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United States Action Figures Market Size and Forecast 2025–2033

How Innovation in Gene Therapy, Neuroprotection, and Global Healthcare Policy Is Reshaping ALS Care

By Aaina OberoiPublished 6 days ago 7 min read

Introduction

The global healthcare industry is witnessing a pivotal transformation in the treatment of rare and complex neurological disorders, and amyotrophic lateral sclerosis (ALS) stands at the forefront of this shift. Once viewed as a disease with limited therapeutic options, ALS is now attracting unprecedented research attention, investment, and innovation. According to Renub Research, the Amyotrophic Lateral Sclerosis Treatment Market is expected to reach US$ 1,093.14 million by 2033, growing from US$ 657.56 million in 2024, at a CAGR of 5.81% from 2025 to 2033.

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This growth is driven by rising ALS prevalence, expanding patient awareness, and a surge in research and development activities targeting neurodegenerative disorders. Breakthroughs in gene therapy, stem cell research, and novel drug delivery technologies are creating new possibilities for managing disease progression and improving patient quality of life. What was once a largely palliative landscape is now evolving into a domain of targeted therapies and precision medicine.

Amyotrophic Lateral Sclerosis Treatment Market Overview

Amyotrophic lateral sclerosis, commonly known as Lou Gehrig’s disease, is a progressive neurodegenerative condition that damages motor neurons in the brain and spinal cord. The disease leads to muscle weakness, paralysis, and ultimately respiratory failure. While ALS remains incurable, modern treatment strategies focus on symptom management, slowing disease progression, and extending survival.

Current treatment options include pharmaceuticals such as riluzole and edaravone, alongside emerging modalities like gene therapy, stem cell transplantation, and neuroprotective medications. Multidisciplinary care—integrating medication, physical therapy, respiratory support, and nutritional management—has become essential in maintaining patient function and quality of life.

The ALS treatment market is shaped by growing healthcare expenditure, heightened disease awareness, and rapid advances in neurological research. Strategic partnerships between pharmaceutical companies and academic institutions are accelerating drug development pipelines, particularly those targeting genetic mechanisms of ALS. Regulatory incentives such as orphan drug designation, fast-track approval pathways, and compassionate-use programs are further encouraging innovation and commercialization. Collectively, these factors are redefining ALS treatment from supportive care to disease-modifying interventions.

Growth Drivers for the Amyotrophic Lateral Sclerosis Treatment Market

Advancements in Gene and Stem Cell Therapy

One of the most significant drivers of market growth is the rapid development of gene and stem cell therapies. Unlike traditional treatments that primarily address symptoms, these advanced modalities aim to tackle the underlying causes of ALS. Gene-editing technologies such as CRISPR and antisense oligonucleotides (ASOs) are being used to target specific mutations linked to ALS, including SOD1 and C9orf72.

Stem cell therapies seek to replace damaged neurons and promote neuroregeneration, potentially slowing disease progression. Several experimental therapies have demonstrated promising outcomes in early-stage clinical trials, including improved motor function and extended survival. With major biotech firms and research institutions investing heavily in these approaches, gene and stem cell therapies are rapidly moving from experimental concepts to viable treatment options, positioning them as key growth engines for the market.

Growing Research Funding and Strategic Collaborations

Global investment in ALS research has expanded significantly as governments, non-profit organizations, and pharmaceutical companies prioritize neurodegenerative disease innovation. High-profile funding initiatives and awareness campaigns have catalyzed breakthroughs in understanding disease mechanisms, identifying biomarkers, and repurposing existing drugs.

Strategic collaborations—such as public–private partnerships, academic alliances, and multinational clinical trial networks—are accelerating therapy development while reducing costs and risks. Pharmaceutical firms are increasingly sharing data, pooling resources, and engaging in co-development agreements to shorten time-to-market. These collaborative ecosystems not only strengthen the therapeutic pipeline but also improve patient access to experimental treatments worldwide. As a result, sustained funding and partnerships continue to fuel long-term market growth.

Regulatory Support and Accelerated Drug Approvals

Regulatory agencies have played a decisive role in advancing the ALS treatment market. Organizations such as the U.S. FDA, European Medicines Agency (EMA), and Japan’s PMDA have introduced policies that fast-track therapies for rare diseases. Orphan drug designations, breakthrough therapy status, and priority review processes encourage pharmaceutical companies to invest in high-risk research by offering faster approvals and extended market exclusivity.

Recent approvals, including Relyvrio (Amylyx Pharmaceuticals) and Radicava ORS (Mitsubishi Tanabe Pharma), highlight the effectiveness of these regulatory frameworks. Compassionate-use programs further enable early patient access to experimental treatments. This proactive regulatory environment not only enhances innovation but also ensures that life-extending therapies reach patients more quickly, sustaining growth across the ALS therapeutics sector.

Challenges in the Amyotrophic Lateral Sclerosis Treatment Market

High Treatment Costs and Limited Accessibility

Despite technological advancements, ALS treatments remain expensive due to complex research processes, costly biologic manufacturing, and lengthy clinical development timelines. High prices limit accessibility in low- and middle-income countries where healthcare coverage and reimbursement structures are often inadequate. Even in developed markets, insurance restrictions and out-of-pocket costs create financial burdens for patients and families.

Beyond drug pricing, the need for lifelong symptom management, assistive devices, and multidisciplinary care further increases total treatment costs. Limited distribution networks for advanced therapies also restrict global availability. Addressing these barriers requires expanded patient assistance programs, pricing transparency, and government-supported reimbursement initiatives to ensure equitable access.

Clinical Complexity and Limited Therapeutic Efficacy

ALS remains one of the most challenging neurological disorders to treat. Its biological heterogeneity, uncertain pathophysiology, and rapid disease progression complicate drug development and clinical trial design. Many therapies that demonstrate promise in preclinical models fail in later stages due to insufficient efficacy or safety concerns.

Existing treatments such as riluzole and edaravone provide only modest benefits, primarily slowing disease progression rather than reversing symptoms. Additionally, recruiting adequate patient populations for clinical trials is difficult given ALS’s rarity, and the lack of validated biomarkers hinders early diagnosis and personalized treatment strategies. Overcoming these obstacles will require deeper molecular research, innovative trial methodologies, and the development of multi-targeted therapeutic approaches.

Regional Market Insights

United States Amyotrophic Lateral Sclerosis Treatment Market

The United States leads the global ALS treatment market, supported by robust research infrastructure, strong regulatory frameworks, and high healthcare spending. The FDA’s orphan drug and fast-track programs foster rapid innovation and commercialization. Companies such as Amylyx Pharmaceuticals, Biogen, and Mitsubishi Tanabe Pharma are at the forefront of developing gene therapies and neuroprotective agents.

Well-established clinical trial networks, advanced diagnostic technologies, and active patient advocacy groups further strengthen the market. Favorable reimbursement policies and sustained R&D investment ensure the U.S. remains the largest contributor to global ALS therapeutic advancement.

Germany Amyotrophic Lateral Sclerosis Treatment Market

Germany’s ALS market benefits from a strong healthcare system, active participation in European research initiatives, and government-funded neurodegenerative disease programs. German biopharmaceutical companies and academic institutions are engaged in clinical research focused on biomarkers and innovative therapies.

Universal healthcare coverage supports access to approved drugs and assistive technologies, while collaborative projects under EU frameworks such as Horizon Europe enhance research capabilities. With its emphasis on scientific rigor and patient-centered care, Germany plays a vital role in shaping ALS treatment across Europe.

China Amyotrophic Lateral Sclerosis Treatment Market

China’s ALS treatment market is expanding rapidly due to increasing disease awareness, government support for rare disease research, and improved healthcare access. National initiatives have strengthened patient registries and diagnostic capabilities. Domestic pharmaceutical firms are developing generic and biosimilar versions of existing drugs, while partnerships with global companies facilitate technology transfer.

Although affordability and access to advanced biologics remain challenges, China’s growing R&D capabilities and policy support position it as a major emerging contributor to ALS therapy innovation in the Asia-Pacific region.

Saudi Arabia Amyotrophic Lateral Sclerosis Treatment Market

Saudi Arabia’s ALS treatment landscape is evolving in line with the nation’s Vision 2030 healthcare transformation strategy. Increased investments in rare disease research, modern hospital infrastructure, and specialized neurology centers are improving diagnosis and treatment capabilities.

The government’s commitment to adopting innovative medicines and fostering international clinical collaborations is expanding therapeutic access. While high drug costs and limited local manufacturing remain obstacles, ongoing healthcare digitalization and global partnerships are expected to strengthen Saudi Arabia’s position in the Middle Eastern ALS market.

Recent Developments in the ALS Treatment Market

In June 2024, the U.S. FDA introduced a new funding opportunity under its Rare Neurodegenerative Disease Grant Program to support clinical research for ALS and related disorders. The initiative aims to accelerate therapy development and promote collaboration between academic and industry researchers.

That same month, Mitsubishi Tanabe Pharma Corporation transferred its European argatroban business to Ethypharm, enabling the company to focus on expanding the availability of Radicava® (edaravone) across Europe.

In February 2024, Biogen’s QALSODY™, the first therapy designed specifically for SOD1-ALS, received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use, marking a major milestone toward European approval.

Earlier, in April 2023, the FDA granted accelerated approval to QALSODY™ (tofersen), making it the third targeted therapy approved for ALS and a significant breakthrough in personalized neurology.

Market Segmentation

By ALS Type:

Sporadic ALS

Familial ALS

By Drug Type:

Marketed Drugs

Emerging Drugs

By Diagnosis Type:

Electromyogram

MRI

Blood and Urine Tests

Spinal Tap

Muscle Biopsy

By Treatment:

Medication

Stem Cell Therapy

Others

By Distribution Channel:

Hospital Pharmacies

Retail Pharmacies

Others

By Region:

North America, Europe, Asia Pacific, Latin America, and Middle East & Africa, with country-level analysis across 25 viewpoints including the United States, Germany, China, Saudi Arabia, India, Japan, Brazil, and the UAE.

Competitive Landscape

Key players in the ALS treatment market include:

AB Science, Corestem Inc., Cytokinetics Inc., Revalesio Corporation, ITF Pharma (Italfarmaco S.p.A.), Ascend Pharmaceuticals LLC, BrainStorm Cell Therapeutics Inc., Mitsubishi Chemical Group Corporation, Eledon Pharmaceuticals Inc., and Ionis Pharmaceuticals Inc.

Each company is analyzed across five dimensions: company overview, key leadership, recent developments and strategies, SWOT analysis, and sales performance. These firms are driving innovation through gene therapy platforms, neuroprotective drugs, and regenerative medicine approaches.

Final Thoughts

The Amyotrophic Lateral Sclerosis Treatment Market is entering a transformative era. With Renub Research projecting growth from US$ 657.56 million in 2024 to US$ 1,093.14 million by 2033, the market reflects both rising disease prevalence and a wave of therapeutic innovation.

Advances in gene therapy, stem cell research, and regulatory support are reshaping ALS care from symptom management to targeted, disease-modifying strategies. While challenges related to cost, accessibility, and clinical complexity persist, sustained research funding, global collaboration, and patient advocacy are steadily overcoming these barriers.

As healthcare systems increasingly prioritize rare and neurodegenerative diseases, the ALS treatment market stands poised to deliver not only commercial growth but also renewed hope for patients and families worldwide.

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About the Creator

Aaina Oberoi

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