PUBLISHER: Global Industry Analysts, Inc. | PRODUCT CODE: 1753130
PUBLISHER: Global Industry Analysts, Inc. | PRODUCT CODE: 1753130
Global Morquio Syndrome Drugs Market to Reach US$3.0 Million by 2030
The global market for Morquio Syndrome Drugs estimated at US$1.6 Million in the year 2024, is expected to reach US$3.0 Million by 2030, growing at a CAGR of 11.1% over the analysis period 2024-2030. Enzyme Replacement Therapy, one of the segments analyzed in the report, is expected to record a 9.3% CAGR and reach US$1.5 Million by the end of the analysis period. Growth in the Hematopoietic Stem Cell Therapy segment is estimated at 13.5% CAGR over the analysis period.
The U.S. Market is Estimated at US$436.0 Thousand While China is Forecast to Grow at 14.8% CAGR
The Morquio Syndrome Drugs market in the U.S. is estimated at US$436.0 Thousand in the year 2024. China, the world's second largest economy, is forecast to reach a projected market size of US$613.8 Thousand by the year 2030 trailing a CAGR of 14.8% over the analysis period 2024-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 8.2% and 9.7% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 8.7% CAGR.
Global Morquio Syndrome Drugs Market - Key Trends & Drivers Summarized
What Is Morquio Syndrome and Why Are Targeted Therapies Critical?
Morquio syndrome, also known as Mucopolysaccharidosis type IV (MPS IV), is a rare autosomal recessive lysosomal storage disorder caused by deficiencies in the GALNS or GLB1 enzymes. This results in the accumulation of glycosaminoglycans (GAGs) in the body, leading to severe skeletal abnormalities, joint dysfunction, vision and hearing impairment, and cardiopulmonary complications. Given the progressive nature and multisystem impact of the disease, drug treatment focuses on enzyme replacement therapy (ERT) and supportive care aimed at slowing progression and managing symptoms.
The ultra-rare status of Morquio syndrome has made it a key focus of orphan drug development efforts. ERT-particularly with elosulfase alfa-has emerged as the primary treatment to address the underlying enzyme deficiency in MPS IV-A. While it does not reverse existing skeletal damage, ERT improves endurance, reduces GAG accumulation, and may enhance respiratory and cardiovascular function over time. In the absence of curative options, timely diagnosis and consistent therapy are essential to prolong mobility and improve quality of life.
What Innovations Are Advancing Therapeutic Efficacy and Access?
Current innovations are centered on improving the delivery, bioavailability, and long-term efficacy of enzyme replacement therapies. Researchers are exploring modified enzymes with longer half-lives and enhanced tissue penetration-especially targeting the skeletal system, which remains poorly responsive to conventional ERT due to vascular limitations. Intrathecal administration methods and fusion protein technologies are being investigated to enhance enzyme access to bone and central nervous system tissues.
Gene therapy is another area of active research, aiming to introduce functional copies of the GALNS or GLB1 gene using viral vectors. Preclinical studies show promise in achieving systemic enzyme expression with potentially fewer dosing requirements. Additionally, substrate reduction therapy (SRT) and chaperone therapy are being explored as adjunct or alternative approaches. These innovations are being enabled by advancements in molecular diagnostics, patient registries, and clinical trial infrastructure for rare diseases.
Which Patient Populations and Geographies Are Driving Demand for Treatment?
Pediatric patients make up the majority of Morquio syndrome cases, with diagnosis typically occurring between ages two and six. Early treatment initiation is associated with better outcomes, particularly in maintaining mobility and slowing cardiopulmonary decline. Demand is most concentrated in North America and Europe, where newborn screening programs, access to genetic testing, and availability of orphan drug reimbursement frameworks support treatment initiation.
In Latin America, the Middle East, and parts of Asia-Pacific, underdiagnosis remains a challenge due to limited awareness and diagnostic infrastructure. However, advocacy groups and international collaborations are increasing patient identification and access to therapies in these regions. The increasing availability of genetic testing and biomarker-based diagnosis is helping identify more patients at earlier stages, driving long-term demand for therapies and supportive care.
The Growth in the Morquio Syndrome Drugs Market Is Driven by Several Factors…
The growth in the Morquio syndrome drugs market is driven by several factors including rising awareness of rare lysosomal storage diseases, improvements in early diagnostic methods, and ongoing innovation in enzyme and gene therapies. Market expansion is supported by orphan drug incentives, favorable regulatory frameworks, and increasing inclusion of Morquio therapies in national rare disease programs. Advances in biomarker monitoring, drug delivery technologies, and real-world data collection are improving clinical outcomes and expanding patient reach. As healthcare systems prioritize earlier intervention and long-term disease management for ultra-rare disorders, demand for novel and more effective Morquio syndrome therapies continues to rise globally.
SCOPE OF STUDY:
The report analyzes the Morquio Syndrome Drugs market in terms of units by the following Segments, and Geographic Regions/Countries:
Segments:
Treatment (Enzyme Replacement Therapy, Hematopoietic Stem Cell Therapy, Gene Therapy, Other Treatments); Disease Type (Morquio A, Morquio B); Form (Solid Form, Liquid Form); Distribution Channel (Hospital Pharmacies, Online Pharmacies, Retail Pharmacies)
Geographic Regions/Countries:
World; United States; Canada; Japan; China; Europe (France; Germany; Italy; United Kingdom; Spain; Russia; and Rest of Europe); Asia-Pacific (Australia; India; South Korea; and Rest of Asia-Pacific); Latin America (Argentina; Brazil; Mexico; and Rest of Latin America); Middle East (Iran; Israel; Saudi Arabia; United Arab Emirates; and Rest of Middle East); and Africa.
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