PUBLISHER: The Business Research Company | PRODUCT CODE: 1670883
PUBLISHER: The Business Research Company | PRODUCT CODE: 1670883
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 Goutieres 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 from The Business Research Company 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 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.
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.
Rare Neurological Disease Treatment Global Market Report 2025 from The Business Research Company 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.
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 from the Business Research Company 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 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.