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. Two treatments are approved for ALS in the markets under study—Mitsubishi Tanabe Pharma’s Radicava (edaravone) and riluzole (Sanofi’s Rilutek, other brands, generics)—but 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? What are the key advantages and disadvantages of these products?
How will the size of the drug-treated ALS population change through 2031?
What are the key unmet needs in the treatment of ALS?
About which emerging therapies are ALS experts most intrigued or optimistic? How would new therapies influence the management of ALS patients?
Which emerging therapies for ALS are likely to launch by 2031? 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 2031
Niche & Rare Disease Landscape & Forecast introduces a new 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.
Amyotrophic lateral sclerosis key findings - November 2022
Commercial outlook
Key findings
Regional sales of key therapies to treat amyotrophic lateral sclerosis: 2021 and 2031
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
Forecast
Sales of key therapies in amyotrophic lateral sclerosis
Etiology and pathophysiology
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
Epidemiology
Introduction
Key findings
Epidemiology populations
Disease definition
Methods
Sources used for diagnosed prevalent cases of amyotrophic lateral sclerosis
Diagnosed and drug-treated prevalent cases of amyotrophic lateral sclerosis: 2021-2031
Disease definition
Methods
Sources used for subtypes of amyotrophic lateral sclerosis
Diagnosed prevalent cases of amyotrophic lateral sclerosis by subtype: 2021-2031
Number of drug-treated prevalent cases of amyotrophic lateral sclerosis: 2021-2031
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
Select clinical trial results for edaravone in the treatment of amyotrophic lateral sclerosis
Key ongoing clinical trials of edaravone in the treatment of amyotrophic lateral sclerosis
Expert insight: edaravone
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
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
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
Key ongoing clinical trials investigating MSC-NTF cells for the treatment of amyotrophic lateral sclerosis
Expert insight: MSC-NTF cells
Expectations for launch and sales opportunity for MSC-NTF cells in amyotrophic lateral sclerosis
Tofersen profile
Key clinical trials investigating tofersen for the treatment of amyotrophic lateral sclerosis
Expert insight: tofersen
Expectations for launch and sales opportunity for tofersen in amyotrophic lateral sclerosis
ION363
Key ongoing clinical trials investigating ION363 for the treatment of amyotrophic lateral sclerosis
Expectations for launch and sales opportunity for ION363 in amyotrophic lateral sclerosis
Select clinical trial results for masitinib in the treatment of amyotrophic lateral sclerosis
Key ongoing clinical trials investigating masitinib for the treatment of amyotrophic lateral sclerosis
Expectations for launch and sales opportunity for masitinib in amyotrophic lateral sclerosis
AMX0035 profile
Key clinical trials investigating AMX0035 for the treatment of amyotrophic lateral sclerosis
Expert insight: AMX0035
Expectations for launch and sales opportunity for AMX0035 in amyotrophic lateral sclerosis
Reldesemtiv profile
Key ongoing clinical trials of reldesemtiv in the treatment of amyotrophic lateral sclerosis
Expectations for launch and sales opportunity for reldesemtiv 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 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
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 investigating FNP122 for the treatment of amyotrophic lateral sclerosis
Expectations for launch and sales opportunity for FNP122 in amyotrophic lateral sclerosis
TUDCA profile
Key ongoing clinical trials investigating TUDCA for the treatment of amyotrophic lateral sclerosis
Expectations for launch and sales opportunity for TUDCA 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
Access and reimbursement overview
Region-specific reimbursement practices
General reimbursement environment: United States
General reimbursement environment: EU5
Appendix
Key abbreviations related to amyotrophic lateral sclerosis
Amyotrophic lateral sclerosis bibliography
Muthana Sweis
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.