Amyotrophic Lateral Sclerosis Therapeutics Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Treatment (Medication [Riluzole, Edaravone, Others], Stem Cell Therapy, Others), By Type (Sporadic ALS, Familial ALS), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Others), By Region and Competition, 2021-2031F

Forecast Period	2027-2031
Market Size (2025)	USD 692.03 Million
CAGR (2026-2031)	5.91%
Fastest Growing Segment	Stem Cell Therapy
Largest Market	North America
Market Size (2031)	USD 976.67 Million

Market Overview

The Global Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market will grow from USD 692.03 Million in 2025 to USD 976.67 Million by 2031 at a 5.91% CAGR. Amyotrophic Lateral Sclerosis (ALS) therapeutics encompass a range of pharmaceutical interventions and disease-modifying agents designed to manage symptoms, decelerate neurodegeneration, and prolong survival for patients suffering from this progressive motor neuron disease. The market is fundamentally driven by the rising global incidence of ALS and an expanding geriatric demographic, which naturally heightens the demand for effective neurological care. Furthermore, substantial capital infusion into genetic research and the identification of novel biomarkers are accelerating the clinical development pipeline, providing a structural foundation for long-term industry expansion distinct from transient market patterns.

According to the I AM ALS organization, in 2024, collaborative advocacy efforts successfully secured $258 million in federal funding to support ALS research and expanded access programs. Despite this robust financial backing, the market faces a significant challenge regarding the exorbitant costs associated with novel therapies, which often encounter stringent reimbursement hurdles and limit patient accessibility. This economic barrier, combined with the high attrition rate of drug candidates during late-stage clinical trials, continues to complicate the commercialization landscape for emerging market entrants.

Key Market Drivers

Strong support from patient advocacy groups and non-profit funding is functioning as a critical catalyst for the market, significantly de-risking early-stage research and fostering a more robust development ecosystem. By channeling substantial capital directly into discovery biology and biomarker development, these organizations are bridging the financial gaps often faced by neurodegenerative therapeutic candidates. According to Target ALS, in their 2024 Annual Report, the organization successfully completed a $250 million capital campaign specifically designed to fuel innovative science and accelerate drug discovery milestones. This influx of non-dilutive funding allows biopharmaceutical companies to pursue high-risk, high-reward targets that might otherwise be deprioritized, ensuring a continuous stream of potential treatments entering the clinical pipeline.

The growing adoption of disease-modifying and symptom-management therapies is simultaneously expanding the commercial landscape, driven by the successful market penetration of novel formulations and genetic medicines. As therapeutic availability improves, patient uptake of established treatments continues to bolster industry revenues, validating the economic viability of ALS therapeutics. According to Mitsubishi Tanabe Pharma, 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 explicitly attributed to the strong performance of its ALS treatment, Radicava ORS, in North America. Furthermore, according to Biogen, October 2024, the company's rare disease franchise, which comprises the ALS therapy Qalsody, generated $495 million in third-quarter revenue, underscoring the substantial market value of targeted neurological interventions.

Download Free Sample Report

Key Market Challenges

The high cost of novel therapies and the associated stringent reimbursement hurdles present a substantial 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 frequently curtailed by economic inaccessibility. When insurers and healthcare systems impose restrictive coverage policies due to elevated drug pricing, patient access is restricted, which directly reduces the revenue volume for pharmaceutical developers. This financial friction not only limits the immediate market uptake of approved products but also signals a precarious return on investment for future stakeholders, potentially dampening the long-term capital flow required for sustained industry growth.

This economic challenge is underscored by recent data illustrating the severe financial pressure on the patient population. According to the ALS Association, in 2025, a study revealed that 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 compel payers to enforce rigorous reimbursement criteria, thereby extending the timeline for market penetration and complicating the commercial landscape for new therapeutic entrants.

Key Market Trends

The acceleration of Antisense Oligonucleotide (ASO) therapies represents a pivotal shift in the market, transitioning the treatment paradigm from broad symptomatic management to precision medicine targeting specific genetic mutations like FUS and SOD1. This trend is characterized by increased licensing activity and high-value commercial partnerships designed to expand the pipeline of genetic medicines capable of silencing toxic protein production. According to Otsuka Pharmaceutical Co., Ltd., November 2024, in the 'Otsuka Obtains Exclusive Global Licensing Rights from Ionis to Ulefnersen' news release, the company secured exclusive global rights 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 validates the growing industrial valuation of gene-silencing mechanisms as a primary modality for future therapeutic interventions.

Concurrently, there is a strategic intensification of research focusing on neuroinflammation and oxidative stress pathways, moving 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 Coya Therapeutics, October 2024, in the 'Coya Therapeutics Announces Closing of $10.0 Million Private Placement' press release, 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.

Segmental Insights

The Stem Cell Therapy segment is recognized as the fastest-growing category in the Global Amyotrophic Lateral Sclerosis Therapeutics Market due to the urgent demand for regenerative treatment options. While standard pharmacological interventions primarily offer symptomatic relief, stem cell research focuses on repairing damaged motor neurons and potentially slowing disease progression. This expansion is further supported by favorable regulatory environments, where agencies such as the U.S. Food and Drug Administration have granted special designations to accelerate clinical development. Consequently, increased investment in these biological approaches is reshaping the competitive landscape of ALS treatment.

Regional Insights

North America leads the Global Amyotrophic Lateral Sclerosis Therapeutics Market due to established healthcare infrastructure and increasing disease awareness across the region. This dominance is supported by the active presence of major pharmaceutical developers focusing on clinical research and new drug launches. Furthermore, the U.S. Food and Drug Administration plays a critical role by granting approvals and orphan drug designations that incentivize therapeutic advancements. Comprehensive reimbursement policies and the strong influence of patient advocacy groups also improve access to treatment, thereby sustaining the market supremacy of North America in this sector.

Recent Developments

• In August 2024, NeuroSense Therapeutics announced positive twelve-month biomarker data from its Phase 2b PARADIGM clinical trial evaluating PrimeC in patients with amyotrophic lateral sclerosis (ALS). The study results revealed a significant decrease in ferritin levels and a corresponding increase in transferrin levels, indicating effective regulation of iron metabolism, a biological pathway often dysregulated in ALS pathology. These biomarker improvements aligned with previously reported clinical data that showed a slowing of disease progression and improved survival rates, further supporting the therapeutic potential of the drug as the company prepares for late-stage clinical development.
• In June 2024, QurAlis Corporation entered into an exclusive global license agreement with Eli Lilly and Company to develop and commercialize QRL-204, a potential first-in-class precision therapy for amyotrophic lateral sclerosis (ALS). The collaboration focuses on a splice-switching antisense oligonucleotide designed to restore UNC13A function, which is often disrupted in ALS patients. Under the terms of the agreement, QurAlis granted global rights to the pharmaceutical partner and is eligible to receive milestone payments totaling up to $577 million, in addition to tiered royalties on net sales, effectively advancing the development of this targeted therapeutic candidate.
• In May 2024, Biogen received marketing authorization from the European Commission for Qalsody (tofersen), a targeted therapy for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene. This regulatory achievement established Qalsody as the first treatment approved in the European Union specifically designed to address a genetic cause of the disease. The authorization was granted under exceptional circumstances, recognizing the therapy's positive benefit-risk profile despite the challenges in gathering comprehensive data for such a rare condition, and the drug maintained its orphan medicinal product designation.
• In April 2024, Amylyx Pharmaceuticals announced the voluntary discontinuation of the marketing authorizations for its amyotrophic lateral sclerosis (ALS) therapy, Relyvrio (marketed as Albrioza in Canada), in the United States and Canadian markets. This significant corporate decision followed the release of topline results from the Phase 3 PHOENIX clinical trial, which failed to meet its primary efficacy endpoint of slowing disease progression compared to a placebo. Consequently, the company initiated a process to withdraw the product while establishing a free drug program to support patients currently undergoing treatment who wished to continue therapy in consultation with their physicians.

Key Market Players

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

By Treatment	By Type	By Distribution Channel	By Region
Medication [Riluzole Edaravone Others] Stem Cell Therapy Others	Sporadic ALS Familial ALS	Hospital Pharmacies Retail Pharmacies Others	North America Europe Asia Pacific South America Middle East & Africa

Report Scope:

In this report, the Global Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

• Amyotrophic Lateral Sclerosis (ALS) Therapeutics Market, By Treatment:

• Medication [Riluzole
• Edaravone
• Others]
• 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
• United States
• Canada
• Mexico
• Europe
• France
• United Kingdom
• Italy
• Germany
• Spain
• Asia Pacific
• China
• India
• Japan
• Australia
• South Korea
• South America
• Brazil
• Argentina
• Colombia
• Middle East & Africa
• South Africa
• Saudi Arabia
• UAE