Amyotrophic Lateral Sclerosis Therapeutics Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, 2021-2031

The Global Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market is projected to expand from USD 692.03 Million in 2025 to USD 976.67 Million by 2031, reflecting a Compound Annual Growth Rate (CAGR) of 5.91%. This market includes a variety of pharmaceutical interventions and disease-modifying agents aiming to manage symptoms, slow neurodegeneration, and extend survival for patients with this progressive motor neuron disease. Key drivers of this growth include the rising global incidence of ALS and an aging population, both of which increase the need for effective neurological care. Additionally, significant investment in genetic research and the discovery of novel biomarkers are advancing the clinical development pipeline, establishing a strong foundation for long-term industry expansion beyond temporary market fluctuations.

According to the I AM ALS organization, collaborative advocacy efforts in 2024 successfully secured $258 million in federal funding to bolster ALS research and expanded access programs. Despite this strong financial support, the market faces considerable challenges due to the high costs of novel therapies, which often encounter strict reimbursement barriers that limit patient accessibility. This economic hurdle, coupled with high attrition rates for drug candidates in late-stage clinical trials, continues to complicate the commercial environment for new market entrants.

Market Drivers

Robust support from patient advocacy groups and non-profit funding acts as a vital catalyst for the market, significantly de-risking early-stage research and cultivating a stronger development ecosystem. By directing major capital into discovery biology and biomarker development, these organizations bridge the financial gaps frequently encountered by neurodegenerative therapeutic candidates. As noted in their 2024 Annual Report, Target ALS successfully completed a $250 million capital campaign specifically aimed at fueling innovative science and accelerating drug discovery milestones. This injection of non-dilutive funding enables biopharmaceutical companies to pursue high-risk, high-reward targets that might otherwise be neglected, ensuring a steady flow of potential treatments entering the clinical pipeline.

The rising adoption of disease-modifying and symptom-management therapies is simultaneously broadening the commercial landscape, fueled by the successful market penetration of new formulations and genetic medicines. As therapeutic availability increases, patient uptake of established treatments continues to support industry revenues, confirming the economic viability of ALS therapeutics. For instance, according to Mitsubishi Tanabe Pharma in November 2024, the company achieved a 6.0% year-on-year revenue increase to 232.6 billion yen for the six months ended September 2024, a growth trajectory attributed to the strong performance of its ALS treatment, Radicava ORS, in North America. Additionally, Biogen reported in October 2024 that its rare disease franchise, which includes the ALS therapy Qalsody, generated $495 million in third-quarter revenue, highlighting the substantial market value of targeted neurological interventions.

Market Challenges

The exorbitant cost of novel therapies and the associated strict reimbursement hurdles create a significant barrier to the commercial expansion of the Global Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market. Although the clinical pipeline is active, the market potential for emerging drugs is often limited by economic inaccessibility. When insurers and healthcare systems enforce restrictive coverage policies due to high drug pricing, patient access is constrained, which directly reduces revenue volume for pharmaceutical developers. This financial friction not only limits the immediate market uptake of approved products but also implies a precarious return on investment for future stakeholders, potentially slowing the long-term capital flow needed for sustained industry growth.

This economic difficulty is highlighted by recent data demonstrating the severe financial pressure on the patient population. According to a 2025 study cited by the ALS Association, medical expenses for Medicare beneficiaries in the first year following an ALS diagnosis exceeded $47,000, a figure more than three times higher than that of the average beneficiary. Such high expenditure levels force payers to implement rigorous reimbursement criteria, thereby extending the timeline for market penetration and complicating the commercial landscape for new therapeutic entrants.

Market Trends

The rise of Antisense Oligonucleotide (ASO) therapies marks a critical shift in the market, moving the treatment paradigm from general symptomatic management to precision medicine targeting specific genetic mutations such as FUS and SOD1. This trend is defined by increased licensing activity and high-value commercial partnerships aimed at expanding the pipeline of genetic medicines capable of silencing toxic protein production. For example, Otsuka Pharmaceutical Co., Ltd. announced in November 2024 that it secured exclusive global rights from Ionis to manufacture and market the investigational ASO ulefnersen for FUS-associated ALS, a deal structured with an upfront payment of $10 million. This strategic commitment confirms the growing industrial valuation of gene-silencing mechanisms as a primary modality for future therapeutic interventions.

Simultaneously, there is a strategic intensification of research focusing on neuroinflammation and oxidative stress pathways, looking beyond motor neuron protection to address the systemic immune dysregulation that drives disease progression. Biopharmaceutical developers are increasingly prioritizing immunomodulatory candidates, such as Regulatory T cell (Treg) enhancing therapies, to mitigate the inflammatory cascade associated with neuronal degeneration. According to a press release from Coya Therapeutics in October 2024, the biotechnology firm successfully closed a private placement of approximately $10 million specifically to fund the clinical and preclinical advancement of its lead biologic candidate, COYA 302, which targets these specific anti-inflammatory pathways. This continued capital inflow into immune-centric pipelines highlights the sector's pivot toward multi-mechanistic approaches intended to halt functional decline.

Key Players Profiled in the Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market

• Biogen
• Mitsubishi Tanabe Pharma
• Sanofi
• Roche
• Amylyx Pharmaceuticals
• Ionis Pharmaceuticals
• Wave Life Sciences
• Orphazyme
• Cleveland Clinic Spinouts
• Lundbeck

Report Scope

In this report, the Global Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market has been segmented into the following categories:

Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market, by Treatment:

• Medication
• Stem Cell Therapy
• Others

Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market, by Type:

• Sporadic ALS
• Familial ALS

Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market, by Distribution Channel:

• Hospital Pharmacies
• Retail Pharmacies
• Others

Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market, by Region:

• North America
• Europe
• Asia-Pacific
• South America
• Middle East & Africa

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market.

Available Customization

The analyst offers customization according to your specific needs. The following customization options are available for the report:

• Detailed analysis and profiling of additional market players (up to five).

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