Amyotrophic Lateral Sclerosis (ALS) – Opportunity Analysis and Forecasts to 2029
- Pages: 128
- Published: September 2020
- Report Code: GDHC116POA
Amyotrophic Lateral Sclerosis (ALS) is a motor neuron degenerative disease which affects both upper motor neurons (UMN) and lower motor neurons (LMN). It is a progressive disease, with patients becoming increasingly dependent on carers and physicians as time from onset increases, and ultimately, it is fatal. ALS can be split into two origins, hereditary – which is known as FALS (Familial ALS) or idiopathic – known as SALS (sporadic ALS).
ALS current treatment includes only two disease-modifying drugs: Sanofi’s Rilutek (riluzole) and Mitsubishi Tanabe’s Radicava/Radicut (edaravone). Two reformulations of riluzole were approved recently: ITF Pharma’s Tiglutik (riluzole oral suspension) in 2018 and Aquestive Therapeutics’ Exservan (riluzole oral film) in 2019. Both reformulations address problems of advanced-stage ALS patients who have difficulty in swallowing (dysphagia). Tiglutik is the first liquid formulation of riluzole to launch in the market; it can also be administrated via percutaneous endoscopic gastrostomy tubes (PEG-tubes), while Exservan is a thin oral film that dissolves on the tongue without the need of any muscle action.
GlobalData estimates that sales of drugs in the ALS market were approximately $282M in 2019 in the 8MM. The US was the largest market, with sales of $197M, representing 70% of all sales from the 8MM. The highest-selling drug in the market was Mitsubishi’s Radicava (edaravone), a free radical scavenger, which generated sales of $162M in the 8MM in 2019.
Global sales in the ALS market are expected to grow to $1.04B by 2029, at a Compound Annual Growth Rate (CAGR) of 13.9%.
Key Questions Answered
What are the key ALS treatments in 2019?
When will the late stage pipeline products launch, and how will it affect drug sales and the overall ALS market in the 8MM?
Which unmet needs will remain unaddressed in the forecast period and what opportunities remain for pharmaceutical companies?
Overview of ALS, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.
Annualized ALS therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in two patient segments (familial and sporadic) forecast from 2019 to 2029.
Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the ALS therapeutics market
Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for ALS therapy. The most promising candidates in Phase IIb and Phase III development are profiled.
Analysis of the current and future market competition in the global ALS therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.
Currently, the only treatment options for ALS are Rilutek (8MM) and Radicava/Radicut (US, Japan, and Canada), neither of which can stop nor significantly slow the progression of the disease. At best, these drugs may increase a patient’s life span by up to six months.
GlobalData anticipates that nine late stage pipeline drugs will launch over the forecast period. These drugs will be used as adjunctive therapies to Rilutek, Tiglutik, Exservan, and Radicava. They aim to address some of the longstanding clinical unmet needs and are expected to be the main driver of growth in the ALS market across the 8MM.
Biogen’s BIIB067 (tofersen) antisense therapy is forecast to be a major player in the ALS market; due to its high ACOT despite its limited patient pool.
There are few therapies in the pipeline that aim at addressing major unmet needs, they will all have to translate promising Phase II results into Phase III successes, which KOLs have stated has been a stumbling block for previous therapies.
Opportunities are expected for pharmaceutical companies to develop drugs which have novel MOAs; which are potential curative agents, or extend patient survival more than the current SOC.
Reasons to buy
The report will enable you to:
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 global ALS therapeutics market.
Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global ALS therapeutics market in the future.
Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.
Collaborative Medicinal Development
Table of Contents
1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Amyotrophic Lateral Sclerosis: Executive Summary
2.1 Global ALS Market Expected to Grow to $1.04B in 2029
2.2 Biogen, Mitsubishi, and BrainStorm Are Forecast to Lead the ALS Market By 2029
2.3 Unmet Need Will Remain for the Identification of Curative Drugs and Biomarkers
2.4 ALS Pipeline Moving Toward Disease-Modifying Drugs That Will Likely Transform Treatment Algorithm Especially for FALS Type
2.5 What Do Physicians Think?
3.2 Related Reports
3.3 Upcoming Related Reports
4 Disease Overview
4.1 Etiology and Pathophysiology
4.2 Classification or Staging Systems
4.4 Quality of Life
5.1 Disease Background
5.2 Risk Factors and Comorbidities
5.3 Global and Historical Trends
5.4 Forecast Methodology
5.4.1 Sources Used
5.4.2 Forecast Assumptions and Methods
5.5 Epidemiological Forecast for ALS (2019-2029)
5.5.1 Total Prevalent Cases of ALS
5.5.2 Sex-Specific Total Prevalent Cases of ALS
5.5.3 Age-Specific Total Prevalent Cases of ALS
5.5.4 Total Prevalent Cases of ALS by Type
5.5.5 Diagnosed Prevalent Cases of ALS
5.5.6 Sex-Specific Diagnosed Prevalent Cases of ALS
5.5.7 Age-Specific Diagnosed Prevalent Cases of ALS
5.5.8 Diagnosed Prevalent Cases of ALS by Type
5.6.1 Epidemiological Forecast Insight
5.6.2 Limitations of the Analysis
5.6.3 Strengths of the Analysis
6 Current Treatment Options
7 Unmet Needs and Opportunity Assessment
7.2 Curative Drugs
7.3 Earlier Diagnosis
7.4 Identification of Biomarkers Associated with ALS
7.5 Extended Patient Survival and Improved Control of Symptoms
8 R&D Strategies
8.1.1 Novel Targets
8.1.2 Disease-Modifying Biologic Therapies
8.2 Clinical Trials Design
8.2.1 Clinically Meaningful Endpoints
8.2.2 Clinical Trial Duration
8.2.3 Patient Recruitment
9 Pipeline Assessment
9.2 Innovative Early-Stage Approaches
10 Pipeline Valuation Analysis
10.1 Clinical Benchmark of Key Pipeline Drugs
10.2 Commercial Benchmark of Key Pipeline Drugs
10.3 Competitive Assessment
10.4 Top-Line 10-Year Forecast
11.3.1 Forecasting Methodology
11.3.2 Diagnosed Patients
11.3.3 Percent Drug-Treated Patients
11.3.4 Drugs Included in Each Therapeutic Class
11.3.5 Launch and Patent Expiry Dates
11.3.6 General Pricing Assumptions
11.3.7 Individual Drug Assumptions
11.3.8 Generic Erosion
11.3.9 Pricing of Pipeline Agents
11.4 Primary Research – KOLs Interviewed for This Report
11.5 Primary Research – Prescriber Survey
11.6 About the Authors
11.6.2 Managing Analyst
11.6.3 Therapy Area Director
11.6.5 Managing Epidemiologists
11.6.6 Global Director of Therapy Analysis and Epidemiology
11.6.7 Global Head and EVP of Healthcare Operations and Strategy
11.7 About GlobalData
11.8 Contact Us
List of Tables
Table 1: ALS: Key Metrics in the 8MM
Table 2: Genes Involved in the Familial Forms of ALS
Table 3: Symptoms of ALS
Table 4: Risk Factors and Comorbidities for ALS
Table 5: Leading Treatments for ALS, 2020
Table 6: Treatment Guidelines for ALS
Table 7: Comparison of Therapeutic Classes in Development for ALS, 2019-2029
Table 8: Innovative Early-Stage Approaches for ALS, 2020
Table 9: Clinical Benchmark of Key Pipeline Drugs – ALS
Table 10: Commercial Benchmark of Key Pipeline Drugs – ALS
Table 11: Commercial Benchmark of Key Pipeline Drugs – ALS
Table 12: Key Events Impacting Sales for ALS, 2019-2029
Table 13: ALS Market – Global Drivers and Barriers, 2019-2029
Table 14: Key Historical and Projected Launch Dates for ALS
Table 15: Key Historical and Projected Patent Expiry Dates for ALS
Table 16: High-Prescribing Physicians (non-KOLs) Surveyed, By Country
List of Figures
Figure 1: Global Sales Forecast by Country for ALS in 2019 and 2029
Figure 2: Competitive Assessment of Marketed and Pipeline Drugs, Benchmarked Against the SOC, Rilutek
Figure 3: Pathophysiology of ALS
Figure 4: Staging Systems for ALS
Figure 5: 8MM, Total Prevalence of ALS (%), Both Sexes, Ages ≥40 Years, 2019
Figure 6: 8MM, Diagnosed Prevalence of ALS, Both Sexes, Ages ≥40 Years, 2019
Figure 7: 8MM, Sources Used and Not Used to Forecast the Diagnosed Prevalent Cases of ALS
Figure 8: 8MM, Sources Used to Forecast the Total and Diagnosed Prevalent Cases of ALS by Type
Figure 9: 8MM, Total Prevalent Cases of ALS, N, Both Sexes, Ages ≥40 Years, 2019
Figure 10: 8MM, Total Prevalent Cases of ALS, N, by Sex, Ages ≥40 Years, 2019
Figure 11: 8MM, Total Prevalent Cases of ALS by Age, N, Both Sexes, 2019
Figure 12: 8MM, Total Prevalent Cases of ALS by Type, N, Both Sexes, Ages ≥40 Years, 2019
Figure 13: 8MM, Diagnosed Prevalent Cases of ALS, N, Both Sexes, Ages ≥40 Years, 2019
Figure 14: 8MM, Diagnosed Prevalent Cases of ALS, N, by Sex, Ages ≥40 Years, 2019
Figure 15: 8MM, Diagnosed Prevalent Cases of ALS by Age, N, Both Sexes, 2019
Figure 16: 8MM, Diagnosed Prevalent Cases of ALS by Type, N, Both Sexes, Ages ≥40 Years, 2019
Figure 17: Unmet Needs and Opportunities in ALS
Figure 18: Overview of the Development Pipeline in ALS
Figure 19: Key Phase IIb and III Trials for Promising Pipeline Agents in ALS
Figure 20: Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against the SOC, Rilutek
Figure 21: Global (8MM) Sales Forecast by Country for ALS in 2019 and 2029
Figure 22: Sales Forecast by Class for ALS in the US in 2019 and 2029
Figure 23: Sales Forecast by Class for ALS in the 5EU in 2019 and 2029
Figure 24: Sales Forecast by Class for ALS in Japan in 2019 and 2029
Figure 25: Sales Forecast by Class for ALS in Canada in 2019 and 2029