89
Table 30: Clinical Benchmarks of the Late-Stage ALS Pipeline 91
Table 31: Commercial Benchmarks of the Late-Stage ALS Pipeline 93
Table 32: Top-Line Sales Forecast ($) for ALS in the 7MM, 2013-2018 97
Table 33: Key Events in the ALS Drug Market, 7MM, 2013-2018 101
Table 34: Drivers and Barriers in the ALS Drug Market, 7MM, 2013-2018 102
Table 35: Key Launch Dates 124
Table 36: Key Patent Expiries 124

List of Figures

Figure 1: Anatomy of Upper and Lower Motor Neurons 16
Figure 2: Neuromuscular Changes in ALS 17
Figure 3: Neurodegeneration in ALS 18
Figure 4: 7MM, Diagnosed Prevalent Cases of ALS, Age ?40 Years, Both Sexes, N, 2013-2023 42
Figure 5: 7MM, Diagnosed Prevalent Cases of ALS, by Age, Both Sexes, N, 2013 44
Figure 6: 7MM, Diagnosed Prevalent Cases of ALS, by Sex, Age ?40 Years, N, 2013 46
Figure 7: 7MM, Age-Standardized Diagnosed Prevalence (%) of ALS, Age ?40 Years, 2013 47
Figure 8: Competitive Assessment of Late-Stage Pipeline Agents in ALS, 2013-2018 96
Figure 9: Global Sales for ALS by Country, 2013-2018 98

Published By: GlobalData
Product Code: GlobalData29951

Did you find what you are/were looking for ? If not, read below and browse through other relevant pages for similar market research reports OR get in touch with us through the form/contact info in your right navigation panel and we´┐Żll share relevant market report titles for you to explore.

Related Reports:

Why ReportsnReports.com?

  • 500,000 market research reports and growing.
  • Top Fortune 500 Organizations trust us for research data.
  • 24/7 support on call as well as emails.
  • Your Details are safe with us.
  • Free support for your research requirements.
  • Report Delivery: Email
  • Delivery Time:
    • Upto 24 hrs - working days
    • Upto 48 hrs max - weekends and public holidays

Research Support / Custom Research

If this business study does not have the data and information analysis you need, contact us with your research requirements. We explore available market research reports to map your needs and share report titles with you. Alternatively, we can also offer custom research that suits your budget and timelines. Share your information requirements here.
Connect With Us
Email: sales@reportsandreports.com Call : + 1 888 391 5441

Subscription Option

Using our subscription option, you get access to market research reports and industry data of Pharmaceuticals market as per your needs. Get the best of Pharmaceuticals research reports by utilizing your research budgets in an optimum way.

More about our Subscription option
Email Alerts

Get Email alerts about market research reports from industries and publishers of your interest: