근위축성측색경화증 치료제 시장 : 치료법, 유형, 치료 스테이지, 투여 경로, 유통 채널별 - 세계 예측(2025-2032년)

The Amyotrophic Lateral Sclerosis Treatment Market is projected to grow by USD 1,188.64 million at a CAGR of 7.07% by 2032.

Concise contextualization of the ALS clinical and commercial landscape to inform strategic prioritization and evidence-driven program decisions

Amyotrophic lateral sclerosis represents one of the most complex neurodegenerative diseases confronting clinicians, researchers, and commercial stakeholders. The clinical heterogeneity of the condition, spanning familial and sporadic etiologies, challenges conventional development paradigms and elevates the importance of precision in trial design, biomarker selection, and therapeutic targeting. As a result, decision-makers must synthesize insights across molecular therapies, symptomatic agents, and supportive care to frame coherent product and portfolio strategies.

Recent advances in antisense modalities, targeted antioxidants, and repurposed symptomatic combinations have shifted clinical conversations from purely supportive management toward disease-modifying intent. Alongside these clinical developments, shifting regulatory expectations and heightened scrutiny on evidence of functional benefit have prompted sponsors to calibrate endpoints and invest in longitudinal natural history datasets. Consequently, commercial leaders must balance near-term access and reimbursement pathways for symptomatic products with longer-term investment in high-cost, high-complexity modalities.

This executive summary distills the most consequential trends, segmentation insights, regional dynamics, and actionable recommendations for organizations operating across the ALS treatment continuum. The goal is to equip executives with a concise, evidence-driven narrative that supports prioritization, risk mitigation, and the design of commercially viable development programs in a rapidly evolving therapeutic landscape.

How converging therapeutic innovation, evidence evolution, and care delivery transformation are redefining development and commercialization for ALS treatments

The ALS treatment landscape is undergoing transformative shifts driven by converging scientific advances, regulatory nuance, and evolving care pathways. Gene-targeted approaches and antisense oligonucleotides have matured from experimental tools into clinical candidates with clear translational potential, prompting biopharma organizations to re-evaluate platform investments and collaboration strategies. At the same time, stem cell therapy research has progressed from preclinical models to early human studies that emphasize safety, route of administration, and patient selection as critical determinants of translational success.

Parallel to therapeutic innovation, real-world evidence generation and digital biomarkers are reshaping how clinical benefit is demonstrated. Remote monitoring technologies and objective functional metrics are increasingly incorporated into trial protocols to capture granular changes in disease trajectory, enabling more sensitive detection of therapeutic signals. Payers and health technology assessment bodies are responding by seeking evidence that links surrogate improvements to meaningful patient-centric outcomes, thus influencing trial designs and post-approval evidence commitments.

Commercially, the distribution and administration complexities of advanced therapies are redefining channel strategies. Intrathecal and intravenous modalities necessitate specialized infusion centers and care coordination, which elevates the role of hospital pharmacies and integrated health systems in enabling patient access. Consequently, manufacturers are establishing center-of-excellence programs and partnering with clinical networks to streamline delivery, support adherence, and reduce logistical friction. Taken together, these shifts underscore an industry pivot toward integrated development and commercialization approaches that account for the entire patient journey from diagnosis to long-term follow-up.

Assessing the supply chain, manufacturing, and access implications of evolving United States tariff policies for neurological therapeutic value chains in 2025

Policy shifts in trade and tariffs have implications for complex therapeutic supply chains that extend beyond simple cost increments, and the unfolding tariff landscape in the United States for 2025 introduces material considerations for manufacturing strategy, procurement, and pricing resilience. Many active pharmaceutical ingredients and specialty excipients used in neurological therapeutics are sourced globally, and increased import duties can amplify input cost volatility. In response, organizations are reassessing supplier diversification, nearshoring options, and multi-sourcing agreements to preserve continuity of supply and mitigate exposure to trade policy shocks.

Beyond raw material costs, tariffs can influence the location of critical production steps such as fill-finish operations, cold chain logistics, and specialized biologics manufacturing. These operational shifts can extend lead times and increase the capital intensity of ensuring regulatory-compliant manufacturing footprints. Consequently, sponsors may accelerate investments in domestic manufacturing capabilities or long-term contractual commitments with contract manufacturing organizations that can absorb policy-driven margin pressure while guaranteeing capacity.

Importantly, the cumulative impact of tariffs also cascades into pricing and access strategies. Payers and procurement entities are likely to scrutinize price trajectories, particularly for high-cost, specialty therapies. Manufacturers may need to adopt more sophisticated value dossiers and risk-sharing arrangements to maintain formulary access under tightening budget constraints. In short, the tariffs scenario for 2025 reinforces the need for proactive supply chain risk management, flexible sourcing, and integrated commercial planning that anticipates regulatory, operational, and payer responses to increased cost pressures.

A segmentation-focused synthesis that links treatment modalities, patient subgroups, administration routes, and distribution channels to clinical and commercial implications

Understanding demand and clinical adoption requires a segmentation-aware perspective that aligns therapeutic modalities with patient subgroups, stages of disease, routes of administration, and distribution pathways. Based on treatment, the landscape divides into medication and therapies, where medication modalities include dextromethorphan/quinidine, edaravone, riluzole, and tofersen, while therapy approaches span gene therapy and stem cell therapy, each presenting distinct clinical profiles, evidence expectations, and delivery requirements. This treatment-based segmentation highlights that small-molecule symptomatic agents and neuroprotective compounds tend to follow established outpatient distribution channels, whereas gene-based and cellular therapies drive requirements for specialized administration settings and long-term safety surveillance.

Based on type, the dichotomy between familial ALS and sporadic ALS matters considerable for both development strategy and trial enrollment. Familial forms often support targeted molecular approaches with genetically defined mechanisms, enabling more precise patient selection and potentially more rapid proof-of-concept studies. Sporadic ALS, by contrast, demands broader population strategies and biomarker-driven stratification to address etiologic heterogeneity and demonstrate generalizability.

Based on treatment stage, early stage treatment, mid stage treatment, and advanced stage treatment each call for tailored endpoints, supportive care integrations, and risk-benefit considerations. Early stage interventions emphasize slowing progression and preserving function, necessitating sensitive outcome measures and proactive patient identification. Mid stage management focuses on symptom modulation and maintaining quality of life, while advanced stage treatments prioritize palliative integration, respiratory support coordination, and caregiver enablement.

Based on route of administration, intrathecal, intravenous, and oral delivery options shape clinical workflow, monitoring requirements, and adherence considerations. Intrathecal and intravenous routes often require hospital-based administration and specialized training, whereas oral therapies facilitate decentralized care but may encounter challenges with pharmacokinetics and central nervous system penetration. Based on distribution channel, hospital pharmacies and retail pharmacies play complementary roles where hospital pharmacies are central to specialty infusion and intrathecal administration while retail pharmacies support outpatient medication continuity and symptomatic management. Synthesizing these segmentation lenses enables stakeholders to map therapeutic attributes to operational capabilities, payer expectations, and patient access pathways.

Comparative regional dynamics outlining how regulatory, clinical research, and access frameworks across major geographies influence ALS development and commercialization

Regional dynamics materially shape regulatory pathways, clinical trial feasibility, and access frameworks for ALS treatments. In the Americas, regulatory authorities and payer systems exert strong influence on dossier design and reimbursement negotiations, while well-established research networks support rapid patient recruitment for trials that test novel mechanisms. The Americas also host concentrated centers of excellence that enable delivery of specialized intrathecal and intravenous therapies, and integrated health systems play a pivotal role in coordinating multidisciplinary care.

In Europe, Middle East & Africa, stakeholders navigate a mosaic of regulatory frameworks and payer models that require tailored evidence strategies and localized market access planning. Centralized regulatory procedures in some jurisdictions can expedite approvals for certain therapeutics, whereas heterogeneous reimbursement pathways across countries demand flexible pricing and risk-sharing models. Additionally, regional clinical networks and academic consortia are increasingly collaborating on multi-country trials that leverage shared natural history datasets and harmonized endpoints.

Asia-Pacific presents diverse development opportunities and operational considerations; several countries are investing in biotech infrastructure and clinical capacity, which opens pathways for local manufacturing partnerships and regional clinical studies. However, differences in diagnostic practices, genetic epidemiology, and healthcare financing require sponsors to adapt trial protocols and value communication strategies. Across all regions, cross-border collaboration, data sharing, and alignment on real-world evidence standards are becoming essential to support global development programs and ensure equitable patient access to emerging therapeutics.

Competitive and collaborative dynamics that reveal how portfolio strategies, manufacturing partnerships, and patient engagement investments shape ALS therapeutic success

The competitive environment in ALS therapeutics is characterized by a mix of established neurological portfolios, specialist biotech innovators, and contract organizations enabling advanced modality development. Leading pharmaceutical and biotech entities are advancing platform approaches that target molecular drivers of disease, while smaller specialists focus on niche indications, biomarker-driven cohorts, or novel delivery solutions. This diversity generates opportunities for strategic alliances, licensing arrangements, and joint development agreements that align scientific expertise with commercial scale.

Partnerships between developers and specialized contract manufacturers or service providers are increasingly central to managing the complexity of biologic and cellular product manufacturing. These collaborations help translate early-stage proof-of-concept into scalable clinical production while addressing regulatory expectations for quality and traceability. Additionally, payer-facing capabilities and global market access expertise are differentiators for companies seeking broad adoption, prompting acquisitions and collaborations that integrate clinical development with health economics and outcomes research capabilities.

Competition also extends to patient engagement and diagnostic ecosystems; organizations investing in patient registries, remote monitoring platforms, and early diagnostic initiatives can accelerate trial enrollment and strengthen real-world evidence generation. Consequently, companies that combine robust R&D pipelines with deep commercial integration, manufacturing resilience, and patient-centric evidence strategies are best positioned to navigate the complexity of the ALS treatment landscape.

Practical, high-impact strategic measures that align development, supply chain, and access initiatives to de-risk programs and accelerate patient access

Industry leaders must align strategic investments with operational realities to capture therapeutic and commercial opportunities while managing risk. First, prioritize integration between clinical development and payer evidence teams so that trial endpoints, natural history comparators, and health economic models converge early in program design. This alignment reduces downstream access friction and supports robust value communication at launch.

Second, diversify sourcing and manufacturing arrangements to enhance supply chain resilience in the face of trade policy shifts and input scarcity. Near-term tactics include multi-sourcing key intermediates, securing capacity in geographically distributed facilities, and investing in flexible fill-finish capabilities that accommodate modality-specific requirements. Third, invest in partnerships that expand diagnostic reach and patient registry participation to shorten enrollment timelines and improve the representativeness of trial populations, particularly for familial and genetically defined cohorts.

Fourth, design commercialization plans that reflect route-of-administration realities by ensuring hospital systems and specialty pharmacies are engaged early to build treatment pathways and center-of-excellence networks. Fifth, adopt adaptive evidence-generation strategies that combine randomized data with well-structured real-world evidence and digital biomarker endpoints, enabling iterative payer dialogue and post-approval data commitments. Collectively, these actions create a resilient playbook for translating scientific breakthroughs into accessible treatments while managing regulatory, operational, and commercial risk.

A mixed-methods research framework combining primary expert engagement and secondary evidence synthesis with rigorous triangulation to validate therapeutic and operational insights

The research approach underpinning this analysis combined a structured review of peer-reviewed literature, regulatory guidance, and public clinical trial registries with targeted expert interviews across clinical, regulatory, supply chain, and commercial domains. Primary engagement with neurologists, clinical trial investigators, health economists, and supply chain executives provided qualitative validation of emerging modality trends, administration challenges, and payer expectations. Secondary research synthesized scientific publications, regulatory briefing documents, and published treatment guidelines to contextualize therapeutic mechanisms and clinical endpoints.

Data triangulation was used to reconcile differing perspectives and to identify consistent themes regarding evidence needs, delivery constraints, and regional differences. Where possible, methodological rigor was preserved by cross-checking interview insights against documented clinical trial designs and public regulatory decisions. The analysis intentionally emphasized mechanistic and operational factors over proprietary market estimates to ensure the findings remain broadly applicable and decision-focused.

Limitations of the methodology include variability in public disclosure across early-stage programs, heterogeneity in regional regulatory processes, and evolving trade policy contexts that may change after the analysis cut-off. To mitigate these limitations, the study prioritized themes supported by multiple independent sources and sought corroboration from clinical and commercial experts with direct ALS experience.

Summative perspective on the intersecting scientific, operational, and payer considerations that will determine near-term translation of ALS treatment advances into patient access

The ALS therapeutic landscape is at an inflection point where molecular precision, novel delivery modalities, and evolving evidence expectations converge to reshape development and access strategies. Advances in gene-targeted and cellular approaches offer real potential for altering disease trajectory for selected patient cohorts, while improvements in symptomatic agents and monitoring technologies enhance quality-of-life management across disease stages. However, realizing these opportunities requires disciplined alignment of clinical evidence, manufacturing capacity, and payer-focused value demonstration.

Operational risks such as supply chain fragility, tariff-driven cost pressures, and the logistical demands of intrathecal and intravenous administration necessitate proactive mitigation and strategic investment. In parallel, segmentation-aware development that differentiates between familial and sporadic forms, maps stage-specific therapeutic objectives, and matches routes of administration to distribution capabilities will be critical to optimizing trial design and commercial rollout. Ultimately, organizations that couple scientific innovation with purposeful engagement across clinical networks, payers, and delivery partners will be best positioned to translate therapeutic advances into sustainable patient access and long-term impact.

Table of Contents

1. Preface

• 1.1. Objectives of the Study
• 1.2. Market Segmentation & Coverage
• 1.3. Years Considered for the Study
• 1.4. Currency & Pricing
• 1.5. Language
• 1.6. Stakeholders

2. Research Methodology

3. Executive Summary

4. Market Overview

5. Market Insights

• 5.1. Emerging antisense oligonucleotide therapies targeting SOD1 and C9orf72 gene mutations for personalized ALS treatment
• 5.2. Integration of digital biomarkers from wearable devices to monitor ALS patient mobility and disease progression in real time
• 5.3. Regulatory approvals and market launch of Relyvrio (sodium phenylbutyrate and taurursodiol) transforming global ALS treatment options
• 5.4. Advances in stem cell-derived motor neuron transplantation techniques showing promise in slowing ALS progression
• 5.5. Development of non-invasive neuromodulation therapies including transcranial magnetic stimulation to preserve motor function in ALS patients
• 5.6. Exploration of mitochondrial function modulators and metabolic pathway drugs to address neuronal energy deficits in ALS

6. Cumulative Impact of United States Tariffs 2025

7. Cumulative Impact of Artificial Intelligence 2025

8. Amyotrophic Lateral Sclerosis Treatment Market, by Treatment

• 8.1. Medication
  • 8.1.1. Dextromethorphan/Quinidine
  • 8.1.2. Edaravone
  • 8.1.3. Riluzole
  • 8.1.4. Tofersen
• 8.2. Therapies
  • 8.2.1. Gene Therapy
  • 8.2.2. Stem Cell Therapy

9. Amyotrophic Lateral Sclerosis Treatment Market, by Type

• 9.1. Familial ALS
• 9.2. Sporadic ALS

10. Amyotrophic Lateral Sclerosis Treatment Market, by Treatment Stage

• 10.1. Advanced Stage Treatment
• 10.2. Early Stage Treatment
• 10.3. Mid Stage Treatment

11. Amyotrophic Lateral Sclerosis Treatment Market, by Route of Administration

• 11.1. Intrathecal
• 11.2. Intravenous
• 11.3. Oral

12. Amyotrophic Lateral Sclerosis Treatment Market, by Distribution Channel

• 12.1. Hospital Pharmacies
• 12.2. Retail Pharmacies

13. Amyotrophic Lateral Sclerosis Treatment Market, by Region

• 13.1. Americas
  • 13.1.1. North America
  • 13.1.2. Latin America
• 13.2. Europe, Middle East & Africa
  • 13.2.1. Europe
  • 13.2.2. Middle East
  • 13.2.3. Africa
• 13.3. Asia-Pacific

14. Amyotrophic Lateral Sclerosis Treatment Market, by Group

• 14.1. ASEAN
• 14.2. GCC
• 14.3. European Union
• 14.4. BRICS
• 14.5. G7
• 14.6. NATO

15. Amyotrophic Lateral Sclerosis Treatment Market, by Country

• 15.1. United States
• 15.2. Canada
• 15.3. Mexico
• 15.4. Brazil
• 15.5. United Kingdom
• 15.6. Germany
• 15.7. France
• 15.8. Russia
• 15.9. Italy
• 15.10. Spain
• 15.11. China
• 15.12. India
• 15.13. Japan
• 15.14. Australia
• 15.15. South Korea

16. Competitive Landscape

• 16.1. Market Share Analysis, 2024
• 16.2. FPNV Positioning Matrix, 2024
• 16.3. Competitive Analysis
  • 16.3.1. AB Science SA
  • 16.3.2. Alector, Inc.
  • 16.3.3. Amylyx Pharmaceuticals, Inc.
  • 16.3.4. Apotex Inc.
  • 16.3.5. Aquestive Therapeutics, Inc.
  • 16.3.6. Ascend Pharmaceuticals LLC
  • 16.3.7. AstraEureka Pharmaceuticals
  • 16.3.8. Biogen Inc.
  • 16.3.9. BrainStorm Cell Limited
  • 16.3.10. Cipla Limited
  • 16.3.11. CorestemChemon Inc.
  • 16.3.12. Coya Therapeutics, Inc.
  • 16.3.13. Cytokinetics, Incorporated
  • 16.3.14. Dewpoint Therapeutics, Inc.
  • 16.3.15. DR. REDDY'S LABORATORIES LIMITED
  • 16.3.16. F. Hoffmann-La Roche Ltd
  • 16.3.17. Genervon Biopharmaceuticals, LLC
  • 16.3.18. GlaxoSmithKline PLC
  • 16.3.19. Implicit Bioscience Ltd.
  • 16.3.20. Ionis Pharmaceuticals, Inc.
  • 16.3.21. ITF Pharma, Inc.
  • 16.3.22. Kringle Pharma, Inc.
  • 16.3.23. Mitsubishi Tanabe Pharma Corporation
  • 16.3.24. Neurimmune AG
  • 16.3.25. Novartis AG
  • 16.3.26. ONO PHARMACEUTICAL CO., LTD.
  • 16.3.27. Orion Corporation
  • 16.3.28. Orphazyme A/S
  • 16.3.29. Otsuka Pharmaceutical Co., Ltd.
  • 16.3.30. Pfizer, Inc.
  • 16.3.31. Sanofi SA
  • 16.3.32. Sun Pharmaceutical Industries Ltd
  • 16.3.33. Teva Pharmaceutical Industries Ltd.
  • 16.3.34. Treeway B.V.
  • 16.3.35. Viatris Inc.
  • 16.3.36. Zydus Lifesciences Limited