Summary

Overview
The global rare neurological disease drugs market reached US$ 139.4 billion in 2023 and is expected to reach US$ 269.7 billion by 2031 growing with a CAGR of 8.6% during the forecast period 2024-2031.
Rare neurological diseases, also referred to as rare neurologic disorders or orphan neurological disorders, are a range of unusual and varied conditions that impact the nervous system. Rare neurological diseases are a heterogeneous group of disorders that primarily affect the central and peripheral nervous systems, as well as the muscles. These conditions are considered "rare" due to their low prevalence, typically defined as affecting less than 1 in 2,000 people.
Rare neurological diseases that target the nervous system of the human body include the brain and spinal cord. There are various rare neurological disorders such as Aicardi syndrome, Aicardi-Goutieres syndrome, Reflex Sympathetic Dystrophy Syndrome, Battaglia-Neri syndrome, Creutzfeldt Jakob Disease, Agnosia, and many others.
There are various therapies used for the treatment of rare neurological diseases drugs such as cognitive behavioral therapy, interpersonal psychotherapy, cyberknife, and others. Drugs such as levetiracetam (Keppra), topiramate (Topamax), lamotrigine (Lamictal), oxcarbazepine (Trileptal), and divalproex sodium (Depakote).
Market Dynamics: Drivers
Increasing Prevalence of Rare Neurological Diseases
The demand for the global rare neurological disease drugs market is driven by multiple factors. One of the primary factors is the increasing prevalence of rare neurological diseases. According to the World Health Organization (WHO), in May 2024, over 1 in 3 people were affected by neurological conditions, the leading cause of illness and disability worldwide.
Some rare neurological diseases include amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy, Vertical gaze palsy, and Huntington's disease, among others. As per the Orphanet report published in November 2023, the prevalence of chronic inflammatory demyelinating polyneuropathy is 3.7 per 100,000 individuals in Europe, and the prevalence of ALS is 3.85 worldwide and 5.2 in the European Union (EU) in 2023. As per the same source, the prevalence of Huntington's disease is 12.0 in the EU in 2023.
Furthermore, key players' strategies such as partnerships & collaborations, a rising number of clinical trials, research studies, and product launches & approvals are expected to propel the market growth. For instance, in July 2023, Biogen Inc. Reata Pharmaceuticals, Inc. announced the companies had entered into a definitive agreement under which Biogen has agreed to acquire Reata for $172.50 per share in cash, reflecting an enterprise value of approximately $7.3 billion.
Also, in April 2023, Orphalan SA, an international orphan drug development and commercialization company, announced the commercial launch in the U.S. for Cuvrior. It is a new trientine tetrahydrochloride (TETA-4HCl). It is now available for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to D-penicillamine.
Similarly, in April 2023, Biogen Inc announced that the U.S. Food and Drug Administration (FDA) has approved QALSODY (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.
Restraints
Factors such as the high cost of treatment, stringent regulatory requirements, lack of awareness & diagnosis, small patient population, limited treatment options, reimbursement challenges, and lack of skilled & trained healthcare professionals are expected to hamper the market.
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Segment Analysis
The global rare neurological disease drugs are segmented based on drug type, route of administration, application, distribution channel, and region.
The small molecules segment accounted for approximately 64.3% of the global rare neurological disease drugs market share
The small molecules segment is expected to hold the largest market share over the forecast period. Small molecule drugs have several advantages that make them well-suited for rare neurological disease treatments. Their small size allows them to easily penetrate the blood-brain barrier and access intracellular targets within the central nervous system.
This enables the modulation of specific protein functions implicated in rare neurological disorders. Additionally, small molecules can be manufactured more quickly and cost-effectively compared to larger biological drugs.
Moreover, key players in the industry strategies such as partnerships & collaborations, product approvals, and financial investments would drive this market growth. For instance, in April 2024, Ipsen and Skyhawk Therapeutics announced the signing of an exclusive worldwide collaboration to discover and develop novel small molecules that modulate RNA for rare neurological diseases.
Similarly, in January 2024, Biogen Inc. announced plans to reprioritize its resources in Alzheimer’s disease (AD), a strategic therapeutic area expected to drive near and long-term growth. The company will continue to advance LEQEMBI (lecanemab-irmb), the first anti-amyloid beta treatment with FDA traditional approval in the United States, and will accelerate the development of potential new treatment modalities, including its ASO targeting tau (BIIB080) and an oral small molecule inhibitor of tau aggregation (BIIB113).
Also, in March 2023, Rapport Therapeutics launched with $100 million in a series A financing to support its novel platform to discover precision-targeted small molecule drugs to treat neurological disorders.
Geographical Analysis
North America accounted for approximately 44.3% of the global rare neurological disease drugs market share
North American region is expected to hold the largest market share over the forecast period owing to the growing incidence of rare neurological disorders, availability of reimbursement for the treatment of rare diseases, and increasing research and development (R&D) in the region.
As fda.gov stated in February 2023, a rare disease is any disease that affects fewer than 200,000 people in the U.S. Approximately 25 to 30 million Americans are living with a rare disease (about 1 in 10 people), and many rare diseases have few or no available treatment options.
Moreover, a major number of key players' presence, well-advanced healthcare infrastructure, and government initiatives such as awareness programs would drive this market growth. For instance, in June 2024, Neurogene Inc. announced that its NGN-401 gene therapy for Rett syndrome has been selected to participate in the U.S. Food and Drug Administration (FDA) Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program.
Similarly, in May 2022, CDER launched the Accelerating Rare Disease Cures (ARC) Program. The ARC Program seeks to harness CDER’s collective expertise and activities to drive scientific and regulatory innovation for rare diseases.
In addition, the rising number of clinical trials, product launched and approvals would propel this market growth in this region. For instance, in March 2023, The FDA signed off on Reata’s Skyclarys (omoveloxolone) for the treatment of Friedreich’s ataxia (FA), a hereditary neurological disorder that strikes during adolescence and leaves patients wheelchair-bound and often cuts short their lives. Skyclarys became the first drug approved for the condition, which affects roughly 5,000 who have been diagnosed in the United States and 22,000 worldwide.
Also, in September 2022, Acadia Pharmaceuticals announced Trofinetide's new drug application for the treatment of Rett syndrome has been accepted for filing and review by the U.S. FDA
Similarly, in June 2024, Lucy Therapeutics announced it has raised $12.5m in additional funding to develop potentially new drugs and advance the company’s research programs for neurological diseases.
Market Segmentation
By Drug Type
• Biologics
• Small Molecules
By Route of Administration
• Intravenous
• Oral
By Application
• Dravet syndrome
• Adrenoleukodystrophy
• Narcolepsy
• Angelman syndrome
• Amyotrophic Lateral Sclerosis
• Others
By Distribution Channel
• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies
By Region
• North America
o U.S.
o Canada
o Mexico
• Europe
o Germany
o U.K.
o France
o Spain
o Italy
o Rest of Europe
• South America
o Brazil
o Argentina
o Rest of South America
• Asia-Pacific
o China
o India
o Japan
o South Korea
o Rest of Asia-Pacific
• Middle East and Africa
Competitive Landscape
The major global players in the rare neurological disease drugs market include Novartis AG, Pfizer, Inc., Johnson & Johnson Services, Inc., US WorldMeds LLC (Solstice Neurosciences LLC), Aquestive Therapeutics Inc., Sanofi S.A., Merck & Co., Inc., CSL Ltd, Merz Pharma GmbH & Co. KGaA, and Kedrion Biopharma Inc. among others.
Key Developments
 In May 2024, Biogen Inc announced the European Commission (EC) has granted marketing authorization under exceptional circumstances and maintained orphan designation for QALSODY (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS).
 In April 2024, NS Pharma Announces Research Alliance with MiNA Therapeutics to Develop Therapies for Rare Diseases of the Central Nervous System.
 In February 2024, Roche Pharma India announced the launch of its blockbuster breakthrough drug, Ocrevus (Ocrelizumab), for the treatment of multiple sclerosis (MS), expanding its neurology portfolio to cater to unmet needs of numerous patients grappling with this debilitating disease in India
 In September 2023, Harmony Biosciences Holdings, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisantf for the treatment of idiopathic hypersomnia (IH).
Why Purchase the Report?
• To visualize the global rare neurological disease drugs market segmentation based on drug type, route of administration, application, distribution channel, and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of global rare neurological disease treatment market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as Excel consisting of key products of all the major players.
The global rare neurological disease drugs market report would provide approximately 69 tables, 68 figures, and 183 pages.
Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

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Table of Contents

Table of Contents
1. Methodology and Scope
1.1. Research Methodology
1.2. Research Objective and Scope of the Report
2. Definition and Overview
3. Executive Summary
3.1. Snippet by Drug Type
3.2. Snippet by Route of Administration
3.3. Snippet by Application
3.4. Snippet by Distribution Channel
3.5. Snippet by Region
4. Dynamics
4.1. Impacting Factors
4.1.1. Drivers
4.1.1.1. Increasing Prevalence of Rare Neurological Diseases
4.1.1.2. Innovative Development of New Drugs for Rare Neurological Diseases
4.1.2. Restraints
4.1.2.1. High Cost of Treatment
4.1.3. Opportunity
4.1.4. Impact Analysis
5. Industry Analysis
5.1. Porter’s Five Force Analysis
5.2. Supply Chain Analysis
5.3. Pricing Analysis
5.4. Regulatory Analysis
6. By Drug Type
6.1. Introduction
6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
6.1.2. Market Attractiveness Index, By Drug Type
6.2. Biologics
6.2.1. Introduction
6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
6.3. Small Molecules
7. By Route of Administration
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
7.1.2. Market Attractiveness Index, By Route of Administration
7.2. Intravenous*
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. Oral
8. By Application
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
8.1.2. Market Attractiveness Index, By Application
8.2. Dravet syndrome *
8.2.1. Introduction
8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
8.3. Adrenoleukodystrophy
8.4. Narcolepsy
8.5. Angelman syndrome
8.6. Amyotrophic Lateral Sclerosis
8.7. Others
9. By Distribution Channel
9.1. Introduction
9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
9.1.2. Market Attractiveness Index, By Distribution Channel
9.2. Hospital Pharmacies*
9.2.1. Introduction
9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
9.3. Retail Pharmacies
9.4. Online Pharmacies
10. By Region
10.1. Introduction
10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
10.1.2. Market Attractiveness Index, By Region
10.2. North America
10.2.1. Introduction
10.2.2. Key Region-Specific Dynamics
10.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
10.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.2.7.1. U.S.
10.2.7.2. Canada
10.2.7.3. Mexico
10.3. Europe
10.3.1. Introduction
10.3.2. Key Region-Specific Dynamics
10.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
10.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.3.7.1. Germany
10.3.7.2. U.K.
10.3.7.3. France
10.3.7.4. Spain
10.3.7.5. Italy
10.3.7.6. Rest of Europe
10.4. South America
10.4.1. Introduction
10.4.2. Key Region-Specific Dynamics
10.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
10.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
10.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.4.7.1. Brazil
10.4.7.2. Argentina
10.4.7.3. Rest of South America
10.5. Asia-Pacific
10.5.1. Introduction
10.5.2. Key Region-Specific Dynamics
10.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
10.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
10.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.5.7.1. China
10.5.7.2. India
10.5.7.3. Japan
10.5.7.4. South Korea
10.5.7.5. Rest of Asia-Pacific
10.6. Middle East and Africa
10.6.1. Introduction
10.6.2. Key Region-Specific Dynamics
10.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
10.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
11. Competitive Landscape
11.1. Competitive Scenario
11.2. Market Positioning/Share Analysis
11.3. Mergers and Acquisitions Analysis
12. Company Profiles
12.1. Novartis AG*
12.1.1. Company Overview
12.1.2. Product Portfolio and Description
12.1.3. Financial Overview
12.1.4. Key Developments
12.2. Pfizer, Inc
12.3. Johnson & Johnson Services, Inc
12.4. US WorldMeds LLC (Solstice Neurosciences LLC)
12.5. Aquestive Therapeutics Inc
12.6. Sanofi S.A.
12.7. Merck & Co., Inc
12.8. CSL Ltd
12.9. Merz Pharma GmbH & Co. KGaA
12.10. Kedrion Biopharma Inc
LIST NOT EXHAUSTIVE
13. Appendix
13.1. About Us and Services
13.2. Contact Us