Summary

Overview
The Global neurometabolic disorders market reached US$ XX million in 2023 and is expected to reach US$ XX million by 2031, growing at a CAGR of XX% during the forecast period 2024-2031.
Neurometabolic disorders are a group of genetic conditions in which a specific enzyme deficiency is characterized by the dysregulated metabolism of the food ingested. The enzyme function is lost due to genetic mutations, and the production of metabolites from the food that are necessary for brain function is down-regulated. This leads to several types of neurometabolic disorders with varying symptoms. Neurometabolic disorders are primarily seen in children, and in rare cases, adult-onset can be seen. Some of the common neurometabolic disorders include phenylketonuria- a disorder of amino acid metabolism, adrenoleukodystrophy- a disorder of peroxisome, and tay-sachs disease – liposomal disorders, etc.
Market Dynamics: Drivers
Rising innovations in products and product approvals
The treatment of neurometabolic disorders usually involves following a diet, vitamin therapy, enzyme replacement therapy, gene therapy, etc. Unlike other neurological disorders, neurometabolic disorders can be treatable. The goal of the treatment usually includes restoring the enzyme activity, reducing the metabolite accumulation, and substitution of deficient components. In recent times, innovations have been made in the treatment of these conditions which involve specifically targeting the gene responsible for enzyme mutations. Several products have been tested and recently got approval from the regulatory bodies to use in clinical settings. These innovative therapies are expected to boost the market growth in the forecast period, as they have opened a new avenue in neurometabolic disorders treatment.
For instance, on March 18, 2024, the U.S. Food and Drug Administration approved the first-ever gene therapy designed for the treatment of a rare neurometabolic disorder called metachromatic leukodystrophy (MLD). The drug Lenmeldy (atidarsagene autotemcel) developed by Orchard Therapeutics plc. is a single-dose compound made by genetically modifying the autologous hematopoietic stem cells.
On July 20, 2022, PTC Therapeutics, Inc. statedthat the European Commission has given the marketing approval for one of its gene therapy products Upstaza. This drug is specifically indicated for aromatic L-amino acid decarboxylase (AADC) deficiency patients who are 18 months or older. This gene therapy drug for neurometabolic disorders is expected to revolutionize the treatment strategy in the upcoming years.
Moreover, the rising advances in diagnostic procedures, rising investments in research and development activities are expected to further boost the market growth.
Restraints
Factors such as the high cost associated with the treatment and less awareness regarding the disease pathophysiology are expected to restrain the market growth.
Segment Analysis
The global neurometabolic disorders market is segmented based on type, treatment type, age group, end user, and region.
The enzyme replacement therapy in the type segment accounted for approximately 56.4% of the global neurometabolic disorders market share
Enzyme replacement therapy has been the mainstay treatment option for several neurometabolic disorders. This therapy aims at supplementing the enzyme which is faulty and unable to carry out its normal physiological function. Enzyme replacement therapy is widely preferred and also approved by regulatory bodies such as the FDA and EU for several highly prevalent and most common neurometabolic disorders such as phenylketonuria, Gauchers disease, Fabre disease, Pompe disease, etc.
For instance, on August 24, 2023, a cross-sectional study published in the Journal of Tropical Pediatrics evaluating the experiences of healthcare professionals in managing neurometabolic conditions in children found that 69% of healthcare workers accepted enzyme replacement therapy as a primary treatment option for inherited metabolic conditions.
Moreover, several manufacturers have been currently developing enzyme replacement therapies targeting specific types of neurometabolic conditions. Some of these drugs are recently launched in the market and some are expected to get marketing authorization in the coming years. This is expected to further strengthen the market hold of enzyme replacement therapy.
For instance, on January 18, 2023, a Chinese-based biopharmaceutical company announced that it has started phase 2 clinical trials of CAN103 which is aimed at the treatment of Gaucher disease. CAN103 is an enzyme replacement therapy (ERT) being developed by CANbridge as part of a rare illness collaboration with WuXi Biologics.
Although gene therapy is currently revolutionizing the neurometabolic disorder treatment market, its high cost, limited availability, and lack of access in low-middle-income countries are expected to limit its market share. Enzyme replacement therapy along with diet modifications has long been followed for these conditions.
Geographical Analysis
North America is expected to dominate the market with 45.3% share in the global neurometabolic disorders market
North America is expected to dominate the market due to the strong presence of market leaders in the region. Moreover, the initiatives taken by regulatory organizations and government agencies to address neurometabolic diseases are a critical component that propels the region to lead the market. With well-developed healthcare facilities and the availability of diagnostic centers all across the region, the patients benefit from timely diagnosis and treatment.
For instance, on September 29, 2023, the U.S. Food and Drug Administration launched a pilot program to help accelerate the discovery of new drugs and biologicals for rare diseases. The program aims at providing frequent advice and communication to sponsors of products currently in clinical trials.
Also, on May 10, 2023, the United States Food and Drug Administration approved CHIESI Farmaceutici S.p.A.'s new enzyme replacement medicine PRX-102 for the treatment of adult patients with Fabry disease. After a 7.5-year research to investigate safety, effectiveness, and tolerability, PRX-102 has hit the market.
COVID-19 Impact Analysis
The global market for neurometabolic disorders was affected by the COVID-19 pandemic. The continuity of care needed by patients with rare conditions has been majorly affected during the pandemic. Due to imposed lockdowns, decreased access to healthcare services and rehabilitation services needed by the patients have been limited. Although telemedicine has been implemented in various regions to avoid face-to-face consultations, access to medications was affected.
For instance, on February 24, 2024, an evidence-based study published in the National Center for Biotechnology Information, stated that patients with phenylketonuria (PKU) and rare metabolic disorders were greatly affected by the pandemic due to limited access to routine care. During the first phase of the COVID-19 pandemic, patients in the United Kingdom had a 12-week hiatus in their home enzyme replacement treatment (ERT) infusions. Despite the challenges, many European countries have introduced telemedicine facilities to meet patients' demands and ensure that they adhere to therapy.
Market Segmentation
By Type
• Phenylketonuria
• Adrenoleukodystrophy
• Gauchers Disease
• Fabre Disease
• Pompe Disease
• Others
By Treatment Type
• Enzyme Replacement Therapy
• Cofactor Replacement Therapy
• Gene Therapy
• Vitamin Therapy
• Others
By Age Group
• Pediatrics
• Adults
By End Users
• Hospitals & Clinics
• Pediatric Care Centers
• Specialty Clinics
• Homecare
• Others
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 players in the neurometabolic disorders market include Pfizer Inc., Novartis AG, Zydus Pharmaceuticals, Inc., Teva Pharmaceuticals USA, Inc., Azurity Pharmaceuticals, Inc., Sanofi, Sun Pharmaceutical Industries Ltd., Bausch Health Companies Inc., Cipla Inc., and Aspen Pharmacure Australia Pty Ltd among others.
Key Developments
 On October 26, 2023, Sanofi presented the data at the 28th Annual Congress of the World Muscle Society (WMS) in the U.S., building a substantial body of evidence supporting the use of Nexviazyme to treat a diverse range of Pompe disease patients in a variety of clinical settings. Nexviazyme is currently marketed in the U.S. for the treatment of late-onset Pompe disease and it is being evaluated in phase 3 studies for infantile-onset Pompe disease.
 On September 28, 2023, Amicus Therapeutics announced that the United States Food and Drug Administration (FDA) had authorized Pombiliti + Opfolda 65mg capsules. This combination therapy is aimed at treating people with late-onset Pompe disease who are not responding to their current enzyme replacement therapy (ERT).
 On March 30, 2022, Orchard Therapeutics plc. Has said that, the company will invest in the development of hematopoietic-based stem cell gene therapy for severe neurometabolic disorders. Apart from this biological drug, the company has been developing 3 other drugs that focus on various types of neurometabolic disorders.

Why Purchase the Report?
• To visualize the global neurometabolic disorders market segmentation based on type, age group, end user 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 neurometabolic disorders 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 neurometabolic disorders market report would provide approximately 62 tables, 55 figures, and 187 Pages.
Target Audience 2024
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

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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 Type
3.2. Snippet by Treatment Type
3.3. Snippet by Age Group
3.4. Snippet by End User
4. Dynamics
4.1. Impacting Factors
4.1.1. Drivers
4.1.1.1. Rising innovations in products and product approvals
4.1.1.2. Rising advances in diagnostic procedures
4.1.2. Restraints
4.1.2.1. High cost associated with the 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
5.5. Unmet Needs
5.6. PESTEL Analysis
5.7. Patent Analysis
5.8. SWOT Analysis
6. COVID-19 Analysis
6.1. Analysis of COVID-19
6.1.1. Scenario Before COVID
6.1.2. Scenario During COVID
6.1.3. Scenario Post COVID
6.2. Pricing Dynamics Amid COVID-19
6.3. Demand-Supply Spectrum
6.4. Government Initiatives Related to the Market During Pandemic
6.5. Manufacturers Strategic Initiatives
6.6. Conclusion
7. By Type
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
7.1.2. Market Attractiveness Index, By Type
7.2. Phenylketonuria*
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. Adrenoleukodystrophy
7.4. Gaucher’s Disease
7.5. Fabre Disease
7.6. Pompe Disease
7.7. Others
8. By Treatment Type
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
8.1.2. Market Attractiveness Index, By Treatment Type
8.2. Enzyme Replacement Therapy*
8.2.1. Introduction
8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
8.3. Cofactor Replacement Therapy
8.4. Gene Therapy
8.5. Vitamin Therapy
8.6. Others
9. By Age Group
9.1. Introduction
9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
9.1.2. Market Attractiveness Index, By Age Group
9.2. Pediatrics*
9.2.1. Introduction
9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
9.3. Adults
10. By End Users
10.1. Introduction
10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End Users
10.1.2. Market Attractiveness Index, By End Users
10.2. Hospitals & Clinics*
10.2.1. Introduction
10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
10.3. Pediatric Care Centers
10.4. Specialty Clinics
10.5. Homecare
10.6. Others
11. Competitive Landscape
11.1. Competitive Scenario
11.2. Market Positioning/Share Analysis
11.3. Mergers and Acquisitions Analysis
12. Company Profiles
12.1. BioMarin Pharmaceutical Inc.
12.1.1. Company Overview
12.1.2. Product Portfolio and Description
12.1.3. Financial Overview
12.1.4. Key Developments
12.2. Sanofi
12.3. Takeda Pharmaceuticals U.S.A., Inc.
12.4. Pfizer Inc.
12.5. Actelion Pharmaceuticals US, Inc.
12.6. Bluebird Bio, Inc.
12.7. Orchard Therapeutics plc
12.8. Amicus Therapeutics, Inc.
12.9. CHIESI Farmaceutici S.p.A.
12.10. Sentynl Therapeutics, Inc.
LIST NOT EXHAUSTIVE
13. Appendix
13.1. About Us and Services
13.2. Contact Us