Product Code: GDHC019POA
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?
- 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.
Table of Contents
1. Table of Contents
- 1.1. List of Tables
- 1.2. List of Figures
- 2.1. Catalyst
- 2.2. Upcoming Related Reports
3. Disease Overview
- 3.1. Etiology and Pathophysiology
- 3.2. Symptoms
- 3.2.1. Prognosis and Diagnosis
- 3.2.2. Quality of Life
- 4.1. Risk Factors and Comorbidities
- 4.2. Global Trends
- 4.2.1. US
- 4.2.2. 5EU
- 4.2.3. Japan
- 4.3. Forecast Methodology
- 4.3.1. Sources Used
- 4.3.2. Sources Not Used
- 4.3.3. Forecast Assumptions and Methods, Incident Cases
- 4.4. Epidemiological Forecast for ALS (2013-2023)
- 4.4.1. Diagnosed Prevalent Cases of ALS
- 4.4.2. Age-Specific Incident Cases of ALS
- 4.4.3. Sex-Specific Diagnosed Prevalent Cases of ALS
- 4.4.4. Age-Standardized Diagnosed Prevalence of ALS
- 4.5. Discussion
- 4.5.1. Epidemiological Forecast Insight
- 4.5.2. Limitations of the Analysis
- 4.5.3. Strengths of the Analysis
5. Current Treatment Options
- 5.1. Overview
- 5.2. Product Profiles - Major Brands
- 5.2.1. Rilutek (riluzole)
6. Unmet Needs Assessment and Opportunity Analysis
- 6.1. Overview
- 6.2. Unmet Needs Analysis
- 6.2.1. Therapies That Can Slow or Halt Disease Progression
- 6.2.2. Appetite-Stimulating Agents
- 6.2.3. Preservation of Muscle Strength/Mobility
- 6.2.4. Biomarkers for Diagnosis/Staging
- 6.2.5. Treatments for Sialorrhea
- 6.2.6. Protection of Cognitive Function
- 6.2.7. Relief of Spasticity, Cramps, and Pain
- 6.2.8. Extending Patient Survival
- 6.2.9. Increasing Patient Function
7. Research and Development Strategies
- 7.1. Overview
- 7.1.1. Anti-Glutamate Agents
- 7.1.2. Antioxidants and Mitochondrial Preservation
- 7.1.3. Growth Factors
- 7.1.4. Anti-Apoptosis
- 7.1.5. Immunosuppression
- 7.1.6. Protein Aggregation
- 7.2. Clinical Trial Design
8. Pipeline Assessment
- 8.1. Overview
- 8.2. Drugs in Late-Stage Clinical Development
- 8.2.1. AB-1010 (masitinib)
- 8.2.2. Radicut (edaravone)
- 8.2.3. Methycobal (mecobalamin)
- 8.2.4. Arimoclomol
- 8.2.5. CK-2017357 (CK-357, tirasemtiv)
- 8.3. Innovative Early-Stage Approaches
9. Pipeline Valuation Analysis
- 9.1. Clinical Benchmarks of Key Pipeline Drugs
- 9.2. Commercial Benchmarks of Key Pipeline Drugs
- 9.3. Competitive Assessment
- 9.4. Top-Line Five-Year Forecast
- 9.4.1. US
- 9.4.2. 5EU
- 9.4.3. Japan
- 10.1. Bibliography
- 10.2. Abbreviations
- 10.3. Methodology
- 10.4. Forecast Methodology
- 10.4.1. Diagnosed and Treated ALS Patients
- 10.4.2. Launch and Patent Expiry Dates
- 10.4.3. General Pricing Assumptions
- 10.4.4. Individual Drug Assumptions
- 10.4.5. Generic Erosion
- 10.4.6. Pricing of Pipeline Agents
- 10.5. Physicians and Specialists Included in This Study
- 10.6. About the Authors
- 10.6.1. Author
- 10.6.2. Epidemiologist
- 10.6.3. Global Head of Healthcare
- 10.7. About GlobalData
- 10.8. Disclaimer
List of Tables
- Table 1: Common MNDs
- Table 2: Genes Related to FALS
- Table 3: Main Presentations of ALS
- Table 4: WFN Anatomic Levels of ALS
- Table 5: Risk Factors and Comorbidities for ALS
- Table 6: Incidence (Cases per 100,000 Population) of ALS in the US, as Reported in Epidemiological Studies
- Table 7: Prevalence (Cases per 100,000 Population) of ALS in the US, as Reported in Epidemiological Studies
- Table 8: Incidence (Cases per 100,000 Population) of ALS in the European Markets, as Reported in Epidemiological Studies
- Table 9: Prevalence (Cases per 100,000 Population) of ALS in the European Markets, as Reported in Epidemiological Studies
- Table 10: El Escorial Diagnostic Criteria for ALS
- Table 11:7MM, Sources of Diagnosed Prevalence Data Used to Forecast the Diagnosed Prevalent Cases of ALS
- Table 12: 7MM, Diagnosed Prevalent Cases of ALS, Age ≥40 Years, Both Sexes, N, 2013-2023
- Table 13: 7MM, Diagnosed Prevalent Cases of ALS, by Age, Both Sexes, N, (Row %), 2013
- Table 14: 7MM, Diagnosed Prevalent Cases of ALS, by Sex, Age ≥40 Years, N (Row %), 2013
- Table 15: Management of ALS Symptoms
- Table 16: Overview of Only FDA- Approved ALS Treatment
- Table 17: Product Profile - Rilutek
- Table 18: Rilutek SWOT Analysis
- Table 19: Overall Unmet Needs - Current Level of Attainment
- Table 20: ALS - Late Stage Pipeline, 2014
- Table 21: AB-1010 Product Profile
- Table 22: AB-1010 SWOT Analysis
- Table 23: Product Profile - Radicut
- Table 24: Methycobal Product Profile
- Table 25: Arimoclomol Product Profile
- Table 26: Arimoclomol SWOT Analysis
- Table 27: CK-2017357 Product Profile
- Table 28: CK-2017357 SWOT Analysis
- Table 29: Key Mid- and Early-Stage Pipeline Drugs in ALS
- Table 30: Clinical Benchmarks of the Late-Stage ALS Pipeline
- Table 31: Commercial Benchmarks of the Late-Stage ALS Pipeline
- Table 32: Top-Line Sales Forecast ($) for ALS in the 7MM, 2013-2018
- Table 33: Key Events in the ALS Drug Market, 7MM, 2013-2018
- Table 34: Drivers and Barriers in the ALS Drug Market, 7MM, 2013-2018
- Table 35: Key Launch Dates
- Table 36: Key Patent Expiries
List of Figures
- Figure 1: Anatomy of Upper and Lower Motor Neurons
- Figure 2: Neuromuscular Changes in ALS
- Figure 3: Neurodegeneration in ALS
- Figure 4: 7MM, Diagnosed Prevalent Cases of ALS, Age ≥40 Years, Both Sexes, N, 2013-2023
- Figure 5: 7MM, Diagnosed Prevalent Cases of ALS, by Age, Both Sexes, N, 2013
- Figure 6: 7MM, Diagnosed Prevalent Cases of ALS, by Sex, Age ≥40 Years, N, 2013
- Figure 7: 7MM, Age-Standardized Diagnosed Prevalence (%) of ALS, Age ≥40 Years, 2013
- Figure 8: Competitive Assessment of Late-Stage Pipeline Agents in ALS, 2013-2018
- Figure 9: Global Sales for ALS by Country, 2013-2018