The rare neurological disease treatment market size has grown strongly in recent years. It will grow from $9.85 billion in 2024 to $10.68 billion in 2025 at a compound annual growth rate (CAGR) of 8.4%. The growth in the historic period can be attributed to biosimilars in rare disease treatment, globalization of clinical trials, healthcare reimbursement policies, orphan drug designations, advancements in genetic research.
The rare neurological disease treatment market size is expected to see strong growth in the next few years. It will grow to $14.84 billion in 2029 at a compound annual growth rate (CAGR) of 8.6%. The growth in the forecast period can be attributed to improved diagnostic tools, precision medicine advancements, patient-centric drug development, integration of real-world evidence, telemedicine for patient support, shift towards personalized therapies. Major trends in the forecast period include technological innovations, ai in drug discovery, blockchain for data security, machine learning in drug discovery.
The anticipated growth of the rare neurological disease treatment market is expected to be driven by the prevalence of rare neurological diseases. These conditions, characterized by low prevalence, encompass disorders such as Huntington's disease, amyotrophic lateral sclerosis (ALS), Rett syndrome, and various forms of muscular dystrophy. Treating rare neurological diseases involves employing diverse approaches to manage symptoms, slow disease progression, and enhance the quality of life for affected individuals. For example, as reported in January 2023 by BioMed Central Ltd., the collective prevalence of Rett syndrome was determined to be 7.1 per 100,000 females in a sample size of 9.57 million women, with a total of 673 confirmed cases. Therefore, the prevalence of rare neurological diseases is a significant driver for the growth of the rare neurological disease treatment market.
The increase in healthcare expenditures is projected to drive the growth of the rare neurological disease treatment market in the future. Healthcare expenditures encompass all costs related to providing health services, family planning initiatives, nutrition programs, and emergency assistance, with a focus on health. Higher healthcare spending enables research, development, access to specialized care, and awareness, all of which are essential for improving treatments and support for rare neurological diseases. For example, in October 2024, the Czech Statistical Office, a governmental organization in the Czech Republic, reported that total healthcare expenditure rose from 579.6 billion in 2021 to 594.7 billion in 2022, representing an increase of about 2.6%. Consequently, the rise in healthcare expenditures is fueling the growth of the rare neurological disease treatment market.
A noteworthy trend gaining traction in the rare neurological disease treatment market is the development of orphan pharmaceuticals. Key companies in the sector are directing their efforts towards creating innovative and specialized treatments to solidify their market positions. For instance, in April 2023, French orphan drug development company Orphalan SA introduced Cuvrior, a novel trientine tetrahydrochloride (TETA-4HCl), in the European market. Cuvrior is commercially available for treating adult patients with stable Wilson disease who have undergone copper reduction therapy and exhibited tolerance to D-penicillamine. This drug received approval from the United States Food and Drug Administration (FDA) and was granted orphan drug exclusivity (ODE) in April 2022 for this specific patient population.
Major players in the rare neurological disease treatment market are placing a strong emphasis on product innovation, including rare disease drug development solutions, to advance therapeutic options and enhance the quality of life for patients affected by rare neurological diseases. Developing treatments for rare neurological diseases necessitates targeted research into specific disease mechanisms and the exploration of innovative therapeutic approaches, such as gene therapies or personalized medicine, to address the complex nature of these conditions. For instance, in February 2023, US-based pharmaceutical company Reata Pharmaceuticals Inc. received approval from the United States Food and Drug Administration (USFDA) for Skyclarys, a drug designed to treat Friedreich’s ataxia, a rare neurological disease. Skyclarys represents an experimental medication addressing unmet needs in treating rare neurological conditions by targeting intricate disease mechanisms. It is undergoing rigorous clinical evaluation for its efficacy and safety profiles.
In February 2024, Harmony Biosciences, a biopharmaceutical company based in the United States, acquired Epygenix Therapeutics, Inc. for an undisclosed sum. The purpose of this acquisition is to improve treatments for rare epilepsy disorders such as Dravet syndrome and Lennox-Gastaut syndrome, thereby expediting the development of innovative therapies to address these unmet medical needs. Epygenix Therapeutics, Inc. is a clinical-stage biopharmaceutical company in the U.S. that focuses on developing and commercializing cutting-edge therapies.
Major companies operating in the rare neurological disease treatment market include Pfizer Inc., Johnson & Johnson Services Inc., F. Hoffmann-La Roche Ltd, Bayer AG, Merck & Co. Inc., AbbVie Inc., Novartis AG, Sanofi-Aventis SA, Bristol-Myers Squibb Company, AstraZeneca PLC, GlaxoSmithKline PLC, Takeda Pharmaceutical Company Limited, Medtronic PLC, Teva Pharmaceutical Industries Ltd., CSL Limited, Biogen Inc., UCB S.A., Eisai Co. Ltd., Otsuka Pharmaceutical Co. Ltd., Jazz Pharmaceuticals PLC, Mitsubishi Tanabe Pharma Corporation, Neurocrine Biosciences Inc., Kedrion Biopharma Inc., Merz Pharma GmbH & Co. KGaA, Harmony Biosciences Holdings Inc., Amicus Therapeutics Inc., Revance Therapeutics Inc., Aquestive Therapeutics Inc., US WorldMeds LLC (Solstice Neurosciences LLC), Stoke Therapeutics Inc., Retrotope Inc.
North America was the largest region in the rare neurological disease’s treatment market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the rare neurological disease treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the rare neurological disease treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
Treatment for rare neurological diseases involves medical interventions and therapies with the goal of managing symptoms, slowing disease progression, and enhancing the quality of life for individuals affected by these uncommon conditions. Rare neurological diseases encompass disorders that affect the nervous system, including the brain, spinal cord, and peripheral nerves.
The primary types of treatment for rare neurological diseases involve drugs, including biologics and small molecules. Biologics, derived from living organisms such as proteins, antibodies, or nucleic acids, play a crucial role in treating rare neurological diseases. Examples include enzyme replacement therapies for lysosomal storage disorders and monoclonal antibodies for immune-mediated neurological disorders. Various therapies, such as cognitive behavioral therapy, interpersonal psychotherapy, and cyberknife, are administered through oral and injectable modes to address specific conditions such as Aicardi syndrome, Aicardi Goutières syndrome, reflex sympathetic dystrophy syndrome, Battaglia-Neri syndrome, Creutzfeldt-Jakob disease, agnosia, and others. Medications are distributed through channels including hospitals, retail pharmacies, drug stores, and online pharmacies.
The rare neurological diseases treatment market research report is one of a series of new reports that provides rare neurological diseases treatment market statistics, including the rare neurological diseases treatment industry's global market size, regional shares, competitors with a rare neurological diseases treatment market share, detailed rare neurological diseases treatment market segments, rare neurological diseases treatment market trends and opportunities, and any further data you may need to thrive in the rare neurological diseases treatment industry. This rare neurological diseases treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The rare neurological disease treatment market consists of revenues earned by entities by providing supportive care services and neurodegenerative research services. The market value includes the value of related goods sold by the service provider or included within the service offering. The rare neurological disease treatment market also includes sales of anticonvulsants, antidepressants, and beta blockers. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
The rare neurological disease treatment market size is expected to see strong growth in the next few years. It will grow to $14.84 billion in 2029 at a compound annual growth rate (CAGR) of 8.6%. The growth in the forecast period can be attributed to improved diagnostic tools, precision medicine advancements, patient-centric drug development, integration of real-world evidence, telemedicine for patient support, shift towards personalized therapies. Major trends in the forecast period include technological innovations, ai in drug discovery, blockchain for data security, machine learning in drug discovery.
The anticipated growth of the rare neurological disease treatment market is expected to be driven by the prevalence of rare neurological diseases. These conditions, characterized by low prevalence, encompass disorders such as Huntington's disease, amyotrophic lateral sclerosis (ALS), Rett syndrome, and various forms of muscular dystrophy. Treating rare neurological diseases involves employing diverse approaches to manage symptoms, slow disease progression, and enhance the quality of life for affected individuals. For example, as reported in January 2023 by BioMed Central Ltd., the collective prevalence of Rett syndrome was determined to be 7.1 per 100,000 females in a sample size of 9.57 million women, with a total of 673 confirmed cases. Therefore, the prevalence of rare neurological diseases is a significant driver for the growth of the rare neurological disease treatment market.
The increase in healthcare expenditures is projected to drive the growth of the rare neurological disease treatment market in the future. Healthcare expenditures encompass all costs related to providing health services, family planning initiatives, nutrition programs, and emergency assistance, with a focus on health. Higher healthcare spending enables research, development, access to specialized care, and awareness, all of which are essential for improving treatments and support for rare neurological diseases. For example, in October 2024, the Czech Statistical Office, a governmental organization in the Czech Republic, reported that total healthcare expenditure rose from 579.6 billion in 2021 to 594.7 billion in 2022, representing an increase of about 2.6%. Consequently, the rise in healthcare expenditures is fueling the growth of the rare neurological disease treatment market.
A noteworthy trend gaining traction in the rare neurological disease treatment market is the development of orphan pharmaceuticals. Key companies in the sector are directing their efforts towards creating innovative and specialized treatments to solidify their market positions. For instance, in April 2023, French orphan drug development company Orphalan SA introduced Cuvrior, a novel trientine tetrahydrochloride (TETA-4HCl), in the European market. Cuvrior is commercially available for treating adult patients with stable Wilson disease who have undergone copper reduction therapy and exhibited tolerance to D-penicillamine. This drug received approval from the United States Food and Drug Administration (FDA) and was granted orphan drug exclusivity (ODE) in April 2022 for this specific patient population.
Major players in the rare neurological disease treatment market are placing a strong emphasis on product innovation, including rare disease drug development solutions, to advance therapeutic options and enhance the quality of life for patients affected by rare neurological diseases. Developing treatments for rare neurological diseases necessitates targeted research into specific disease mechanisms and the exploration of innovative therapeutic approaches, such as gene therapies or personalized medicine, to address the complex nature of these conditions. For instance, in February 2023, US-based pharmaceutical company Reata Pharmaceuticals Inc. received approval from the United States Food and Drug Administration (USFDA) for Skyclarys, a drug designed to treat Friedreich’s ataxia, a rare neurological disease. Skyclarys represents an experimental medication addressing unmet needs in treating rare neurological conditions by targeting intricate disease mechanisms. It is undergoing rigorous clinical evaluation for its efficacy and safety profiles.
In February 2024, Harmony Biosciences, a biopharmaceutical company based in the United States, acquired Epygenix Therapeutics, Inc. for an undisclosed sum. The purpose of this acquisition is to improve treatments for rare epilepsy disorders such as Dravet syndrome and Lennox-Gastaut syndrome, thereby expediting the development of innovative therapies to address these unmet medical needs. Epygenix Therapeutics, Inc. is a clinical-stage biopharmaceutical company in the U.S. that focuses on developing and commercializing cutting-edge therapies.
Major companies operating in the rare neurological disease treatment market include Pfizer Inc., Johnson & Johnson Services Inc., F. Hoffmann-La Roche Ltd, Bayer AG, Merck & Co. Inc., AbbVie Inc., Novartis AG, Sanofi-Aventis SA, Bristol-Myers Squibb Company, AstraZeneca PLC, GlaxoSmithKline PLC, Takeda Pharmaceutical Company Limited, Medtronic PLC, Teva Pharmaceutical Industries Ltd., CSL Limited, Biogen Inc., UCB S.A., Eisai Co. Ltd., Otsuka Pharmaceutical Co. Ltd., Jazz Pharmaceuticals PLC, Mitsubishi Tanabe Pharma Corporation, Neurocrine Biosciences Inc., Kedrion Biopharma Inc., Merz Pharma GmbH & Co. KGaA, Harmony Biosciences Holdings Inc., Amicus Therapeutics Inc., Revance Therapeutics Inc., Aquestive Therapeutics Inc., US WorldMeds LLC (Solstice Neurosciences LLC), Stoke Therapeutics Inc., Retrotope Inc.
North America was the largest region in the rare neurological disease’s treatment market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the rare neurological disease treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the rare neurological disease treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
Treatment for rare neurological diseases involves medical interventions and therapies with the goal of managing symptoms, slowing disease progression, and enhancing the quality of life for individuals affected by these uncommon conditions. Rare neurological diseases encompass disorders that affect the nervous system, including the brain, spinal cord, and peripheral nerves.
The primary types of treatment for rare neurological diseases involve drugs, including biologics and small molecules. Biologics, derived from living organisms such as proteins, antibodies, or nucleic acids, play a crucial role in treating rare neurological diseases. Examples include enzyme replacement therapies for lysosomal storage disorders and monoclonal antibodies for immune-mediated neurological disorders. Various therapies, such as cognitive behavioral therapy, interpersonal psychotherapy, and cyberknife, are administered through oral and injectable modes to address specific conditions such as Aicardi syndrome, Aicardi Goutières syndrome, reflex sympathetic dystrophy syndrome, Battaglia-Neri syndrome, Creutzfeldt-Jakob disease, agnosia, and others. Medications are distributed through channels including hospitals, retail pharmacies, drug stores, and online pharmacies.
The rare neurological diseases treatment market research report is one of a series of new reports that provides rare neurological diseases treatment market statistics, including the rare neurological diseases treatment industry's global market size, regional shares, competitors with a rare neurological diseases treatment market share, detailed rare neurological diseases treatment market segments, rare neurological diseases treatment market trends and opportunities, and any further data you may need to thrive in the rare neurological diseases treatment industry. This rare neurological diseases treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The rare neurological disease treatment market consists of revenues earned by entities by providing supportive care services and neurodegenerative research services. The market value includes the value of related goods sold by the service provider or included within the service offering. The rare neurological disease treatment market also includes sales of anticonvulsants, antidepressants, and beta blockers. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
Table of Contents
1. Executive Summary2. Rare Neurological Disease Treatment Market Characteristics3. Rare Neurological Disease Treatment Market Trends and Strategies4. Rare Neurological Disease Treatment Market - Macro Economic Scenario including the impact of Interest Rates, Inflation, Geopolitics and Covid and Recovery on the Market32. Global Rare Neurological Disease Treatment Market Competitive Benchmarking and Dashboard33. Key Mergers and Acquisitions in the Rare Neurological Disease Treatment Market34. Recent Developments in the Rare Neurological Disease Treatment Market
5. Global Rare Neurological Disease Treatment Growth Analysis and Strategic Analysis Framework
6. Rare Neurological Disease Treatment Market Segmentation
7. Rare Neurological Disease Treatment Market Regional and Country Analysis
8. Asia-Pacific Rare Neurological Disease Treatment Market
9. China Rare Neurological Disease Treatment Market
10. India Rare Neurological Disease Treatment Market
11. Japan Rare Neurological Disease Treatment Market
12. Australia Rare Neurological Disease Treatment Market
13. Indonesia Rare Neurological Disease Treatment Market
14. South Korea Rare Neurological Disease Treatment Market
15. Western Europe Rare Neurological Disease Treatment Market
16. UK Rare Neurological Disease Treatment Market
17. Germany Rare Neurological Disease Treatment Market
18. France Rare Neurological Disease Treatment Market
19. Italy Rare Neurological Disease Treatment Market
20. Spain Rare Neurological Disease Treatment Market
21. Eastern Europe Rare Neurological Disease Treatment Market
22. Russia Rare Neurological Disease Treatment Market
23. North America Rare Neurological Disease Treatment Market
24. USA Rare Neurological Disease Treatment Market
25. Canada Rare Neurological Disease Treatment Market
26. South America Rare Neurological Disease Treatment Market
27. Brazil Rare Neurological Disease Treatment Market
28. Middle East Rare Neurological Disease Treatment Market
29. Africa Rare Neurological Disease Treatment Market
30. Rare Neurological Disease Treatment Market Competitive Landscape and Company Profiles
31. Rare Neurological Disease Treatment Market Other Major and Innovative Companies
35. Rare Neurological Disease Treatment Market High Potential Countries, Segments and Strategies
36. Appendix
Executive Summary
Rare Neurological Disease Treatment Global Market Report 2025 provides strategists, marketers and senior management with the critical information they need to assess the market.This report focuses on rare neurological disease treatment market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
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Description
Where is the largest and fastest growing market for rare neurological disease treatment? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The rare neurological disease treatment market global report answers all these questions and many more.The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market’s historic and forecast market growth by geography.
- The market characteristics section of the report defines and explains the market.
- The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
- The forecasts are made after considering the major factors currently impacting the market. These include:
- The forecasts are made after considering the major factors currently impacting the market. These include the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.
- Market segmentations break down the market into sub markets.
- The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth. It covers the growth trajectory of COVID-19 for all regions, key developed countries and major emerging markets.
- The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
- The trends and strategies section analyses the shape of the market as it emerges from the crisis and suggests how companies can grow as the market recovers.
Scope
Markets Covered:
1) By Drug Type: Biologics; Small Molecules2) By Therapy: Cognitive Behavioral Therapy; Interpersonal Psychotherapy; Cyberknife; Other Therapies
3) By Mode of Administration: Oral; Injectable; Other Modes of Administration
4) By Disorders: Aicardi Syndrome; Aicardi-Goutières Syndrome; Reflex Sympathetic Dystrophy Syndrome; Battaglia-Neri Syndrome; Creutzfeldt Jakob Disease; Agnosia; Other Disorders
5) By Distribution Channel: Hospital Pharmacies; Retail Pharmacies and Drug Store; Online Pharmacies
Subsegments:
1) By Biologics: Monoclonal Antibodies; Gene Therapies; Recombinant Proteins2) By Small Molecules: Targeted Small Molecules; Neuroprotective Agents; Anti-inflammatory Small Molecules
Key Companies Mentioned: Pfizer Inc.; Johnson & Johnson Services Inc.; F. Hoffmann-La Roche Ltd; Bayer AG; Merck & Co. Inc.
Countries: Australia; Brazil; China; France; Germany; India; Indonesia; Japan; Russia; South Korea; UK; USA; Canada; Italy; Spain
Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
Time Series: Five years historic and ten years forecast.
Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita.
Data Segmentation: Country and regional historic and forecast data, market share of competitors, market segments.
Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes.
Delivery Format: PDF, Word and Excel Data Dashboard.
Companies Mentioned
- Pfizer Inc.
- Johnson & Johnson Services Inc.
- F. Hoffmann-La Roche Ltd
- Bayer AG
- Merck & Co. Inc.
- AbbVie Inc.
- Novartis AG
- Sanofi-Aventis SA
- Bristol-Myers Squibb Company
- AstraZeneca PLC
- GlaxoSmithKline PLC
- Takeda Pharmaceutical Company Limited
- Medtronic PLC
- Teva Pharmaceutical Industries Ltd.
- CSL Limited
- Biogen Inc.
- UCB S.A.
- Eisai Co. Ltd.
- Otsuka Pharmaceutical Co. Ltd.
- Jazz Pharmaceuticals PLC
- Mitsubishi Tanabe Pharma Corporation
- Neurocrine Biosciences Inc.
- Kedrion Biopharma Inc.
- Merz Pharma GmbH & Co. KGaA
- Harmony Biosciences Holdings Inc.
- Amicus Therapeutics Inc.
- Revance Therapeutics Inc.
- Aquestive Therapeutics Inc.
- US WorldMeds LLC (Solstice Neurosciences LLC)
- Stoke Therapeutics Inc.
- Retrotope Inc.
Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 200 |
| Published | February 2025 |
| Forecast Period | 2025 - 2029 |
| Estimated Market Value ( USD | $ 10.68 Billion |
| Forecasted Market Value ( USD | $ 14.84 Billion |
| Compound Annual Growth Rate | 8.6% |
| Regions Covered | Global |
| No. of Companies Mentioned | 31 |
