Global Mucopolysaccharidosis Treatment Market - 2023-2030

Overview

Mucopolysaccharidoses (MPS) are a group of rare lysosomal storage diseases (LSD) with multiorganic and severe symptoms. MPS occur worldwide in various forms though have a low incidence. The prevalent type of MPS varies among different continents, indicating that it may be associated with region and ethnic background. Undegraded glycosaminoglycans (GAGs) induced by deficiency of enzymes are the primary cause of MPS. Clinical features differ depending on the specific enzyme deficiency including coarse facial features, cognitive retardation, hepatosplenomegaly, hernias, kyphoscoliosis, corneal clouding, etc

Mucopolysaccharidoses are differentiated biochemically by their associated enzyme deficiency and can be classified into 7 types, designated MPS I to MPS IX (excluding MPS V and MPS VIII), and some types are further categorized into subtypes. In total, MPS are classified into 11 types and subtypes. The clinical symptoms of MPS differ depending on the specific enzyme deficiency, but major clinical features are neurological symptoms, facial dysmorphism, cardiac and valvular diseases, skeletal dysfunction, respiratory problems, and ocular disorders.

Market Dynamics: Drivers and Restraints

Increasing Disease approvals by regulatory authorities for better treatment outcomes

Increasing disease approvals by regulatory authorities for better treatment outcomes is one of the significant factors that helps the market to grow during 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 August 2022 Regenxbio Inc. announced its intention to file a Biologics License Application (BLA) in 2024 using the FDA's accelerated approval pathway for RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome. The Company also announced that a pivotal program for RGX-121 is active and enrolling patients. RGX-121 is an investigational, one-time AAV Therapeutic using the NAV AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme.

Moreover, in June 2021, BioMarin Pharmaceuticals Pharmaceuticals Inc. 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 Disease 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.

Furthermore, the market is driven by various other factors like advancements in enzyme replacement therapy, increased awareness and early diagnosis, emerging novel therapies, adoption of new strategies by key players and others will further drive the market during the forecast period.

Market Dynamics: Restraint

High costs of therapeutics coupled with poor diagnosis are expected to hinder the 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 IJSS 380,000 per year. Patients in emerging economies like India cannot afford this treatment because of the high price. Also, the lack of awareness about the disease, complications associated with the disease, regulatory challenges and among others also hampers the market growth rate during the forecast period.

Segment Analysis

The global mucopolysaccharidosis treatment is segmented based on disease type, treatment type, end-user and region.

The enzyme replacement therapy from the treatment type segment accounted for approximately 42.2% of the market share

The enzyme replacement therapy from the treatment type segment accounted for approximately 42.2% and it is expected to be dominated during the forecast period. Enzyme replacement therapy refers to the treatment of congenital enzyme deficiencies using purified human, animal or recombinant enzyme preparations.

Enzyme replacement therapy (ERT) has been the standard therapeutic option for some types of MPS because of the ability to start immediate treatment with feasibility and safety and to improve prognosis. For instance, in November 2021 GC Pharma stated that EMA Grants Orphan Drug Designation to Hunterase ICV, The World's First Enzyme Replacement Therapy for Mucopolysaccharidosis Type II Administered by ICV Injection.

Moreover, in January 2021 GC Pharma, a South Korean biopharmaceutical company, and Clinigen K.K. ("Clinigen"), headquartered in Tokyo, received Japan manufacturing and marketing approval for Hunterase ICV (intracerebroventricular) Injection 15 mg (generic name: idursulfase-beta (recombinant)) as a treatment for mucopolysaccharidosis type II (Hunter syndrome). An enzyme-replacement therapy drug for intravenous injection is already in use in Japan and other countries as a treatment for systemic symptoms of mucopolysaccharidosis type I.

Geographical Analysis

North America accounted for approximately 38.4% of the market share in 2022

North America is estimated to hold about 38.4% of the total market share throughout the forecast period, owing to the factors coupled with higher awareness among the patient population towards advanced treatment options, FDA approvals, increasing diagnostic sophistication, presence of significant clinical trials, and the presence of major clinical-stage biopharmaceutical companies are their pipeline candidates, are responsible for the highest share of the region in the global market.

For instance, in June 2023 Denali Therapeutics Inc. recently announced new interim data from the ongoing open-label, single-arm Phase 1/2 study of DNL310 in children with MPS II (Hunter syndrome). DNL310 is an investigational enzyme replacement therapy designed to cross the BBB and address the behavioural, cognitive, and physical manifestations of MPS II.

Moreover, in January 2023 Orchard Therapeutics, a global gene therapy leader, announced the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for OTL-203, a hematopoietic stem cell (HSC) gene therapy being developed for the treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The company expects to initiate a global registrational trial evaluating the efficacy and safety of OTL-203 compared to the standard of care in the second half of 2023.

COVID-19 Impact Analysis

The COVID-19 pandemic has significantly impacted the mucopolysaccharidosis treatment market, leading to delays in research and clinical trials, and affecting access to healthcare, supply chain disruptions, and economic challenges. Safety concerns and restrictions have led to potential delays in the development and approval of mucopolysaccharidosis treatments. The pandemic has also impacted healthcare spending and insurance coverage.

Market Segmentation

By Disease Type

• MPS Type 1 ((Hurler syndrome)
• MPS Type 2 (Hunter syndrome)
• MPS Type 3 (Sanfilippo syndrome)
• MPS Type 4 (Morquio syndrome)
• MPS Type 6 (Maroteaux-Lamy syndrome)
• MPS Type 7 (Sly syndrome)
• MPS Type 9 (Natowicz syndrome)

By Treatment Type

• Enzyme Replacement Therapy
• Haematopoietic Stem Cell Transplantation (HSCT)
• Gene Therapy
• Others

By End User

• Hospitals
• Infusion Centers
• Ambulatory Surgical Centers

By Region

• North America
  • U.S.
  • Canada
  • Mexico
• Europe
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • Rest of Europe
• South America
  • Brazil
  • Argentina
  • Rest of South America
• Asia-Pacific
  • China
  • India
  • Japan
  • Australia
  • Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major global players in the mucopolysaccharidosis treatment market include: BioMarin Pharmaceuticals, CanbridgePharma, Cerezyme, Leadiant Biosciences, Lysogene, Denali Therapeutics, Regenxbio, JCR Pharmaceuticals, Orchard Therapeutics, Inventiva and among others.

Key Developments

• In September 2023 JCR Pharmaceuticals Co., Ltd announced key results from the 52-week interim data of its global phase I/II study with JR-171 in individuals with mucopolysaccharidosis type I (MPS I, also known as Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier ("BBB")-penetrating form of recombinant α-L-iduronidase that was developed using JCR's proprietary J-Brain Cargo technology.
• In March 2021, JCR Pharmaceuticals Co., Ltd. Announced the approval of IZCARGO (pabinafusp alfa 10 mL, intravenous drip infusion) by the Ministry of Health, Labour and Welfare (MHLW) for the treatment of mucopolysaccharidosis type II (MPS II, or Hunter syndrome).
• In September 2020 REGENXBIO Inc., a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, announced the expansion of the RGX-121 program for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome, to gain additional insight into the neurodegenerative manifestations of the disease and evaluate RGX-121 in a broader patient population.

Why Purchase the Report?

• To visualize the global mucopolysaccharidosis treatment market segmentation-based disease type, treatment type, end-user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development
• Excel data sheet with numerous mucopolysaccharidosis treatment market-level data points with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Disease mapping available in excel consisting of key diseases of all the major players.

The global mucopolysaccharidosis treatment market report would provide approximately 69 tables, 70 figures, and 185 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies