Morquio Syndrome Therapy Market Grows with Enzyme Replacement Innovations and Orphan Drug Designations | BioMarin, JCR Pharmaceuticals Co., Ltd., Novartis AG, Takeda Pharmaceutical Company Limited.

The global Morquio Syndrome Therapy Market size was valued US$ 1.5 Mn by 2032 in 2024, and is estimated to reach 5.4 million by 2033, growing at a CAGR of 11.62% during the forecast period (2025-2033).

Morquio Syndrome Therapy Market Report by DataM Intelligence offers a detailed assessment of the global pharmaceutical and healthcare landscape, highlighting market size, key players, and critical growth drivers. With increasing attention to rare disease treatments, enzyme and gene therapy innovations, and evolving patient support ecosystems, the report delivers strategic insights for stakeholders in lysosomal disorder therapeutics from 2025 to 2033.

The global therapy market for Morquio Syndrome is expected to grow steadily, across major regions including the U.S. and Japan. In Japan, BioMarin's Vimizim (elosulfase alfa) became the first approved treatment for Morquio A syndrome under the country's Orphan Drug program in 2025. Additionally, new R&D efforts including gene therapy development by Ultragenyx and others-are accelerating to offer potentially curative options

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Morquio syndrome therapy primarily includes enzyme replacement treatments, supportive care, and surgical interventions for this rare lysosomal storage disorder. The market is small but growing, with rising rare disease research funding and orphan drug designations fueling R&D.

Morquio Syndrome Therapy Market Competitors Overview:

BioMarin, JCR Pharmaceuticals Co., Ltd., Novartis AG, Takeda Pharmaceutical Company Limited., Alexion Pharmaceuticals, Inc., Regenxbio Inc., Ultragenyx Pharmaceutical Inc., Abbvie Inc., and Abeona Therapeutics Inc. among others.

Important Industry Updates:

In August 2023, a unique initiative strives to eliminate hindrance, delivering $97 million in funds and expertise to assist investigators in testing gene therapy in individuals with Morquio and seven other ultrarare diseases as soon as next year. The program, a coalition of nonprofits, government, and industry, is directed by the non-profit Foundation for the National Institutes of Health. Furthermore, the growing awareness and prevalence of the condition is also contributing to the regional market growth during the forecast period.

Methodology and Scope

The Morquio Syndrome Therapy Market Report is built on a rigorous research methodology that integrates primary interviews, expert insights, and validated secondary data from company reports, industry publications, and reliable databases. Utilizing both bottom-up and top-down approaches, the analysis ensures high data accuracy through triangulation. The report covers current market dynamics, growth opportunities, and key strategic developments across global and regional markets.

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Morquio Syndrome Therapy Market Segments Overview:

By Type (Enzyme Replacement Therapy, Supportive/Symptomatic Therapy, Others) By Therapy (MPS IV Type A, MPS IV Type B), By Route of Administration (Injectable, Oral, Other), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies, Others)

Regional Overview for Morquio Syndrome Therapy Market:

⇥ North America (U.S., Canada, Mexico)
⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)
⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)
⇥ South America (Colombia, Brazil, Argentina, Rest of South America)
⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

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