Mucopolysaccharidosis Treatment Global Market Report 2024

The mucopolysaccharidosis treatment market size has grown strongly in recent years. It will grow from $2.2 billion in 2023 to $2.38 billion in 2024 at a compound annual growth rate (CAGR) of 8.5%. The growth observed in the historic period can be ascribed to several factors, including the widespread adoption of enzyme replacement therapy (ERT), advancements in diagnostic tools, increased advocacy and awareness initiatives, ongoing developments in gene therapy, and the continuous enhancement of healthcare infrastructure.

The mucopolysaccharidosis treatment market size is expected to see strong growth in the next few years. It will grow to $3.19 billion in 2028 at a compound annual growth rate (CAGR) of 7.5%. The anticipated growth in the forecast period can be attributed to various factors, including significant advancements in gene editing, regulatory changes with increased support, initiatives to improve patient access, the emergence of new therapeutic modalities, and the application of precision medicine approaches. Key trends expected in the forecast period encompass the implementation of caregiver support programs, a heightened focus on symptom management, educational and training initiatives, expanded global access to treatments, and notable progress in the development of biomarkers.

The anticipated growth in the mucopolysaccharidosis treatment market is expected to be propelled by the increasing prevalence of various forms of mucopolysaccharidosis. This group of disorders involves the accumulation of mucopolysaccharides, a complex carbohydrate, in bodily tissues due to a deficiency of specific enzymes required for digestion. Mucopolysaccharidosis (MPS) therapy plays a crucial role in preventing disease progression by reducing glycosaminoglycan (GAG) formation in the body, managing symptoms, and averting complications through diverse therapeutic approaches. As evidenced by a report from the National Library of Medicine in February 2021, the prevalence of MPS I and MPS VI births was reported at 3.62 per 100,000 live births. MPS III constituted 11% of all cases, with a calculated rate of 1.8 per 100,000 live births. This escalating prevalence across various forms of mucopolysaccharidosis is expected to be a significant driver for the mucopolysaccharidosis treatment market.

The growth in healthcare spending is contributing to the expansion of the mucopolysaccharidosis treatment market. Healthcare spending, referring to the total financial resources allocated to the healthcare sector within a specific region or country, is on the rise. This increased spending is directed towards mucopolysaccharidosis (MPS) treatment, facilitating improved access to specialized therapies, research initiatives, and enhanced medical infrastructure. For instance, according to a report by the American Medical Association in March 2023, health spending in the United States increased by 2.7% in 2021, reaching $4.3 trillion, or $12,914 per capita. This upward trend in healthcare spending is expected to drive further growth in the mucopolysaccharidosis treatment market, ultimately enhancing the management and outcomes for individuals affected by this rare genetic disorder.

Product innovation emerges as a pivotal trend gaining momentum in the mucopolysaccharidosis treatment market, with major companies actively pursuing new product developments to maintain their competitive edge. Illustrating this trend is the approval of IZCARGO (Pabinafusp Alfa) by JCR Pharmaceuticals in March 2021 for the treatment of mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, in Japan. This milestone marked the first-ever enzyme replacement therapy (ERT) for MPS II capable of crossing the blood-brain barrier through intravenous administration, leveraging JCR’s proprietary J-Brain Cargo Technology. This innovative product, a recombinant iduronate-2-sulfatase enzyme replacement therapy, holds significant promise for individuals with lysosomal storage disorders such as MPS II.

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 October 2021, Takeda Pharmaceutical Company, a Japan-based pharmaceutical giant, entered into a partnership with JCR Pharmaceutical. This collaboration aims to facilitate the development and commercialization of JR-141, an experimental antibody fusion protein designed to target the human transferrin receptor for treating Hunter syndrome, a subtype of mucopolysaccharidosis. This strategic alliance underscores a concerted effort to expedite the delivery of innovative treatments to patients and enhance their quality of life.

Major companies operating in the mucopolysaccharidosis treatment market report are 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 2023. 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.

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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