Mucopolysaccharidosis II (MPS II) Marketed and Pipeline Drugs Assessment, Clinical Trials and Competitive Landscape
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Mucopolysaccharidosis II Marketed and Pipeline Drugs Report Overview
Mucopolysaccharidosis II (MPS II) is an inherited lysosomal storage disorder of carbohydrate metabolism that occurs almost exclusively in males. In patients with MPS II, glycosaminoglycans accumulate within tissues and organs, contributing to the signs and symptoms of the disease. MPS II affects multiple organs and physiologic systems. It is a progressively debilitating disorder; however, the rate of progression varies among affected individuals.
MPS II is caused by genetic changes in the iduronate 2-sulfatase (IDS) gene and is inherited in an X-linked manner. There is a wide range in severity of symptoms present in individuals with MPS II. In many individuals with MPS II, the condition is caused by relatively small changes (e.g., certain missense or nonsense mutations) in the IDS gene or deletion or insertion of genetic material like single-base deletions or insertions that affect gene function.
The Mucopolysaccharidosis II marketed and pipeline drugs market research report includes an assessment of the disease epidemiology, leading marketed products with sales forecast, pricing, and reimbursement, late-to-mid-stage pipeline products with phase transition success rate (PTSR), and the likelihood of approval (LoA) for Mucopolysaccharidosis II.
Mucopolysaccharidosis II Pipeline Drugs Segmentation by Mechanisms of Action
The key mechanisms of action for MPS II pipeline drugs are enzyme activator and enzyme replacement. Most of the MPS II pipeline drugs are enzyme activators.
Mucopolysaccharidosis II Pipeline Drugs Analysis by MoA, 2023 (%)
For more MoA insights into the MPS II pipeline drugs market, download a free report sample
Top Sponsors in the MPS II Marketed and Pipeline Drugs Market
Some of the top sponsors in the MPS II marketed and pipeline drugs market are JCR Pharmaceuticals Co Ltd, Shire Pharmaceuticals International UC, Generium, Denali Therapeutics Inc, RegenxBio Inc, The Lundquist Institute, Takeda Pharmaceutical Co Ltd, GC Biopharma Corp, Nihon University, Memorial Sloan Kettering Cancer Center, Fate Therapeutics Inc, ArmaGen Inc, Masonic Cancer Center, Homology Medicines Inc, and Clinigen Ltd. Commercial sponsors dominated the clinical trials space in MPS II over the past 10 years with JCR Pharmaceuticals sponsoring the highest number of clinical trials overall, followed by Shire Pharmaceuticals International and Generium.
Mucopolysaccharidosis II Pipeline Drugs Market Report Overview
Key Mechanisms of Action | Enzyme Activator and Enzyme Replacement |
Top Sponsors (Marketed and Pipeline Drugs) | JCR Pharmaceuticals Co Ltd, Shire Pharmaceuticals International UC, Generium, Denali Therapeutics Inc, RegenxBio Inc, The Lundquist Institute, Takeda Pharmaceutical Co Ltd, GC Biopharma Corp, Nihon University, Memorial Sloan Kettering Cancer Center, Fate Therapeutics Inc, ArmaGen Inc, Masonic Cancer Center, Homology Medicines Inc, and Clinigen Ltd |
Segments Covered in the Report
MPS II Pipeline Drugs Mechanisms of Action Outlook (Number of Drugs, 2023)
- Enzyme Activator
- Enzyme Replacement
Scope
GlobalData’s Mucopolysaccharidosis II (MPS II) Marketed and Pipeline Drugs Assessment, Clinical Trials, and Competitive Landscape combines data from the Pharma Intelligence Center with in-house analyst expertise to provide a competitive assessment of the disease marketplace.
Components of the report include:
- Disease Landscape
- Disease Overview
- Epidemiology Overview
- Treatment Overview
- Marketed Products Assessment
- Breakdown by Mechanism of Action, Molecule Type, Route of Administration
- Product Profiles with Sales Forecast
- Pricing and Reimbursement Assessment
- Annual Therapy Cost
- Time to Pricing and Time to Reimbursement
- Pipeline Assessment
- Breakdown by Development Stage, Mechanism of Action, Molecule Type, Route of Administration
- Product Profiles with Sales Forecast
- Late-to-mid-stage Pipeline Drugs
- Phase Transition Success Rate and Likelihood of Approval
- Clinical Trials Assessment
- Breakdown of Trials by Phase, Status, Virtual Components, Sponsors, Geography, and Endpoint Status
- Enrolment Analytics, Site Analytics, Feasibility Analysis
- Deals Landscape
- Mergers, Acquisitions, and Strategic Alliances by Region
- Overview of Recent Deals
- Commercial Assessment
- Key Market Players
- Future Market Catalysts
Reasons to Buy
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
- Develop business strategies by understanding the trends shaping and driving the MPS II market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global MPS II market in the future.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective counterstrategies to gain a competitive advantage.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.
Table of Contents
Frequently asked questions
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What are the key mechanisms of action for MPS II pipeline drugs?
The key mechanisms of action for MPS II pipeline drugs are enzyme activator and enzyme replacement.
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What is Mucopolysaccharidosis II?
Mucopolysaccharidosis II (MPS II) is an inherited lysosomal storage disorder of carbohydrate metabolism that occurs almost exclusively in males.
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What is one of the primary causes of MPS II?
MPS II is caused by genetic changes in the iduronate 2-sulfatase (IDS) gene and is inherited in an X-linked manner.
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Who are the top sponsors for MPS II marketed and pipeline drugs market?
Some of the top sponsors for the MPS II marketed and pipeline drugs market are JCR Pharmaceuticals Co Ltd, Shire Pharmaceuticals International UC, Generium, Denali Therapeutics Inc, RegenxBio Inc, The Lundquist Institute, Takeda Pharmaceutical Co Ltd, GC Biopharma Corp, Nihon University, Memorial Sloan Kettering Cancer Center, Fate Therapeutics Inc, ArmaGen Inc, Masonic Cancer Center, Homology Medicines Inc, and Clinigen Ltd.
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