Amyotrophic Lateral Sclerosis (ALS) - Market Insight, Epidemiology and Market Forecast - 2034

Key Highlights

• The market size of ALS in 2023, in the 7MM was ALS ~ USD 1.00 billion.
• The current standard of care that involves the prescription of riluzole has been effective in slowing the disease progression, thereby increasing the life span of patients.
• The most recent development in ALS was the addition of RELYVRIO for the treatment of ALS. On September 29, 2022, the US FDA approved RELYVRIO (sodium phenylbutyrate and taurursodiol) for the treatment of adults with ALS.
• After the setback with RELYVRIO, RADICAVA emerges as the leading contender in the current ALS market and is anticipated to achieve the highest revenue.
• After the approval of RADICAVA ORS, there was a notable increase in sales for RADICAVA, which had previously been declining for IV RADICAVA. The launch of RADICAVA ORS resulted in a high uptake of the drug in both the US and Japan
• Transposon Therapeutics' TPN-101 and Ionis Pharmaceuticals' Ulefnersen (ION363) are pioneering therapies tailored for specific mutations like C9ORF72 and FUS in ALS. These developments highlight a significant unmet need among patients with mutation-specific ALS, with current attention primarily focused on key mutations such as SOD1, C9ORF72, and FUS. However, there remains a notable gap in targeting mutations like TARDBP, OPTN, ANG, and others, presenting a compelling opportunity for future research and therapeutic advancements in the ALS landscape. Whereas in the majority of ALS patients, mutations still remain undiscovered.
• Currently, the drugs used for the treatment of ALS include EXSERVAN (Riluzole oral film), RELYVRIO, QALSODY, RADICAVA (edaravone injection), and RADICAVA ORS (edaravone oral suspension), NEUDEXTA, TIGLUTEK, RILUTEK along with other symptomatic treatment such as anti-epileptic drugs, opioids, NSAIDs, diuretics, SSRIs, antidepressants, etc. for the treatment of ALS.
• Expected launch of potential therapies, Ulefnersen (Ionis Pharmaceuticals), Ibudilast (MediciNova), Masitinib (AB Science), NurOwn (Brainstorm-Cell Therapeutics), CNM-Au8 (Clene Nanomedicine), and others may increase the market size in the coming years, assisted by an increase in the diagnosed prevalent population of ALS. It is expected that these therapies will help the market of ALS post-launch, in the 7MM during the forecast period (2024-2034).

This report delivers an in-depth understanding of ALS, historical and forecasted epidemiology as well as the ALS market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The ALS market report provides current treatment practices, emerging drugs, ALS market share of individual therapies, and current and forecasted ALS market size from 2020 to 2034, segmented by seven major markets. The report also covers current ALS treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

Amyotrophic Lateral Sclerosis (ALS) Understanding and Treatment Algorithm

Amyotrophic Lateral Sclerosis Overview

ALS, often referred to as Lou Gehrig's disease, is a progressive neurodegenerative disorder affecting the nerve cells in the brain and spinal cord, leading to muscle weakness, paralysis, and ultimately respiratory failure. While the exact cause remains unclear, a combination of genetic and environmental factors likely contributes to its onset. Diagnosis relies on neurological examination and various tests to rule out other conditions. Although there is currently no cure for ALS, treatment focuses on managing symptoms and improving quality of life. Research efforts continue to explore potential therapies and better understand the disease.

Amyotrophic Lateral Sclerosis Diagnosis

Diagnosing ALS involves a thorough neurological examination, medical history review, and various tests to exclude other possible conditions. Electromyography (EMG) and nerve conduction studies evaluate nerve and muscle function, while imaging scans like MRI rule out alternative causes of symptoms. Despite these efforts, ALS diagnosis remains challenging without a definitive test. Early and accurate diagnosis is crucial for initiating symptom management and support services. Biomarkers, like cerebrospinal fluid neurofilament levels, though not yet integrated into clinical practice, show promise for diagnosis and monitoring treatment effects. They could aid in patient stratification and research studies, particularly in cases where symptoms are ambiguous or overlap with other conditions.

Amyotrophic Lateral Sclerosis Treatment

Treatment for ALS aims to manage symptoms, improve quality of life, and slow disease progression. Medications such as riluzole and edaravone may be prescribed to help delay progression, although their effects are modest. Physical therapy, occupational therapy, and speech therapy can help maintain mobility, function, and communication abilities. Assistive devices such as wheelchairs, braces, and communication aids are often utilized to enhance independence. Additionally, respiratory support may be necessary as the disease progresses. While there is currently no cure for ALS, a multidisciplinary approach involving healthcare professionals, caregivers, and support networks is essential in providing comprehensive care and support to individuals living with the disease.

Amyotrophic Lateral Sclerosis Epidemiology

The ALS epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by the Total Prevalent Population of ALS, Diagnosed Prevalence of ALS, Type-specific Distribution of ALS, Gender-specific Distribution of ALS, Mutation-specific Distribution of ALS, Distribution Based on Site of Onset of ALS, and Age-specific Distribution of ALS in the 7MM market covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• Among the 7MM, the US accounted for the highest prevalent cases of ALS in 2023, with around 26,000 cases; these cases are expected to increase during the forecast period.
• Amongst EU4 and the UK, the diagnosed prevalent cases of ALS were highest in Germany, while the lowest number of cases was in Spain in 2023.
• The major sites of onset of ALS are the bulbar, and spinal, along with other uncertain regions. In 2023, the patients with the spinal as a site of onset accounted highest cases, i.e., 15,000, followed by the bulbar site of onset.
• According to the estimates, in Japan, it is observed that ALS was most prevalent in the 70-79 years age group, followed by 60-69 years, and 50-59 years.

Amyotrophic Lateral Sclerosis Drug Chapters

The drug chapter segment of the ALS report encloses a detailed analysis of the marketed and late-stage (Phase III) pipeline drug. The marketed drugs segment encloses drugs such as RADICAVA (Mitsubishi Tanabe Pharma Corporation), RELYVRIO (Amylyx Pharmaceuticals), TIGLUTIK (ITF Pharma), and others. Furthermore, the current key players for emerging drugs and their respective drug candidates include AB Science (Masitinib), Brainstorm Cell Therapeutics (NurOwn), and others. The drug chapter also helps understand the ALS clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, and the latest news and press releases.

Marketed Drugs

RADICAVA/RADICUT (edaravone injection) and RADICAVA ORS: Mitsubishi Tanabe Pharma Corporation

RADICAVA is a product of Mitsubishi Tanabe Pharma that contains the active substance edaravone. It is a novel neuroprotective agent that slows down ALS progression. Notably, RADICAVA is the second drug to be approved for the treatment of ALS after more than two decades from the first US FDA approval of riluzole. Edaravone is available as a clear, colorless liquid provided as a sterile injection solution supplied for IV infusion; the substituted 2-pyrazolin-5-one class has the chemical name 3-methyl-1-phenyl-2-pyrazolin-5-one. The mechanism by which RADICAVA exerts its therapeutic effect in patients with ALS is unknown; however, it has anti-oxidant properties (Cruz, 2018b). Even after getting approval, the company is still conducting various clinical trial studies to better understand the drug’s effects on a larger scale.

RADICAVA received marketing approval in May 2017 and an exclusivity end date is in May 2024. RADICAVA ORS received marketing approval in May 2022.

RELYVRIO: Amylyx Pharmaceuticals

RELYVRIO (AMX0035) is an investigational neuroprotective therapy being developed to minimize neuronal death and dysfunction. In ALS and other neurodegenerative disorders, the drug targets endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways and blocks stress to maintain a balance between them. AMX0035 is a fixed-dose co formulation of two active compounds, namely, sodium phenylbutyrate (PB) and taurursodiol (tauroursodeoxycholic acid [TUDCA].

Currently, the drug is being studied in a Phase III PHOENIX study for treating ALS. Recently, in March 2024, the company reported topline results from the Phoenix trial, where the study did not meet prespecified primary or secondary endpoints.

Within the next 8 weeks, Amylyx will continue to engage with regulatory authorities and the ALS community to share topline data. The company will share plans for RELYVRIO in ALS, which may include voluntarily withdrawing RELYVRIO from the market. At this time, RELYVRIO will continue to be available for people living with ALS. The company has voluntarily decided to pause promotion; however, related patient support services will remain in place.

Emerging Drugs

Masitinib: AB Science

Masitinib (AB1010) is an orally administered tyrosine kinase inhibitor. It modulates mast cells and macrophages’ activity - important cells for immunity - by targeting a limited number of kinases without inhibiting, at therapeutic doses, kinases associated with known toxicities. Masitinib distinguishes itself from other ALS developmental drugs by exerting neuroprotection in both central and peripheral nervous systems. Based on its unique mechanism of action, masitinib can be developed in many conditions in oncology, inflammatory diseases, and certain diseases of the central nervous system. AB Science completed a Phase II/III trial and has attained positive results, followed by a green signal from the US FDA on the IND application. However, the drug is under investigation for a Phase III trial in patients with ALS.

In January 2024, AB Science SA announced that the Committee for Medicinal Products for Human Use (CHMP) has proposed that AB Science submit a written response to the List of Outstanding Issues at D195 of the procedure instead of addressing these issues through the oral explanation. AB Science expects an opinion from the CHMP in the second quarter of 2024.

NurOwn (MSC-NTF cells): Brainstorm Cell Therapeutics

MSC-NTF cells (NurOwn) are autologous bone marrow-derived mesenchymal stem cells (MSC) induced in culture to secrete high levels of neurotrophic factors (NTFs) that support neuronal growth and survival. Thus, MSC-NTF cells combine MSC’s immunomodulatory therapeutic benefits with enhanced neurotrophic factor secretion.

In October 2023, the company announced a strategic realignment to enable accelerated development of NurOwn for the treatment of ALS. This realignment is designed to

1) Support the company’s plans to conduct a double-blind, placebo-controlled Phase IIIb US clinical trial for NurOwn in ALS with an open-label extension.

2) Continue to publish data from NurOwn’s Phase III clinical trial on biomarkers, long-term safety and survival, and the expanded access program, providing transparency around NurOwn data and progressing ALS drug development.

Drug Class Insight

Tyrosine kinase inhibitor

Tyrosine kinase inhibitors (TKIs) have been investigated in ALS research as potential therapeutic agents. TKIs target specific enzymes involved in cell signaling pathways, which may play a role in neurodegeneration seen in ALS. One TKI that has been studied in ALS is masitinib. Masitinib is a TKI that inhibits several tyrosine kinases, including c-Kit, platelet-derived growth factor receptor (PDGFR), and Lyn kinase. These kinases are involved in various cellular processes, including cell growth, survival, and inflammation, which are relevant to the pathophysiology of ALS. Clinical trials investigating masitinib in ALS have shown promising results in terms of slowing disease progression and improving survival rates in some patients. However, further research is needed to confirm its efficacy and safety in larger populations of ALS patients.

Amyotrophic Lateral Sclerosis Market Outlook

Currently, there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease. Scarcely any drugs have been approved by the FDA that can slow the course of the disease and improve the quality of life. Therefore, the management of ALS remains supportive and symptom-based. In recent years, research on new treatment strategies has increased, taking heed of gene therapy, cellular therapy, and neuroprotective agents. There are limited approved drugs that slow disease progression by prolonging autonomy and increasing survival rates (measured by the ALS functional rating scale (ALSFRS-R). Moreover, approved by the US FDA to treat ALS, including riluzole, NUEDEXTA, RADICAVA, TIGLUTIK, RELYVRIO, and QALSODY. Medications are also prescribed to help manage symptoms of ALS, including pain, muscle cramps, stiffness, excess saliva and phlegm, and the pseudobulbar effect (involuntary or uncontrollable episodes of crying and/or laughing, or other emotional displays). Drugs also are available to help individuals with pain, depression, sleep disturbances, and constipation.

For the treatment of ALS, Riluzole is recommended as first-line therapy in all three regions - Japan, the US, and Europe. Riluzole was first approved in the US by the FDA in 1995, and it was later approved in many other countries in the ensuing decades. It is a medication that appears to prolong the life of some people with ALS by at least a few months. Riluzole seems to do two things: block sodium and calcium channels and increase glutamate clearance. Currently, no other drug is globally approved for slowing the progression of ALS. RILUTEK, TIGLUTIK, and EXSERVAN are brand names for different formulations of riluzole, a medicine used in the treatment of ALS. RILUTEK is an oral tablet, TIGLUTIK is an oral suspension, and EXSERVAN is an oral film. NEUDEXTA is approved for the treatment of pseudobulbar effects in conditions such as multiple sclerosis and ALS.

Although the pipeline holds multiple promising therapies in various stages of development, the failure rates of clinical trials are quite high for ALS. Therapies like arimoclomol (Orphazyme), levosimendan (Orion Pharmaceuticals), ravulizumab (Ultomiris), and Zilucoplan (UCB Pharma), are some recent failures in the list of therapies for ALS, and more may follow in the future. Nevertheless, the current pipeline holds great potential as it contains disease-modifying agents, symptomatic treatments, and therapies targeting specific mutations, which could help in fulfilling the unmet treatment needs of ALS patients.

Key Findings

• The total market size in the US for ALS was estimated to be ~USD 920 million in 2023, which is expected to grow during the forecast period (2024-2034).
• RADICAVA emerges as the leading contender in the current ALS market and is anticipated to achieve the highest revenue, i.e., USD 923 million in the US by 2034.
• In 2034, among the emerging therapies, the highest revenue was generated by Masitinib in combination with riluzole, i.e., USD 37 million in Japan.

Amyotrophic Lateral Sclerosis Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024-2034. The landscape of ALS treatment has experienced a transformation with the uptake of novel drugs. These innovative therapies are redefining standards of care. Furthermore, the increased uptake of these transformative drugs is a testament to the unwavering dedication of Neurologists, professors of neurology, and director of the Neuromuscular Reference Center at the University Hospital, and professors in the Department of Translational Neuroscience. This momentous shift in treatment paradigms is a testament to the power of research, collaboration, and human resilience.

Amyotrophic Lateral Sclerosis Pipeline Development Activities

The report provides insights into therapeutic candidates in Phase III, Phase II, and Phase I/II. It also analyzes key players involved in developing targeted therapeutics. Companies like AB Science and Brainstorm Cell Therapeutics actively engage in late-stage research and development efforts for ALS. The pipeline of ALS possesses many potential drugs and there is a positive outlook for the therapeutics market, with expectations of growth during the forecast period (2024-2034).

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for ALS emerging therapy.

KOL- Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on the ALS evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Neurology specialists, Neuroscience specialists, and others.

The analysts connected with 30+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Centers such as the University of Nebraska Medical Center, Centers for Disease Control and Prevention, Department of Translational Neuroscience, etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or ALS market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst’s discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Market Access and Reimbursement

The treatment and management of ALS are expensive. The significant expense for ALS treatment comes from the patient’s pocket, with palliative care and management having a major share of the out-of-pocket expenditure. Expensive drugs like RADICAVA pose a burden for ALS patients as not everyone can get access to the drug due to its high cost.

The Patient Advocate Foundation’s (PAF) Copay Relief (CPR) Program also has an ALS fund that provides copay, co-insurance, and deductible. The fund has been developed in response to patients who have contacted PAF for help with their medication expenses and failed to receive it. The fund provides a maximum award level of USD 5,000 per year. The eligibility criteria for receiving the fund are that the patient must be insured, the insurance must cover the medication for ALS, and the patient must reside and receive treatment in the US. Currently, the fund is not accepting applications due to insufficient donations.

Medicare Part B coverage for RADICAVA

Under Medicare Part B, 80% of costs for a RADICAVA treatment are covered (after the annual deductible is paid), while the remaining 20% must be covered either by the patient or with supplemental coverage.

Scope of the Report

• The report covers a segment of key events, an executive summary, and a descriptive overview of ALS, explaining its causes, signs, symptoms, pathogenesis, and currently used therapies.
• Comprehensive insight into the epidemiology segments and forecasts, disease progression, and treatment guidelines has been provided.
• Additionally, an all-inclusive account of the emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the ALS market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive ALS.

Amyotrophic Lateral Sclerosis Report Insights

• Patient Population
• Therapeutic Approaches
• Amyotrophic Lateral Sclerosis Pipeline Analysis
• Amyotrophic Lateral Sclerosis Market Size and Trends
• Existing and Future Market Opportunity

Amyotrophic Lateral Sclerosis Report Key Strengths

• Eleven Years Forecast
• The 7MM Coverage
• Amyotrophic Lateral Sclerosis Epidemiology Segmentation
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

Amyotrophic Lateral Sclerosis Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Analyst Views)

FAQs

• What was the ALS market size, the market size by therapies, market share (%) distribution in 2020, and what would it look like by 2034? What are the contributing factors for this growth?
• What can be the future treatment paradigm for ALS?
• What are the disease risks, burdens, and unmet needs of ALS? What will be the growth opportunities across the 7MM concerning the patient population with ALS?
• What are the current options for the treatment of ALS? What are the current guidelines for treating ALS in the 7MM?
• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitations of existing therapies?
• What is the patient share in Amyotrophic Lateral Sclerosis?

Reasons to Buy

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving ALS.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis ranking of class-wise potential current and emerging therapies under the analyst view section to provide visibility around leading classes.
• Highlights of access and reimbursement policies of current therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.