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 Pioneering New Frontiers In Market Innovation

• The Rise of Digital Therapeutics and AI: A significant trend is the integration of technology, with the global digital therapeutics (DTx) market for CNS diseases valued at US$ 234.5 million in 2024. Companies are leveraging artificial intelligence for faster drug discovery, with the global AI in drug discovery market projected to reach US$ 6.93 billion in 2025. AI is being used to optimize trial design, enhance patient recruitment, and even for de novo peptide design to create novel therapeutics. This digital shift aims to improve patient outcomes through software-based interventions and accelerate the development of more precise medicines.
• Targeting Neuroinflammation: Research is increasingly focused on neuroinflammation as a key driver of central nervous system therapeutic market. Beyond the traditional targets of amyloid and tau, the 2025 Alzheimer's pipeline shows a growing emphasis on modulating the brain's immune response. Investigational drugs are targeting pathways like the NLRP3 inflammasome to restore balanced immune activity, moving away from broad suppression. This reflects a deeper understanding that chronic inflammation accelerates pathology in conditions like Alzheimer's, Parkinson's, and multiple sclerosis, opening a major new therapeutic avenue.
• Psychedelics Entering Mainstream Research: Psychedelic compounds are gaining significant traction as potential breakthrough therapies for psychiatric disorders. Following promising clinical data, there is a major push for regulatory review, although the FDA has requested additional research for MDMA-assisted therapy for PTSD. Nonetheless, federal agencies like the VA are actively funding and supporting research into psychedelics for conditions like PTSD and depression. This represents a paradigm shift, moving these substances from the fringe to the forefront of psychiatric drug development.

Psychedelic Medicine Breakthroughs Reshape Mental Healthcare Treatment Paradigms

Demand for novel mental health solutions is surging, with psychedelic-assisted therapies transitioning from niche research to a pivotal force in the Central Nervous System therapeutic market. This is quantified by significant clinical and operational momentum. MindMed’s Phase 2b trial for its psychedelic candidate MM120 enrolled 198 participants for generalized anxiety disorder. Cybin’s Phase 2 study of its psilocybin analog CYB003 for depression included a cohort of 34 participants, analyzing multiple dosage arms. Further, Beckley Psytech's trial for its short-acting psychedelic BPL-003, which is under atai Life Sciences, involved 84 participants. Atai Life Sciences maintains a broad portfolio of at least 11 psychedelic and related compounds in its pipeline as of early 2025.

The infrastructure to deliver these potential treatments is expanding rapidly; there are now between 1,200 and 1,500 ketamine clinics operating in the U.S. central nervous system therapeutic market as of 2024. One provider, Numinus Wellness, managed 15 clinical trials and conducted 17,661 client appointments in the second quarter of 2024 alone. Regulatory engagement is intensifying, exemplified by the FDA advisory committee's recent review of an MDMA-based therapy, where the panel voted 9-2 against its effectiveness based on trial data. Another panel vote resulted in a 10-1 decision that the therapy's benefits did not outweigh its risks. Despite regulatory hurdles, the clinical investigation continues, with Lykos Therapeutics' application for MDMA-assisted therapy supported by six Phase 2 and two Phase 3 trials.

Gene And Cell Therapies Promise Cures For Rare Neurological Disorders

A powerful demand driver in the Central Nervous System therapeutic market is the rapid advancement of curative gene and cell therapies for rare, debilitating neurological diseases. The clinical pipeline is maturing, substantiated by concrete patient and program numbers. Orchard Therapeutics is advancing OTL-200, a gene therapy for MLD, supported by studies enrolling a total of 39 children. As of early 2024, there are eight approved cell and gene therapies on the market for various CNS disorders. Bluebird bio is scaling up access for its therapy SKYSONA, establishing a network of 35 qualified treatment centers across the U.S.

The patient impact is growing, with Novartis reporting that over 3,000 patients have been treated with its gene therapy Zolgensma as of early 2024. Pipeline development is robust in the central nervous system therapeutic market; REGENXBIO’s Phase 1/2/III trial for RGX-121 in Hunter syndrome aims to enroll approximately 6 children in its next stage. Voyager Therapeutics showcases a diverse pipeline with at least four wholly-owned programs for diseases like Alzheimer's, Parkinson's, and ALS. The company’s partnership with Novartis for a TRACER-generated capsid could yield over 305 million in milestone payments. Another collaboration with Neurocrine is expected to generate up to 35 million in milestones for Voyager by 2026. These partnerships are vital, with IND submissions for Friedreich's ataxia and GBA1 gene therapy programs slated for 2025.

 Segmental Analysis 

Neurovascular Disease Segment's Unwavering 42% Hold on the Market

Neurovascular diseases command 42% of the central nervous system therapeutic market due to the high volume of affected individuals. Stroke alone impacts 12.2 million new people annually, creating urgent demand. An aging global population and the severe nature of these disorders fuel significant R&D investment. The economic burden is substantial, with the global cost of stroke exceeding US$ 890 billion annually. Demand is also powered by innovations in diagnostics and minimally invasive techniques improving patient outcomes. Supporting this progress, over US$ 2 million in NIH funding is advancing novel stroke treatments, while a 2025 study with 372 patients is evaluating alteplase use up to 24 hours post-stroke. The Irish government has also allocated €7.3 million to its National Stroke Strategy.

• Key Diseases Fueling Unprecedented Demand in Neurovascular Therapeutics

Demand within this segment of the Central nervous System therapeutic market is propelled by key diseases. Ischemic stroke is most prominent, with 7.6 million new cases yearly. Cerebral aneurysms and arteriovenous malformations (AVMs) also create strong demand for preventative treatments to avoid rupture. These conditions affect a massive population, including 110 million people worldwide who have experienced a stroke. Reducing long-term disability for 5 million stroke survivors annually drives innovation. The Brain Aneurysm Foundation will award $580,000 in 2025 for 14 research projects, while The Bee Foundation offers grants up to $25,000 for early detection. Nearly 700,000 new subarachnoid hemorrhages occur globally each year, highlighting the scale.

• Global Patient Population Highlights the Immense Neurovascular Challenge

The immense global patient population underscores the central nervous system therapeutic market’s scale. Approximately 94 million people live with a history of stroke, with 15 million new cases annually. In the U.S., 795,000 people have a stroke each year, including 185,000 recurrent events requiring ongoing prevention. The issue is not confined to the elderly; 15% of strokes affect those aged 15 to 49. The 5 million annual stroke-related deaths represent a significant unmet need. Clinical trials show promise, with VNS-REHAB demonstrating a 5-point gain and stem cell therapies an 11.4-point improvement on the Fugl-Meyer Assessment. The Aneurysm and AVM Foundation also offers research grants up to $25,000 in 2025.

• Breakthrough Therapeutics Revolutionizing Neurovascular Disease Treatment and Care

The central nervous system therapeutic market is driven by advanced medical devices and pharmaceuticals. For ischemic stroke, mechanical thrombectomy is now standard care, while embolization coils treat cerebral aneurysms. Thrombolytic drugs like alteplase remain critical, with 2025 studies exploring their use up to 24 hours post-event. Minimally invasive procedures are a key trend improving recovery. Emerging technologies include neurostimulation systems like Vivistim for rehabilitation. Novel therapies involving mesenchymal stem cells also represent the next frontier. Investment continues with aneurysm research grants in 2025 from $20,000 to $50,000 and Ireland's Budget 2025 allocating €5 million to expand stroke services. A recent trial enrolled patients across 72 sites, showing research scale.

Regional Analysis 

North America's Dominance Driven By Robust Funding and Regulatory Velocity

North America commands the Central nervous system therapeutic market, holding a market share of over 47%. The region's leadership is underpinned by substantial government investment and an accelerated regulatory environment. For the fiscal year 2025, the U.S. National Institute of Neurological Disorders and Stroke (NINDS) has a budget request of $2,833.8 million to advance brain research. In a significant 2024 commitment, the Canadian government awarded Brain Canada $80 million over four years, which will be matched for a total investment of $160 million. To foster emerging talent, another Brain Canada program will support up to 20 grants of $100,000 each for early-career investigators in 2024.

Regulatory approvals in the U.S. central nervous system therapeutic market are a key indicator of market vitality. In 2024, the FDA approved several critical CNS therapies, including Kisunla (donanemab) for Alzheimer's disease and Cobenfy, the first new schizophrenia drug in decades. The agency also cleared multiple neurology-focused devices. In total, the FDA authorized 50 novel medicines in 2024, outpacing other global agencies. Further solidifying its research infrastructure, the U.S. NIH's BRAIN Initiative continues to receive substantial funding, enabling programs at institutions like UCSF. The NIH is also actively soliciting applications for 180 open funding opportunities related to neurological disorders as of mid-2025. A joint U.S.-Japan Brain Research Cooperative Program held a major networking session in October 2024 to foster international collaboration.

European Union Accelerates Brain Health Initiatives and Collaborative Research

Europe is solidifying its position in the Central nervous system therapeutic market through major collaborative funding initiatives and a steady stream of regulatory approvals. The European Medicines Agency (EMA) recommended 114 drugs for approval in 2024, including key neurology treatments. A landmark development is the European Partnership for Brain Health, a Horizon Europe program set to launch in 2025 with a budget of €56,500,000. Another draft Horizon Europe call for 2025 allocates €50 million for innovative interventions in mental and neurodevelopmental disorders. The partnership aims to unite 21 participants from 11 countries to advance collaborative research.

In the United Kingdom, dementia research received a significant boost in 2024 with a £20 million commitment from the National Institute for Health and Care Research (NIHR) to the UK Dementia Research Institute. The government has also committed to increasing total dementia research funding to £160 million by 2024. On the regulatory front in central nervous system therapeutic market, the EMA approved Leqembi for early Alzheimer's disease and confirmed orphan designation for 15 medicines, including the neurology drug Qalsody. The EMA is also actively engaging with emerging science, holding a workshop on psychedelics in 2024 to prepare for future therapeutic applications. These concerted efforts underscore a region committed to tackling complex neurological challenges.

Asia Pacific Emerges With Accelerated Drug Approvals and Research Investment

The Asia Pacific region is rapidly advancing its role in the global Central nervous system therapeutic market, driven by streamlined regulatory processes and targeted research funding. In China, the Center for Drug Evaluation (CDE) accepted 19,563 drug registration applications in 2024. Demonstrating a commitment to innovation, China's CDE approved 91 breakthrough therapy applications in 2024, a significant number of which were for neurological and anti-infective drugs. A new pilot program in China for innovative drugs saw its first approval in just 21 days. Out of nine clinical trials included in the pilot, five have already submitted IND applications.

Japan is also heavily investing in central nervous system therapeutic market, with its government requesting a budget of 2,400.8 billion yen for MEXT's science and technology initiatives in fiscal year 2024. The Japan Agency for Medical Research and Development (AMED) was allocated a budget of 148.9 billion yen for 2024 to support various projects, including dementia research. To foster global partnerships, the Japan-U.S. Brain Research Cooperation Program is funding seminars in 2024 with a maximum budget of JPY 2,500,000 per event. Meanwhile, Australia’s Medical Research Future Fund (MRFF) distributed 578 competitive grants between 2022 and 2024, with universities managing 519 of these projects. The MRFF's 2024 BioMedTech Incubator grant is specifically designed to support early-stage medical innovation.

Billion-Dollar Deals and Breakthroughs Powering Central Nervous System Therapeutic Market

• AbbVie Acquires Cerevel Therapeutics: In a landmark deal solidifying its neuroscience pipeline, AbbVie completed its acquisition of Cerevel Therapeutics for approximately $8.7 billion in early 2024.
• Bristol Myers Squibb Finalizes Karuna Therapeutics Purchase: Bristol Myers Squibb closed its $14 billion acquisition of Karuna Therapeutics, gaining the promising schizophrenia drug KarXT (Cobenfy).
• Novo Nordisk Acquires Ocedurenone from KBP Biosciences: In a deal worth up to $1.3 billion, Novo Nordisk acquired Ocedurenone for uncontrolled hypertension, which has CNS implications, from KBP Biosciences.
• Alto Neuroscience Launches Successful IPO: Demonstrating strong investor confidence in precision psychiatry, Alto Neuroscience raised $129 million in its Initial Public Offering in February 2024.
• Engrail Therapeutics Secures Major Series B Funding: Engrail Therapeutics raised an oversubscribed $157 million in Series B financing to advance its pipeline of therapies for anxiety, depression, and other CNS disorders, adding fuel to the central nervous system therapeutic market growth.
• Latigo Biotherapeutics Launches with $135 Million Series A: Focused on developing non-opioid pain therapeutics, Latigo Biotherapeutics launched with a substantial $135 million Series A financing round.
• Alumis Raises $259 Million for Neuroinflammatory Disease Pipeline: Alumis closed an upsized $259 million Series C financing to support its development of therapies for neuroinflammatory and autoimmune diseases.
• PsychoGenics Acquires Cordoba Services: To bolster its preclinical CNS research capabilities, PsychoGenics acquired the CRO Cordoba Services S.L. in March 2024.
• Rapport Therapeutics Secures $150 Million Series B: Rapport Therapeutics raised $150 million in a Series B financing round to advance its precision neuroscience medicines for epilepsy and other CNS disorders.
• GSK Acquires Aiolos Bio: In a deal valued at $1 billion upfront with up to $400 million in milestones, GSK acquired Aiolos Bio, signaling a move that could have future applications in neuro-inflammatory conditions.

Top Companies in the Central Nervous System Therapeutic Market

• AstraZeneca plc
• Amgen Inc.
• ACADIA Pharmaceuticals Inc.
• Biogen Inc.
• GlaxoSmithKline plc
• Roche Holding AG
• Merck & Co., Inc.
• Otsuka Holdings Co., Ltd.
• Novartis AG
• Sage Therapeutics, Inc.
• GW Pharmaceuticals plc
• Marinus Pharmaceuticals, Inc.
• Sunovion Pharmaceuticals Inc.
• Eli Lilly and Company
• Other Prominent Players

By Disease

• CNS Trauma
• CNS Cancer
• Infectious Diseases
• Mental Health
  • Anxiety Disorders
  • Mood Disorders
  • Epilepsy
  • Psychotic Disorders
  • Others
• Neurodegenerative Diseases
  • Parkinson’s Disease
  • Alzheimer’s Disease
  • Amyotrophic Lateral Sclerosis
  • Multiple Sclerosis
  • Huntington’s Disease
  • Others
• Neurovascular Diseases
• Others

By Region

• North America
  • The U.S.
  • Canada
  • Mexico
• Europe
  • Western Europe
    • The UK
    • Germany
    • France
    • Italy
    • Spain
    • Rest of Western Europe
  • Eastern Europe
    • Poland
    • Russia
    • Rest of Eastern Europe
• Asia Pacific
  • China
  • India
  • Japan
  • Australia & New Zealand
  • South Korea
  • ASEAN
  • Rest of Asia Pacific
• Middle East
  • Saudi Arabia
  • Kuwait
  • UAE
  • Qatar
  • Oman
  • Bahrain
  • Rest of Middle East
• Africa
  • South Africa
  • Egypt
  • Nigeria
  • Kenya
  • Rest of Africa
• South America
  • Argentina
  • Brazil
  • Rest of South America