Pharmaceutical Industry Today
Morquio Syndrome Therapy Market Size, Share, Growth and Research Report 2023-2030
Report Overview
The Global Morquio Syndrome Therapy Market reached US$ YY million in 2022 and is expected to reach US$ YY million by 2030, growing at a high CAGR during the forecast period 2023-2030.
Morquio syndrome, also known as MPS IVA (Mucopolysaccharidosis IVA), is a rare genetic disorder that affects a child's bones, spine, organs, and physical abilities. Children with this condition have a deficiency or insufficient production of enzymes responsible for metabolizing sugar chains that are naturally produced in the body. These sugar chains accumulate in cells, blood, tendons, and ligaments, causing wear and tear over time.
In 2014, the FDA approved a recombinant human GALNS enzyme replacement therapy called elosulfase alfa, marketed as Vimizim, for the treatment of MPS IVA. Other therapies for MPS IV are primarily symptomatic and supportive in nature.
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Market Dynamics
The global market for Morquio Syndrome is poised for growth due to recent approvals for existing therapies and the increasing support from various regulatory authorities. For example, in June 2019, BioMarin Pharmaceutical Inc. received approval from the National Medical Products Administration (NMPA) for Vimizim (elosulfase alfa) in the treatment of individuals with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim became the first approved treatment for this condition in China.
Furthermore, in March 2022, the National Institute for Health and Care Excellence (NICE) issued conclusive draft guidance recommending elosulfase alfa (also known as Vimizin and manufactured by BioMarin) for regular use within the NHS for the treatment of mucopolysaccharidosis type 4A (also known as MPS 4A and Morquio A syndrome).
In addition to these regulatory approvals, the growing awareness, increasing prevalence of Morquio Syndrome, and ongoing research activities are anticipated to contribute to the global market's growth during the forecast period.
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Market Segmentation
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
Market Key Players
Major players are 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.
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