MUCOPOLYSACCHARIDOSIS (MPS) TREATMENT MARKET ANALYSIS

The global mucopolysaccharidosis (MPS) treatment market is estimated to be valued at US$ 2.27 Bn in 2023 and is expected to exhibit a CAGR of  10.1% during the forecast period (2023-2030).

Analysts’ Views on Global Mucopolysaccharidosis (MPS) Treatment Market:

Key factors driving revenue growth in the mucopolysaccharidosis (MPS) treatment market are a rise in the cases of Sanfilippo syndrome or Hunter syndrome, also known as mucopolysaccharidosis (MPS), among the population across the globe. Revenue growth in the market is driven by an increase in initiatives taken by both public and private organizations to develop treatments. Rising demand for various medications to treat the symptoms of conditions such as lysosomal storage disease and government assistance in the form of funding for research and development activities have an additional impact on the market.

Figure 1. Global Mucopolysaccharidosis (MPS) Treatment Market Share (%), By Treatment Type, 2023

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Global Mucopolysaccharidosis (MPS) Treatment Market– Driver

Increasing product approvals by regulatory authorities for better treatment outcomes

Increasing product approvals by regulatory authorities for better treatment outcomes is expected to propel the market growth over the forecast period. Key players operating in the market are focusing on obtaining approvals for their advanced therapeutics, and this is expected to drive the market growth over the forecast period. For instance, in June 2021, Biomarine Pharmaceuticals Inc. is a global biotechnology company announced developing and commercializing innovative biopharmaceuticals for rare diseases driven by genetic causes. It had received approval for its Vimizim (elosulfase alfa) from the National Medical Products Administration (NMPA) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in China approved for this condition.

Adoption of inorganic growth strategies by key players

Adoption of inorganic growth strategies by key players such as collaborations and partnerships is expected to drive growth of the global mucopolysaccharidosis (MPS) treatment market over the forecast period. For instance, in May 2020, Ultragenyx Pharmaceutical Inc., which is focused on developing the first-ever approved treatments for rare and ultrarare diseases and moving the rare disease community forward, and Rentschler Fill Solutions, a contract manufacturing organization (CMO) and aseptic fill and finish services expert company, collaborated for the production of injectable Mepsevii (vestronidase alfa), a recombinant form of the human enzyme beta-glucuronidase, to treat patients with mucopolysaccharidosis (MPS) VII, a rare genetic disorder.

Figure 2. Global Mucopolysaccharidosis (MPS) Treatment Market Share (%), by Region, 2023

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Global Mucopolysaccharidosis (MPS) Treatment Market- Regional Analysis

Among regions, North America is estimated to hold a dominant position in the global mucopolysaccharidosis (MPS) treatment market over the forecast period. North America is estimated to hold 34.1% of the market share in 2023. The  mucopolysaccharidosis (MPS) treatment market is expected to witness significant growth, driven by the increasing focus on developing advanced products by companies. Rising product launches and approvals in the North America region are expected to boost the market growth over the forecast period. For instance, in August 2022, REGENXBIO Inc., a clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy, announced its plans to file a Biologics License Application (BLA) in 2024 using the U.S. FDA's accelerated approval pathway for RGX-121 for the treatment of mucopolysaccharidosis Type II (MPS II). In February 2022, Homology Medicines, Inc. is a genetic medicines company, presented data on HMI-203, a gene therapy candidate for the treatment of Hunter syndrome (MPS II), under a phase I dose-escalation clinical study in adults with Mucopolysaccharidosis, at the Annual WorldSymposium Meeting held in U.S.

Global Mucopolysaccharidosis (MPS) Treatment Market – Impact of Coronavirus (COVID-19) Pandemic

As for the impact of COVID-19 on the MPS treatment market, it's important to note that the pandemic has had significant effects on the healthcare industry as a whole. Some potential impacts on the MPS treatment market due to COVID-19 might include:

Delayed Research and Clinical Trials: COVID-19 restrictions and safety concerns might have led to delays in ongoing research and clinical trials for MPS treatments, potentially affecting their development and approval timelines.

Access to Healthcare: The pandemic's strain on healthcare systems might have affected the accessibility of MPS treatments for patients, especially those who required regular in-person care or hospital visits.

Supply Chain Disruptions: COVID-19 has caused disruptions in global supply chains, which might have impacted the availability of MPS medications and other related healthcare products.

Economic Challenges: Economic downturns and financial hardships faced by individuals and governments due to the pandemic could have affected healthcare spending and insurance coverage for MPS treatments.

Global Mucopolysaccharidosis (MPS) Treatment Market Segmentation:

The global mucopolysaccharidosis (MPS) treatment market report is segmented  Treatment Type, MPS Type, End User, and Region.

By Treatment, the market is segmented into enzyme replacement therapy and stem cell therapy. Out of which, the enzyme replacement therapy segment is expected to hold a dominant position in the global mucopolysaccharidosis (MPS) treatment market during the forecast period, and this is attributed to the increasing prevalence of mucopolysaccharidosis (Hunter syndrome).

By MPS Type, the market is segmented into MPS-I, MPS-II, MPS-IV, MPS-VI, MPS-VII, and Others (MPS-III and MPS-IX). Out of which, the MPS-II segment is expected to dominate the market over the forecast period, and this is attributed to the increasing the prevalence of MPS-II coupled with presence of multiple treatment options

By End User, the market is segmented into hospitals, specialty clinics, and others. Out of which, the hospitals segment is expected to dominate the market over the forecast period, and this is attributed increase in hospital expenditure.

Among all the segments, the treatment segment is expected to dominate the market over the forecast period, and this is attributed to the easy availability and continuous approvals remains key factors, which is expected to boost the growth of the segment market over the forecast period.

Global Mucopolysaccharidosis (MPS) Treatment Market Cross Sectional Analysis:

Enzyme replacement therapy (ERT) is a standard of care for several types of rare diseases and consists of replacing the deficient or absent enzyme with a functional recombinant version through intravenous administration in North America. For instance, as per an article published in October 2021, it has been observed that pabinafusp alfa is effective against both the somatic and central nervous system (CNS) symptoms of patients with MPS-II as it can pass through the blood-brain barrier to reach the central nervous tissues. Thus, the high efficacy and safety of the novel enzyme drug are expected to increase its adoption for treating patients with Huntington's syndrome, thereby propelling the market growth.

Global Mucopolysaccharidosis (MPS) Treatment Market: Key Developments

In September 2022, ROBIO Inc., a medical device technology company developing diagnostic equipment for the oncology space, announced that the UK MHRA, Research Ethics Committee (REC), and Health Research Authority (HRA) approved AVROBIO's (Lentiviral-Based Gene Therapy) collaborators for the initiation of the Phase 1/2 clinical trial of investigational autologous hematopoietic stem cell (HSC) gene therapy in infants diagnosed with neuronopathic mucopolysaccharidosis or hunter disease.

In February 2022, Denali Therapeutics Inc., which is dedicated to defeating neurodegenerative diseases through rigorous therapeutic discovery and development, presented new longer-term data from an ongoing phase 1/2 clinical trial of DNL310, an investigational brain-penetrant enzyme replacement therapy intended to treat both the central nervous system and peripheral manifestations of MPS II (hunter syndrome), at the World Symposium.

In June 2022, Gilead Sciences, Inc.,  a research-based biopharmaceutical company focused on the discovery, development, and commercialization of innovative medicines, announced that more than 80 abstracts will be presented at the International Liver Congress (ILC) 2022, placed from June 22-26, 2022. Key oral presentations will include Week 48 primary endpoint data from the Pivotal Phase 3 program of Hepcludex (bulevirtide) evaluating its efficacy and safety for the treatment of hepatitis delta virus (HDV) and the impact of the treatment on patient-reported outcomes.

Global Mucopolysaccharidosis (MPS) Treatment Market: Key Trends

Growing development of enzyme replacement therapy products by companies

The growing development of enzyme replacement therapy products by companies is also likely to increase the availability of ERT therapies in the market, further augmenting Mucopolysaccharidosis (MPS) Treatment Market growth. For  instance, in March 2021, the Ministry of Health, Labour and Welfare (MHLW) approved IZCARGO (pabinafusp alfa 10 mL, intravenous drip infusion) for the treatment of MPS II in Japan. It is a recombinant iduronate-2-sulfatase enzyme replacement therapy (ERT) developed using J-Brain Cargo, a proprietary technology developed by JCR, to deliver therapeutics across the blood-brain barrier (BBB).

Global Mucopolysaccharidosis (MPS) Treatment Market: Restraint

High cost of therapeutics coupled with poor diagnosis

High costs of therapeutics coupled with poor diagnosis are expected to hinder growth of the global mucopolysaccharidosis (MPS) treatment market during the forecast period. Vimizin (elosulfase-alfa) is a treatment for MPS IV (Morquio Syndrome). It was launched in the market at a cost of US$ 380,000 per year. Patients in emerging economies like India cannot afford this treatment because of the high price.

Key market players are therefore focusing on cost-effective treatment.

Global Mucopolysaccharidosis (MPS) Treatment Market- Key Players

Major players operating in the global mucopolysaccharidosis (MPS) treatment market include Takeda Pharmaceutical Company Limited, BioMarin Pharmaceuticals, Inc., Ultragenyx Pharmaceutical Inc., Sarepta Therapeutics, Abeona Therapeutics Inc., Eloxx Pharmaceuticals, Esteve, Immusoft Corporation, Inventiva, GC Pharma, JCR Pharmaceuticals Co Ltd., RegenxBio Inc., Sangamo Therapeutics, Inc., Bioasis Technologies Inc., and Paradigm Biopharmaceuticals Ltd.

*Definition: Mucopolysaccharidosis refers to a group of inherited conditions in which the body is unable to properly breakdown mucopolysaccharides, a long chain of sugar molecules that are found in the body. As a result, these sugars build up in cells, blood, and connective tissues, which can lead to several health problems. There are seven forms of mucopolysaccharidosis. Generally, most of the affected people appear healthy at birth and experience a period of normal development, followed by a decline in physical or mental function. As the condition progresses, it may affect appearance, physical abilities, organ and system functioning, and in most cases, cognitive development. The underlying genetic cause varies by form.