Global Morquio Syndrome (MPS-IV) Drug Market, By Product Type (Solid Dosage Form, Liquid Dosage Form), By Treatment (Enzyme Replacement Therapy, Hematopoietic Stem Cell Therapy, Gene Therapy, Others), By Disease Type (Morquio A, Morquio B), By Distributio

Mucopolysaccharidosis type IV (MPS IV) is a rare disease in which the body is missing or does not have enough of an enzyme needed to break down long chains of sugar molecules. These chains of molecules are called glycosaminoglycans (formerly called mucopolysaccharides). It is caused by a deficiency in the enzymes N-acetylgalactosamine-6-sulfatase (GALNS) or beta-galactosidase which is responsible for breaking down keratan sulfate and chondroitin sulfate. The accumulation of these sugars in cells, tissues, and organs can lead to skeletal dysplasia, short stature, joint abnormalities, and respiratory issues like airway obstruction, heart defects, hearing loss, and corneal opacity. There is no cure for MPS-IV and the treatment options are limited to symptom management. However, enzyme replacement therapy (ERT) and hematopoietic stem cell transplant (HSCT) have shown some success in management of symptoms and improvement in quality of life for patients. MPS IV is an inherited disorder. This means, it is typically passed down through families. If both parents carry a nonworking copy of a gene that is related to this condition, each of their children has a 25% (1 in 4) chance of developing the disease. This is called an autosomal recessive trait.

Market Dynamics:

Global morquio syndrome (MPS-IV) drug market is primarily driven by the rising prevalence of this rare genetic disorder, growing awareness about available treatment options and strong pipeline drugs with novel mechanisms of action. However, high cost of therapy due to rarity of disease as well as challenges that are associated with production of recombinant enzymes are some of the factors restraining the market growth. Nonetheless, development of improved treatment modalities, increasing research funding for rare diseases and orphan drug designation providing market exclusivity offers lucrative opportunities for the key market players in this market.

Key features of the study:

• This report provides in-depth analysis of the global morquio syndrome (MPS-IV) drug market, and provides market size (US$ Mn) and compound annual growth rate (CAGR %) for the forecast period (2025–2032), considering 2024 as the base year
• It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by key players
• It profiles key players in the global morquio syndrome (MPS-IV) drug market based on the following parameters – company highlights, products portfolio, key highlights, financial performance, and strategies - Key companies covered as a part of this study include BioMarin Pharmaceutical Inc., Takeda Pharmaceutical Company Limited., Pfizer Inc., Sanofi, Novo Nordisk A/S, Concert Pharmaceuticals, Inc., Alexion Pharmaceuticals Inc., Novartis AG, Amgen Inc., Sangamo Therapeutics, and JCR Pharmaceuticals Co., Ltd.
• Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
• The global morquio syndrome (MPS-IV) drug market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
• Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global morquio syndrome (MPS-IV) drug market

• Global Morquio Syndrome (MPS-IV) Drug Market, By Product Type
• Solid Dosage Form
• Tablets
• Capsules
• Liquid Dosage Form
• Injectable
• Others
• Global Morquio Syndrome (MPS-IV) Drug Market, By Treatment
• Enzyme Replacement Therapy
• Hematopoietic Stem Cell Therapy
• Gene Therapy
• Others
• Global Morquio Syndrome (MPS-IV) Drug Market, By Disease Type
• Morquio A
• Morquio B
• Global Morquio Syndrome (MPS-IV) Drug Market, By Distribution Channel
• Hospital Pharmacies
• Online Pharmacies
• Retail Pharmacies
• Global Morquio Syndrome (MPS-IV) Drug Market, By Region
• North America
• Europe
• Asia Pacific
• Latin America
• Middle East & Africa
• Company Profiles
• BioMarin Pharmaceutical Inc.
• Takeda Pharmaceutical Company Limited.
• Pfizer Inc.
• Sanofi
• Novo Nordisk A/S
• Concert Pharmaceuticals, Inc.
• Alexion Pharmaceuticals Inc.
• Novartis AG
• Amgen Inc.
• Sangamo Therapeutics
• JCR Pharmaceuticals Co., Ltd.