Amyotrophic Lateral Sclerosis (ALS) – Opportunity Analysis and Forecasts to 2029

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?

Scope

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.

Key Highlights

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.

Companies mentioned

Sanofi

Mitsubishi Tanabe

Brainstorm Therapeutics

Avanir

ViroMed

Ionis Pharmaceuticals

ViiV

GlaxoSmithKline

Biogen

Orphazyme

Orion Pharmaceuticals

Pfizer

Aquestive Therapeutics

Alexion

Amylyx

Neuralstem

Retrotope

Bruschettini

ITF Pharam

Roche

Treeway BV

Cytokientics

AB Science

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 Introduction

3.1 Catalyst

3.2 Related Reports

3.3 Upcoming Related Reports

4 Disease Overview

4.1 Etiology and Pathophysiology

4.1.1 Etiology

4.1.2 Pathophysiology

4.2 Classification or Staging Systems

4.3 Symptoms

4.3.1 Complications

4.4 Quality of Life

5 Epidemiology

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 Discussion

5.6.1 Epidemiological Forecast Insight

5.6.2 Limitations of the Analysis

5.6.3 Strengths of the Analysis

6 Current Treatment Options

6.1 Overview

7 Unmet Needs and Opportunity Assessment

7.1 Overview

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 Overview

8.1.1 Novel Targets

8.1.2 Disease-Modifying Biologic Therapies

8.1.3 Reformulations

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

9.1 Overview

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

10.4.1 US

10.4.2 5EU

10.4.3 Japan

10.4.4 Canada

11 Appendix

11.1 Bibliography

11.2 Abbreviations

11.3 Methodology

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

11.4.1 KOLs

11.4.2 Payers

11.5 Primary Research – Prescriber Survey

11.6 About the Authors

11.6.1 Analyst

11.6.2 Managing Analyst

11.6.3 Therapy Area Director

11.6.4 Epidemiologist

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

11.9 Disclaimer

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

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