Global Hurler Syndrome Treatment Market - 2025-2033

The Global Hurler Syndrome Treatment Market reached US$ 377.65 million in 2024 and is expected to reach US$ 529.34 million by 2033, growing at a CAGR of 3.9% during the forecast period 2025-2033.

Hurler syndrome is also known as mucopolysaccharidosis (MPS I), one of the eleven mucopolysaccharidoses (MPS) disorders. It was formerly referred to as gargoylism. It is caused by an inherited lysosomal problem due to a genetic defect. There is no alpha L iduronidase, an enzyme that affects the destruction process of glycosaminoglycans (GAGs or mucopolysaccharides).

Market Dynamics: Drivers & Restraints

Growing adoption of Enzyme Replacement Therapy (ERT)

The global Hurler syndrome treatment market is thriving due to the increasing adoption of enzyme replacement therapy (ERT). ERT slows disease progression by supplementing deficient enzymes, improving patient survival and quality of life. Advancements in enzyme formulations, government support, orphan drug incentives, and healthcare investments are making ERT more accessible. As pharmaceutical companies focus on optimizing enzyme therapies, the market expands.

Enzyme replacement therapy (ERT) can alleviate Hurler syndrome symptoms by breaking down glycosaminoglycans in the body. However, ERT cannot cross the blood-brain barrier to reach the developing brain, causing neurocognitive decline and intellectual disability. This is because ERT cannot address brain function, leaving patients at risk for neurocognitive decline and intellectual disability.

Limited Availability of Treatment Options in Developing Regions

Availability of medical care is often sparse in underdeveloped or developing countries. This usually implies that the market of Hurler syndrome in these areas is also limited since there are minimum treatment options to save lives. For example, enzyme replacement therapy (ERT) and haematopoietic or bone marrow transplantation (HSCT), are the most effective treatments for the condition may not be readily available in these countries. However, in most of the poor countries, the required health care infrastructure and professionals capable of carrying out treatment or making the diagnosis promptly may not be able to be staged as a result of the demand pattern just highlighted.

Segment Analysis

The global hurler syndrome treatment market is segmented based on treatment type, Age Group, end user, and region.

Treatment Type:

Enzyme Replacement Therapy (ERT) in the treatment type segment is expected to dominate the market with the highest share.

As of now, only one enzyme replacement therapy, i.e., laronidase, is approved for the treatment of Hurler syndrome. Sold under the brand name Aldurazyme, manufactured by Sanofi, this therapy has been available in the market since 2003 across 75 countries. As compared to other treatment options, which are mostly experimental, enzyme replacement therapy turned out to be the most promising approach for the treatment of Hurler syndrome. By addressing the underlying enzymatic deficiency associated with the condition, Aldurazyme has become a long-term care option for the patient population. The drug has generated sales of 321 million in 2024, majorly generating sales from recurring prescriptions. Moreover, several enzyme replacement therapies are in the clinical pipeline, which are anticipated to make market entry in the forecast period.

Geographical Analysis

North America is expected to hold a significant position in the Hurler Syndrome Treatment market share

North America holds a substantial position in the hurler syndrome treatment market due to advanced healthcare infrastructure and the presence of approved therapies. The United States sees a very dominant role, owing to the increase in government initiatives and the wide range of orphan drug designations encouraging innovative product launches.

For instance, in November 2023, Orchard Therapeutics received Fast Track designation from the FDA for OTL-203, an investigational hematopoietic stem cell gene therapy for the treatment of the Hurler subtype of mucopolysaccharidosis. This designation aims to expedite the development of medicines that address serious medical needs, potentially allowing for quicker submission and review timelines for therapeutic candidates. Once the product is launched, the population in the U.S. may get early market access to this therapy and benefit from it.

Moreover, the only approved drug for hurler syndrome, i.e., Aldurazyme (laronidase) by Sanofi, has been widely available in the U.S. since 2003, and the company generates major sales from the country.

Competitive Landscape

The major global players in the Hurler Syndrome Treatment market include Sanofi and BioMarin, among others.

Various other emerging players include, Orchard Therapeutics, JCR Pharmaceuticals, Immusoft, Armagen, Regenxbio, Paradigm Biopharmaceuticals Ltd, and others.

Key Developments
• In February 2024, Orchard Therapeutics, acquired by Kyowa Kirin, has randomized the first patient in a registrational trial at M Health Fairview Masonic Children's Hospital to evaluate the efficacy and safety of OTL-203, an investigational hematopoietic stem cell gene therapy, in patients with Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares OTL-203 treatment to standard care with allogeneic hematopoietic stem cell transplant (HSCT) and is expected to enroll 40 MPS-IH patients.
• In December 2023, Orchard Therapeutics, acquired by Kyowa Kirin, has randomized the first patient in a registrational trial at M Health Fairview Masonic Children's Hospital to evaluate the efficacy and safety of OTL-203, an investigational hematopoietic stem cell gene therapy, in patients with Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares OTL-203 treatment to standard care with allogeneic hematopoietic stem cell transplant (HSCT) and is expected to enroll 40 MPS-IH patients.

Why Purchase the Report?
• Pipeline & Innovations: Reviews ongoing clinical trials, product pipelines, and forecasts upcoming advancements in medical devices and pharmaceuticals.
• Product Performance & Market Positioning: Analyze product performance, market positioning, and growth potential to optimize strategies.
• Real-World Evidence: Integrates patient feedback and data into product development for improved outcomes.
• Physician Preferences & Health System Impact: Examines healthcare provider behaviors and the impact of health system mergers on adoption strategies.
• Market Updates & Industry Changes: This covers recent regulatory changes, new policies, and emerging technologies.
• Competitive Strategies: Analyze competitor strategies, market share, and emerging players.
• Pricing & Market Access: Reviews pricing models, reimbursement trends, and market access strategies.
• Market Entry & Expansion: Identifies optimal strategies for entering new markets and partnerships.
• Regional Growth & Investment: Highlights high-growth regions and investment opportunities.
• Supply Chain Optimization: Assesses supply chain risks and distribution strategies for efficient product delivery.
• Sustainability & Regulatory Impact: Focuses on eco-friendly practices and evolving regulations in healthcare.
• Post-market Surveillance: Uses post-market data to enhance product safety and access.
• Pharmacoeconomics & Value-Based Pricing: Analyzes the shift to value-based pricing and data-driven decision-making in R&D.

The global Hurler Syndrome Treatment market report delivers a detailed analysis with 61 key tables, more than 59 visually impactful figures, and 220 pages of expert insights, providing a complete view of the market landscape.

Target Audience 2024
• Manufacturers: Pharmaceutical, Medical Device, Biotech Companies, Contract Manufacturers, Distributors, Hospitals.
• Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
• Treatment Type & Innovation: AI/Robotics Providers, R&D Professionals, Clinical Trial Managers, Pharmacovigilance Experts.
• Investors: Healthcare Investors, Venture Fund Investors, Pharma Marketing & Sales.
• Consulting & Advisory: Healthcare Consultants, Industry Associations, Analysts.
• Supply Chain: Distribution and Supply Chain Managers.
• Consumers & Advocacy: Patients, Advocacy Groups, Insurance Companies.
• Academic & Research: Academic Institutions.