Mucopolysaccharidosis Treatment Market Report 2025

The mucopolysaccharidosis treatment market size has grown strongly in recent years. It will grow from $2.4 billion in 2024 to $2.59 billion in 2025 at a compound annual growth rate (CAGR) of 8%. The growth in the historic period can be attributed to enzyme replacement therapy (ERT), improved diagnostic tools, advocacy and awareness, gene therapy development, and healthcare infrastructure development.

The mucopolysaccharidosis treatment market size is expected to see strong growth in the next few years. It will grow to $3.51 billion in 2029 at a compound annual growth rate (CAGR) of 7.9%. The growth in the forecast period can be attributed to advancements in gene editing, regulatory changes and support, patient access initiatives, emerging therapeutic modalities, and precision medicine approaches. Major trends in the forecast period include caregiver support programs, focus on symptom management, education and training initiatives, expanded global access, and advances in biomarker development.

The rising prevalence of various forms of mucopolysaccharidosis is anticipated to boost the growth of the mucopolysaccharidosis treatment market in the future. Mucopolysaccharide is a type of complex carbohydrate that accumulates in body tissues due to the absence of the specific enzyme required for its breakdown. Mucopolysaccharidosis (MPS) therapy helps slow disease progression by reducing glycosaminoglycan (GAG) accumulation, managing symptoms, and preventing complications through various treatment approaches. For example, in July 2024, the Orphanet Journal of Rare Diseases, a peer-reviewed open-access medical journal based in France, reported that the incidence of mucopolysaccharidosis IVa (MPS IVa) is estimated at approximately 1 in 201,000 live births, although this number is believed to vary significantly across populations. Similarly, the birth prevalence of mucopolysaccharidosis VI (MPS VI) is estimated to range from 1 in 43,261 to 1 in 1,505,160, depending on the population. As a result, the increasing prevalence of different forms of mucopolysaccharidosis is expected to drive the mucopolysaccharidosis treatment market.

The rise in healthcare spending is contributing to the expansion of the mucopolysaccharidosis treatment market. Healthcare spending refers to the total financial resources dedicated to the healthcare sector within a particular region, country, or organization. The increase in healthcare spending is being directed toward mucopolysaccharidosis (MPS) treatment, aiming to improve access to specialized therapies, research, and medical infrastructure, thus enhancing the management and outcomes for individuals affected by this rare genetic disorder. For example, in July 2024, the American Medical Association, a U.S.-based professional organization of physicians and medical students, reported that health spending in the United States grew by 4.1% in 2022, reaching $4.5 trillion, or $13,493 per capita. Therefore, the rise in healthcare spending is driving the growth of the mucopolysaccharidosis treatment market moving forward.

Product innovation is an emerging trend gaining traction in the mucopolysaccharidosis treatment market. Leading companies in this market are concentrating on developing new products to maintain their competitive edge. For example, in September 2022, Ultragenyx Pharmaceutical Inc., a U.S.-based biopharmaceutical company, launched Vestronidase alfa (Mepsevii) in Japan for the treatment of Mucopolysaccharidosis Type VII (MPS VII or Sly Syndrome). This enzyme replacement therapy is designed to address the enzyme deficiency in MPS VII, helping break down glycosaminoglycans (GAGs) and reducing their buildup in tissues and organs. Mepsevii improves patients' mobility and respiratory function, offering a transformative treatment for individuals with this rare and progressive disorder. Its approval marks a significant advancement in providing essential care for rare disease patients in Japan.

Major players in the mucopolysaccharidosis treatment market are directing their focus towards cell therapies as a promising avenue for addressing rare genetic disorders. A case in point is ISP-001, developed by Immusoft, a US-based biotechnology company. ISP-001 represents a pioneering approach utilizing cell therapy to administer gene-encoded medicine, targeting the root cause of Mucopolysaccharidosis I (MPS I). In September 2022, Immusoft achieved a significant milestone with the FDA clearance of its Investigational New Drug (IND) application for ISP-001, marking a groundbreaking advancement in engineered B cell therapy for MPS I treatment.

In January 2024, Kyowa Kirin Co., Ltd., a Japan-based pharmaceutical company, acquired Orchard Therapeutics for an undisclosed sum. This acquisition is notable as it merges Kyowa Kirin’s extensive experience in biopharmaceuticals with Orchard’s innovative gene therapy platform, aimed at providing transformative treatments for patients with severe genetic disorders. Orchard Therapeutics, a UK-based biotech company, specializes in treatments for mucopolysaccharidosis, further enhancing the capabilities of both companies in addressing rare genetic diseases.

Major companies operating in the mucopolysaccharidosis treatment market include Pfizer Inc., Johnson & Johnson, Roche Holding AG, Merck & Co. Inc., AbbVie Inc., Novartis AG, Sanofi S.A., Bristol-Myers Squibb Company, GlaxoSmithKline plc, Takeda Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., BioMarin Pharmaceuticals Inc., Mallinckrodt Pharmaceuticals, Green Cross Corporation, Sarepta Therapeutics Inc., Esteve SA, JCR Pharmaceuticals Co. Ltd., Ultragenyx Pharmaceutical Inc., RegenxBio Inc., Sangamo Therapeutics Inc., Audentes Therapeutics Inc., Eidos Therapeutics Inc., Neurogene Inc., Immusoft Corporation, Bioasis Technologies Inc., Paradigm Biopharmaceuticals Ltd., Rimedion Inc., Eloxx Pharmaceuticals Inc., iBio Inc., Abeona Therapeutics Inc.

North America was the largest region in the mucopolysaccharidosis treatment market in 2024. The regions covered in the mucopolysaccharidosis treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the mucopolysaccharidosis treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The treatment of Mucopolysaccharidosis (MPS) involves addressing hereditary illnesses characterized by the body's inability to break down glycosaminoglycan (long sugar chains) due to the deficiency or malfunction of specific enzymes, often lysosomal enzymes.

Two primary types of treatments for mucopolysaccharidosis are enzyme replacement therapy and stem cell therapy. Enzyme replacement therapy is a medical intervention designed for patients with chronic diseases resulting from enzyme deficiency or dysfunction. This approach involves providing replacement enzymes to individuals affected by various forms of mucopolysaccharidosis, including types I, II, IV, VI, and others. These replacement enzymes are administered through intravenous and intracerebroventricular routes of administration. The therapy is implemented in diverse healthcare settings, including hospitals, specialty clinics, and other medical facilities.

The mucopolysaccharidosis treatment market research report is one of a series of new reports that provides mucopolysaccharidosis treatment market statistics, including mucopolysaccharidosis treatment industry global market size, regional shares, competitors with a mucopolysaccharidosis treatment market share, detailed mucopolysaccharidosis treatment market segments, market trends and opportunities, and any further data you may need to thrive in the mucopolysaccharidosis treatment industry. This mucopolysaccharidosis treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

The mucopolysaccharides treatment market includes revenues earned by entities by providing medication services such as enzyme replacement therapy, stem cell transplantation, gene therapy, substrate reduction therapy, and palliative care. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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