Amyotrophic Lateral Sclerosis – Landscape & Forecast – Disease Landscape & Forecast
Amyotrophic lateral sclerosis (ALS) is a progressively debilitating disorder of the central nervous system characterized by motor neuron degeneration. ALS causes progressive weakness and atrophy of the muscles and loss of voluntary functions, including breathing, swallowing, use of limbs, and eventually death. Four treatments are approved for ALS in the markets under study: Mitsubishi Tanabe Pharma’s Radicava (edaravone); riluzole (Advanz Pharma / Sanofi’s Rilutek, other brands, generics); and, most recently, Amylyx Pharmaceuticals’ Relyvrio (sodium phenylbutyrate and taurursodiol [AMX0035]) and Ionis Pharmaceuticals / Biogen’s Qalsody (tofersen). However, pressing unmet need remains. Although the ALS pipeline comprises diverse programs designed to be neuroprotective, enhance respiratory function, or restore lost neurons in the spinal cord, clinical success in ALS has historically been a formidable challenge. Thus, substantial clinical and commercial opportunity awaits developers that can develop a safe, effective therapy for this crippling disease.
QUESTIONS ANSWERED
- What role do riluzole and, in the United States, Radicava play in the treatment of ALS patients? How are Relyvrio and Qalsody being incorporated into medical practice? What are the key advantages and disadvantages of these products?
- How will the size of the drug-treated ALS population change through 2032?
- What are the key unmet needs in the treatment of ALS?
- Which emerging therapies are ALS experts most intrigued or optimistic about? How would new therapies influence the management of ALS patients?
- Which emerging therapies for ALS are likely to launch by 2032? What commercial impact will they have on the 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:
- Geography: United States and EU5
- Primary research: Six country-specific interviews with thought-leading neurologists; supported by survey data collected for this study
- Epidemiology: Diagnosed prevalent and drug-treated cases of ALS by country, diagnosed prevalence by disease type (familial or sporadic)
- Forecast: Drug-level sales and patient share of key ALS therapies through 2032
- Emerging therapies: Phase 3/PR: 12; Phase 2: 7; Phase 1: 3
SOLUTION ENHANCEMENT
Niche & Rare Disease Landscape & Forecast features a Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.
Table of contents
- Amyotrophic Lateral Sclerosis - Landscape & Forecast - Disease Landscape & Forecast
- Key Findings
- Amyotrophic Lateral Sclerosis Key Findings - September 2023
- Commercial Outlook
- Key findings
- Regional sales of key therapies to treat amyotrophic lateral sclerosis: 2022 and 2032
- Amyotrophic lateral sclerosis SWOT analysis
- 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
- Key findings
- Forecast
- Sales of key therapies in amyotrophic lateral sclerosis
- Etiology and Pathophysiology
- Etiology and pathophysiology
- Disease overview
- Expert insight
- 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
- Disease burden
- Expert insight: disease burden
- Prognostic factors
- Expert insight: prognostic factors
- Key pathways and drug targets
- Etiology and pathophysiology
- Epidemiology
- Introduction
- Key findings
- Epidemiology populations
- Disease definition, methods, and sources used
- Diagnosed and drug-treated prevalent cases of amyotrophic lateral sclerosis: 2022-2032
- Disease definition, methods, and sources used
- Diagnosed prevalent cases of amyotrophic lateral sclerosis by subtype: 2022-2032
- Number of drug-treated prevalent cases of amyotrophic lateral sclerosis: 2022-2032
- Introduction
- 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
- 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: treatment goals
- 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
- Key results from select clinical trials investigating riluzole for the treatment of amyotrophic lateral sclerosis
- Expert insight: use of riluzole in amyotrophic lateral sclerosis
- Key results from select clinical trials investigating edaravone for the treatment of amyotrophic lateral sclerosis
- Key ongoing clinical trials of edaravone in the treatment of amyotrophic lateral sclerosis
- Expert insight: edaravone
- Key results from select clinical trials results investigating Relyvrio for the treatment of amyotrophic lateral sclerosis
- Key ongoing clinical trials of Relyvrio in the treatment of amyotrophic lateral sclerosis
- Expert insight: Relyvrio
- Select clinical trial results for Qalsody in the treatment of amyotrophic lateral sclerosis
- Key ongoing clinical trials of Qalsody in the treatment of amyotrophic lateral sclerosis
- Expert insight: Qalsody
- Select clinical trial results for Nuedexta in the treatment of amyotrophic lateral sclerosis
- Expert insight on the use of Nuedexta in 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
- Key findings
- Unmet Need Overview
- Current and 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
- Expert insight: MSC-NTF cells
- Expectations for launch and sales opportunity for MSC-NTF cells in amyotrophic lateral sclerosis
- Key ongoing clinical trials of ION363 in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for ION363 in amyotrophic lateral sclerosis
- Key results of select clinical trials investigating masitinib for the treatment of amyotrophic lateral sclerosis
- Key ongoing clinical trials of masitinib in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for masitinib in amyotrophic lateral sclerosis
- Reldesemtiv profile
- Expectations for launch and sales opportunity for reldesemtiv in amyotrophic lateral sclerosis
- Ibudilast profile
- Key ongoing clinical trials of ibudilast in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for ibudilast in amyotrophic lateral sclerosis
- FNP122 profile
- Key ongoing clinical trials of FNP122 in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for FNP122 in amyotrophic lateral sclerosis
- TUDCA profile
- Key ongoing clinical trials of TUDCA in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for TUDCA in amyotrophic lateral sclerosis
- Expert insight: HEALEY ALS Platform Trial
- Key ongoing clinical trials of verdiperstat in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for verdiperstat in amyotrophic lateral sclerosis
- Key ongoing clinical trials of CNM-Au8 in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for CNM-Au8 in amyotrophic lateral sclerosis
- Key ongoing clinical trials of pridopidine in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for pridopidine in amyotrophic lateral sclerosis
- Key ongoing clinical trials of SLS-005 (trehalose) in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for SLS-005 (trehalose) in amyotrophic lateral sclerosis
- Key ongoing clinical trials of ABBV-CLS-7262 in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for ABBV-CLS-7262 in amyotrophic lateral sclerosis
- Key ongoing clinical trials of DNL343 in the treatment of amyotrophic lateral sclerosis
- Expectations for launch and sales opportunity for DNL343 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
- Key findings
- Access and Reimbursement Overview
- Region-specific reimbursement practices
- General reimbursement environment: United States
- General reimbursement environment: EU5
- Region-specific reimbursement practices
- Appendix
- Key abbreviations related to amyotrophic lateral sclerosis
- Amyotrophic lateral sclerosis bibliography
- Key Findings
Muthana Sweis, M.P.H., Healthcare Research & Data Analyst, Infectious, Niche, and Rare Diseases. Muthana conducts primary and secondary research for a range of indications and authors reports related to his findings. He earned his master’s degree in public health from DePaul University.
Julie Davis, M.P.H., Senior Epidemiologist, Epidemiology. Before joining Clarivate, Ms. Davis was a research assistant at Tufts Medical Center, where she studied accelerated osteoarthritis and other rheumatic diseases. She also has experience working with zoonotic disease transmission in Ethiopia and excess mortality in Puerto Rico stemming from Hurricane Maria. She was part of the Health Emergencies team at the Pan American Health Organization, working on disaster preparedness and disease risk assessments. Ms. Davis holds a master’s degree in global health epidemiology and disease control from George Washington University and a B.S. in health sciences from James Madison University.