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.

Key Mechanisms of Action ·       Enzyme Activator

·       Enzyme Replacement

Top Sponsors (Marketed and Pipeline Drugs) ·       JCR Pharmaceuticals Co Ltd

·       Shire Pharmaceuticals International UC

·       Generium

·       Denali Therapeutics Inc

·       RegenxBio Inc

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Top Sponsors in the MPS II Drugs Market

A few 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, and The Lundquist Institute 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 Drugs  Market Analysis by Sponsors

Mucopolysaccharidosis II Drugs  Market Analysis by Sponsors

Buy the Full Report for More Sponsor’s Insights into the MPS II Drugs Market, Download a Free Report Sample

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 (%)

Mucopolysaccharidosis II Pipeline Drugs Analysis by MoA, 2023 (%)

Buy the Full Report for More MoA Insights into the MPS II pipeline drugs market, Download a Free Report Sample

Segments Covered in the Report

MPS II Pipeline Drugs Mechanisms of Action Outlook (Number of Drugs, 2023)

  • Enzyme Activator
  • Enzyme Replacement


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

1 Preface_x000D_

1.1 Contents_x000D_

1.2 Report Scope_x000D_

1.3 List of Tables and Figures_x000D_

1.4 Abbreviations_x000D_

2 Key Findings_x000D_

3 Disease Landscape_x000D_

3.1 Disease Overview_x000D_

3.2 Epidemiology Overview_x000D_

3.3 Treatment Overview _x000D_

4 Marketed Drugs Assessment_x000D_

4.1 Leading Marketed Drugs_x000D_

4.2 Overview by Mechanism of Action_x000D_

4.3 Overview by Route of Administration_x000D_

4.4 Marketed Drugs Profiles and Sales Forecasts_x000D_

5 Pricing and Reimbursement Assessment_x000D_

5.1 Annual Therapy Cost_x000D_

5.2 Time to Pricing and Reimbursement_x000D_

6 Pipeline Drugs Assessment_x000D_

6.1 Phase III Pipeline Drugs_x000D_

6.2 Overview by Development Stage_x000D_

6.3 Overview by Molecule Type_x000D_

6.4 Overview by Mechanism of Action_x000D_

6.5 Overview by Route of Administration_x000D_

6.6 Drug Specific Phase Transition Success Rate (PTSR) and Likelihood of Approval (LoA)_x000D_

6.7 Therapy Area and Indication-specific PTSR and LoA_x000D_

7 Clinical Trials Assessment_x000D_

7.1 Historical Overview_x000D_

7.2 Overview by Phase_x000D_

7.3 Overview by Status_x000D_

7.4 Overview by Phase for Ongoing and Planned Trials_x000D_

7.5 Trials with Virtual Components_x000D_

7.6 Geographic Overview_x000D_

7.7 Single-Country and Multinational Trials by Region_x000D_

7.8 Top 20 Sponsors with Breakdown by Phase_x000D_

7.9 Top 20 Sponsors with Breakdown by Status_x000D_

7.10 Overview by Endpoint Status_x000D_

7.11 Overview by Race and Ethnicity_x000D_

7.12 Enrollment Data_x000D_

7.13 Top 20 countries for Trial Sites_x000D_

7.14 Top 20 Sites Globally_x000D_

7.15 Feasibility Analysis – Geographic Overview_x000D_

7.16 Feasibility Analysis – Benchmark Models_x000D_

8 Deals Landscape_x000D_

8.1 Mergers, Acquisitions, and Strategic Alliances by Region_x000D_

8.2 Recent Mergers, Acquisitions, and Strategic Alliances_x000D_

9 Commercial Assessment_x000D_

9.1 Key Market Players_x000D_

10 Future Market Catalysts_x000D_

11 Appendix_x000D_

11.1 Methodology_x000D_

11.2 Methodology – Sales Forecast_x000D_

11.3 Methodology – Pricing and Reimbursement_x000D_

11.4 Methodology – PTSR and LoA Analysis_x000D_

11.5 About the Authors_x000D_

11.6 Contact Us_x000D_

11.7 Disclaimer

Frequently asked questions

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