The global neurometabolic disorders market size was estimated to be USD 71.85 billion in 2023 and is expected to reach USD 126.94 billion by 2034 with a CAGR of 5.31% during the forecast period 2024-2034. Increasing incidence and awareness, advancements in research and development, government initiatives and support, collaborations and partnerships, orphan drug designation and incentives, technological advances in diagnostics, and collaborative efforts will drive market growth.
New treatment methods are being developed as a result of ongoing research efforts to comprehend the underlying genetic and biochemical processes of neurometabolic diseases. Increased possibilities are a result of advancements in gene therapy, enzyme replacement treatments, and other therapeutic techniques.
By disease type, the Gaucher's disease segment accounted for the highest revenue-grossing segment in the global neurometabolic disorders market in 2023 owing to the significant prevalence of Gaucher's disease, the availability of effective therapies, and increased awareness among healthcare professionals and the general population. For instance, the FDA approved Avalglucosidase alfa (Zavesca) in August 2023 to treat Pompe disease, therefore broadening their product line. Additionally, the Pompe disease segment is predicted to grow at the fastest CAGR during the forecast period owing to the increased understanding of Pompe disease's genetic and biochemical basis, ongoing advancements in therapeutic interventions such as enzyme replacement therapy (ERT), and a rising emphasis on early diagnosis and intervention.
By route of administration, the parenteral segment accounted for the highest revenue-grossing segment in the global neurometabolic disorders market in 2023 owing to the increased prevalence of severe neurometabolic disorders necessitating precise and rapid administration of therapies, the preference for direct and efficient drug delivery, and advancements in parenteral formulations offering improved bioavailability and therapeutic outcomes. For instance, BioMarin Pharmaceutical Inc. expanded into new illnesses when the FDA approved Vimizim (elapraseptal) in June 2023 for the treatment of Morquio A Syndrome. Additionally, the oral segment is predicted to grow at the fastest CAGR during the forecast period owing to the increasing emphasis on patient convenience and adherence, advancements in oral drug formulations offering enhanced bioavailability and efficacy, and ongoing developments in oral therapies for neurometabolic disorders.
By end-user, the hospitals and clinics segment accounted for the highest revenue-grossing segment in the global neurometabolic disorders market in 2023 owing to the central role of hospitals and clinics in the diagnosis, treatment, and management of neurometabolic disorders, the availability of specialized medical professionals and infrastructure for comprehensive patient care, and the increasing number of patients seeking medical attention for these rare genetic conditions. For instance, Amicus Therapeutics, a prospective gene therapy method, announced encouraging Phase 1/2 study findings for AT-GAA for Pompe Disease in October 2023. Additionally, the research institutes segment is predicted to grow at the fastest CAGR during the forecast period owing to the escalating focus on advancing scientific understanding of neurometabolic disorders, fostering collaborative research initiatives, and driving innovation in the development of novel therapeutic approaches.
North American region is anticipated to have the highest revenue share during the forecast period owing to the robust healthcare infrastructure, a high prevalence of neurometabolic disorders, advanced research and development activities, and increased awareness among healthcare professionals and the general population. Additionally, the Asia Pacific region is predicted to grow at the fastest CAGR during the forecast period owing to increasing awareness, improving healthcare infrastructure, rising healthcare expenditures, and a growing focus on rare disease research and treatment. For instance, Vertex Pharmaceuticals and CRISPR Therapeutics announced in September 2023 that they will be working together to develop gene-editing medicines for several rare disorders, including MPS IIIA.
This comprehensive research report focuses on the global and regional market size and forecasts from 2023 to 2034.
New treatment methods are being developed as a result of ongoing research efforts to comprehend the underlying genetic and biochemical processes of neurometabolic diseases. Increased possibilities are a result of advancements in gene therapy, enzyme replacement treatments, and other therapeutic techniques.
By disease type, the Gaucher's disease segment accounted for the highest revenue-grossing segment in the global neurometabolic disorders market in 2023 owing to the significant prevalence of Gaucher's disease, the availability of effective therapies, and increased awareness among healthcare professionals and the general population. For instance, the FDA approved Avalglucosidase alfa (Zavesca) in August 2023 to treat Pompe disease, therefore broadening their product line. Additionally, the Pompe disease segment is predicted to grow at the fastest CAGR during the forecast period owing to the increased understanding of Pompe disease's genetic and biochemical basis, ongoing advancements in therapeutic interventions such as enzyme replacement therapy (ERT), and a rising emphasis on early diagnosis and intervention.
By route of administration, the parenteral segment accounted for the highest revenue-grossing segment in the global neurometabolic disorders market in 2023 owing to the increased prevalence of severe neurometabolic disorders necessitating precise and rapid administration of therapies, the preference for direct and efficient drug delivery, and advancements in parenteral formulations offering improved bioavailability and therapeutic outcomes. For instance, BioMarin Pharmaceutical Inc. expanded into new illnesses when the FDA approved Vimizim (elapraseptal) in June 2023 for the treatment of Morquio A Syndrome. Additionally, the oral segment is predicted to grow at the fastest CAGR during the forecast period owing to the increasing emphasis on patient convenience and adherence, advancements in oral drug formulations offering enhanced bioavailability and efficacy, and ongoing developments in oral therapies for neurometabolic disorders.
By end-user, the hospitals and clinics segment accounted for the highest revenue-grossing segment in the global neurometabolic disorders market in 2023 owing to the central role of hospitals and clinics in the diagnosis, treatment, and management of neurometabolic disorders, the availability of specialized medical professionals and infrastructure for comprehensive patient care, and the increasing number of patients seeking medical attention for these rare genetic conditions. For instance, Amicus Therapeutics, a prospective gene therapy method, announced encouraging Phase 1/2 study findings for AT-GAA for Pompe Disease in October 2023. Additionally, the research institutes segment is predicted to grow at the fastest CAGR during the forecast period owing to the escalating focus on advancing scientific understanding of neurometabolic disorders, fostering collaborative research initiatives, and driving innovation in the development of novel therapeutic approaches.
North American region is anticipated to have the highest revenue share during the forecast period owing to the robust healthcare infrastructure, a high prevalence of neurometabolic disorders, advanced research and development activities, and increased awareness among healthcare professionals and the general population. Additionally, the Asia Pacific region is predicted to grow at the fastest CAGR during the forecast period owing to increasing awareness, improving healthcare infrastructure, rising healthcare expenditures, and a growing focus on rare disease research and treatment. For instance, Vertex Pharmaceuticals and CRISPR Therapeutics announced in September 2023 that they will be working together to develop gene-editing medicines for several rare disorders, including MPS IIIA.
This comprehensive research report focuses on the global and regional market size and forecasts from 2023 to 2034.
Report Scope:
- Base Year: 2023
- Forecast Period: 2024-2034
- Study Coverage
- Market Forecast by Disease Type, Route of Administration, and End-User
- Market Forecast for 5 Regions and 17+ Countries
- North America (U.S. and Canada)
- Europe (Germany, France, UK, Spain, Italy, Russia, Rest of Europe)
- Asia Pacific (China, Japan, India, Australia, South Korea, Rest of APAC)
- Latin America (Brazil, Mexico, Argentina, Rest of LATAM)
- MEA (South Africa, GCC, Rest of MEA)
- Exhaustive Company Profiles of the Top 10+ Major Market Players
- 20% Free Customization Available to Meet Your Exact Requirement.
Segmentation: Neurometabolic Disorders Market Report 2023 - 2034
Neurometabolic Disorders Market Analysis & Forecast by Disease Type 2023 - 2034 (Revenue USD Bn)
- Fabry Disease
- Gaucher's Disease
- Pompe Disease
- Maroteaux-Lamy Syndrome
- Others
Neurometabolic Disorders Market Analysis & Forecast by Route of Administration 2023 - 2034 (Revenue USD Bn)
- Parenteral
- Oral
Neurometabolic Disorders Market Analysis & Forecast by End-User 2023 - 2034 (Revenue USD Bn)
- Hospitals and Clinics
- Research Institutes
- Pharmaceutical Companies
Neurometabolic Disorders Market Analysis & Forecast by Region 2023 - 2034 (Revenue USD Bn)
- North America
- U.S.
- Canada
- Europe
- Germany
- France
- UK
- Spain
- Italy
- Rest of Europe
- Asia Pacific
- China
- Japan
- India
- Australia
- South Korea
- Rest of APAC
- Latin America
- Brazil
- Mexico
- Argentina
- Rest of LATAM
- Middle East & Africa
- South Africa
- GCC
- Rest of MEA
Table of Contents
1. Research Methodology
2. Introduction
4. Market Environment Analysis
5. Market Dynamics
7. Neurometabolic Disorders Market: Disease Type Estimates & Trend Analysis
8. Neurometabolic Disorders Market: Route of Administration Estimates & Trend Analysis
9. Neurometabolic Disorders Market: End-User Estimates & Trend Analysis
10. Regional Market Analysis
11. North America Neurometabolic Disorders Market
12. Europe Global Neurometabolic Disorders Market
13. Asia Pacific Global Neurometabolic Disorders Market
14. Latin America Global Neurometabolic Disorders Market
15. MEA Global Neurometabolic Disorders Market
16. Competitor Analysis
17. Company Profiles
Companies Mentioned
- Sanofi Genzyme
- Shire (now part of Takeda)
- Biomarin Pharmaceutical Inc.
- Genzyme Corporation
- Alexion Pharmaceuticals
- Actelion Pharmaceuticals (a part of Johnson & Johnson)
- Amicus Therapeutics
- Teva Pharmaceutical Industries Ltd.
- Vertex Pharmaceuticals
- Ultragenyx Pharmaceutical Inc.
- Recordati Rare Diseases
- Orphazyme
- BioMarck Pharmaceuticals
- Codexis Inc.
- Audentes Therapeutics.
Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 200 |
| Published | March 2024 |
| Forecast Period | 2023 - 2034 |
| Estimated Market Value ( USD | $ 71.85 Billion |
| Forecasted Market Value ( USD | $ 126.94 Billion |
| Compound Annual Growth Rate | 5.3% |
| Regions Covered | Global |
| No. of Companies Mentioned | 15 |
