According to a new report published by Introspective Market Research, titled, Morquio Syndrome (MPS-IV) Drug Market by Type, End User, and Region, The Global Morquio Syndrome (MPS-IV) Drug Market Size Was Valued at USD 1.69 Billion in 2023 and is Projected to Reach USD 4.8 Billion by 2032, Growing at a CAGR of 12.4%. The Morquio Syndrome (MPS-IV) Drug Market is witnessing strong growth due to advancements in enzyme replacement and gene therapy research. Morquio Syndrome, or mucopolysaccharidosis type IV, is a rare genetic disease caused by a deficiency in the GALNS or beta-galactosidase enzyme, leading to the accumulation of glycosaminoglycans in cells and tissues. This results in skeletal abnormalities, joint issues, and respiratory complications.
Rising genomic research, orphan drug incentives, and growing awareness of rare diseases are key factors fueling the market’s expansion. Pharmaceutical innovations and clinical pipelines from major firms such as BioMarin, JCR Pharmaceuticals, and Sanofi are significantly contributing to treatment accessibility for patients across North America, Europe, and Asia Pacific.
The Morquio Syndrome (MPS-IV) Drug Market is segmented into Type, End User, and Region.
By Type, the market is categorized into Enzyme Replacement Therapy (ERT), Gene Therapy, and Others.
By End User, the market is categorized into Hospitals, Specialty Clinics, and Research Institutes.
By Region, the market is categorized into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa.
The primary driver for the Morquio Syndrome (MPS-IV) Drug Market is the increased adoption of enzyme replacement therapies and orphan drug designations. BioMarin’s VIMIZIM (elosulfase alpha) remains the leading FDA-approved drug that has transformed disease management by improving patients' physical functioning and quality of life. Orphan drug policies, tax incentives, and R&D funding for rare disorders are amplifying pharmaceutical interest and investment in the treatment landscape.
A key opportunity lies in the rapid development of gene therapies and advanced biomanufacturing platforms that promise longer-lasting effects and fewer infusions. Emerging CRISPR-based technologies and AI-driven prognostic tools for patient stratification have opened new possibilities for precision treatment. Expansion into developing regions, aided by telemedicine and patient-assistance programs, presents additional potential for international market penetration.
Morquio Syndrome (MPS-IV) Drug Market, Segmentation
The Morquio Syndrome (MPS-IV) Drug Market is segmented on the basis of Type, End User, and Region.
Type
The Type segment is further classified into Enzyme Replacement Therapy (ERT), Gene Therapy, and Others. Among these, the Enzyme Replacement Therapy (ERT) segment accounted for the highest market share in 2023. The ERT segment’s dominance arises from the success of VIMIZIM (elosulfase alpha) and the consistent therapeutic performance it offers across pediatric and adult populations. Its combination with supportive therapies has extended patient lifespan and functional activity levels.
End User
The End User segment is further classified into Hospitals, Specialty Clinics, and Research Institutes. Among these, the Hospitals segment accounted for the highest market share in 2023. Hospitals are primary centers for diagnosis, treatment infusion, and post-therapy monitoring of patients with Morquio Syndrome. The expansion of specialized care units and multidisciplinary rare-disease clinics supports greater adoption of advanced treatment regimens.
Some of The Leading/Active Market Players Are–
- BioMarin Pharmaceutical (U.S.)
- Shire plc (U.K.)
- Sanofi (France)
- Genzyme Corporation (U.S.)
- JCR Pharmaceuticals Co., Ltd. (Japan)
- Ultragenyx Pharmaceutical Inc. (U.S.)
- Sangamo Therapeutics (U.S.)
- ArmaGen Inc. (U.S.)
- Alexion Pharmaceuticals Inc. (U.S.)
- GREEN CROSS Corporation (South Korea)
- Esteve Pharmaceuticals (Spain)
- MandalMed Inc. (U.S.)
- Concert Pharmaceuticals Inc. (U.S.)
- BioStrategies LC (U.S.)
- Other active players.
Key Industry Developments
In July 2025, BioMarin announced the clinical evolution of next-generation ERT molecules targeting improved central nervous system delivery for Morquio A treatment. The development focuses on enhancing enzyme permeability across the blood-brain barrier, addressing neurological symptoms previously unmanaged by existing therapies.
In February 2025, JCR Pharmaceuticals and Sanofi launched a joint research program exploring gene-editing platforms to integrate enzyme manufacturing directly through patient-derived vectors. This collaboration aims to reduce treatment frequency and cost, revolutionizing long-term disease management.
Key Findings of the Study
- Enzyme Replacement Therapy (ERT) was the dominant treatment type in 2023.
- North America accounted for the largest market share in 2023.
- Ongoing advancements in gene therapy are driving market growth.
- Strategic collaborations and orphan drug designations are fostering innovation and investment.
- Growing telemedicine support is expanding treatment accessibility in emerging markets.
