세계의 레트 증후군 시장 : 경쟁 구도

This reports provides a data-driven overview of the current and future competitive landscape in Rett Syndrome therapeutics.

In the 16 countries covered in GlobalData's epidemiology forecast for Rett syndrome (RS), there are projected to be 97,327 diagnosed prevalent cases of RS in 2024, decreasing to 93,614 by 2029.

Treatment guidelines for RS are still evolving but are generally well-defined for symptom management and multidisciplinary care.

There are 51 molecules in development for RS, with only one drug in the pre-registration stage-trofinetide, which is already approved in the US and is being developed in the EU in Phase III.

A total of 42 clinical trials were initiated for RS in the last 10 years. The highest number of trials for RS were initiated in 2019, with nine trials, followed by 2023 with six trials.

In deals involving companies with RS assets, licensing agreements were the predominant deal type in the Asia-Pacific region, while partnerships dominated in North America and Africa. In Europe, acquisitions, partnerships, and licensing agreements were evenly distributed.

Scope

GlobalData's Rett Syndrome: 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, 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 Rett Syndrome market.
• Drive revenues by understanding the key trends, innovative products and technologies, and companies likely to impact the global Rett Syndrome market in the future.
• Formulate effective sales and marketing strategies by understanding the competitive landscape and analyzing the performance of various competitors.
• Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
• Organize your sales and marketing efforts by identifying the market categories that present the maximum opportunities for consolidations, investments, and strategic partnerships.

Table of Contents

1 Table of Contents

1 Preface

• 1.1 Contents
• 1.2 Report Scope
• 1.3 List of Tables and Figures
• 1.4 Abbreviations

2 Key Findings

3 Disease Landscape

• 3.1 Disease Overview
• 3.2 Epidemiology Overview
• 3.3 Treatment Overview

4 Marketed Drugs Assessment

• 4.1 Leading Marketed Drugs
• 4.2 Overview by Mechanism of Action
• 4.3 Overview by Molecule Type
• 4.4 Product Profiles and Sales Forecast

5 Pricing and Reimbursement Assessment

• 5.1 Annual Cost of Therapy
• 5.2 Time to Pricing and Reimbursement

6 Pipeline Drugs Assessment

• 6.1 Mid-to-late-stage Pipeline Drugs
• 6.2 Overview by Development Stage
• 6.3 Overview by Mechanism of Action
• 6.4 Overview by Molecule Type
• 6.5 Drug Specific Phase Transition Success Rate (PTSR) and Likelihood of Approval (LoA)
• 6.6 Therapy Area and Indication-specific PTSR and LoA

7 Clinical Trials Assessment

• 7.1 Historical Overview
• 7.2 Overview by Phase
• 7.3 Overview by Status
• 7.4 Overview by Phase for Ongoing and Planned Trials
• 7.5 Trials with Virtual Components
• 7.6 Overview of Trials by Geography
• 7.7 Single-Country and Multinational Trials by Region
• 7.8 Top 20 Sponsors with Breakdown by Phase
• 7.9 Top 20 Sponsors with Breakdown by Status
• 7.10 Overview by Endpoint Status
• 7.11 Overview by Race and Ethnicity
• 7.12 Enrollment Data
• 7.13 Top 20 countries for Trial Sites
• 7.14 Top 20 Sites Globally
• 7.15 Feasibility Analysis - Geographic Overview
• 7.16 Feasibility Analysis - Benchmark Models

8 Deals Landscape

• 8.1 Mergers, Acquisitions, and Strategic Alliances by Region
• 8.2 Recent Mergers, Acquisitions, and Strategic Alliances

9 Commercial Assessment

• 9.1 Key Market Players

10 Future Market Catalysts

11 Appendix

• 11.1 Methodology
• 11.2 Methodology - Sales Forecast
• 11.3 Methodology - Pricing and Reimbursement
• 11.4 Methodology - PTSR and LoA Analysis
• 11.5 About the Authors
• 11.6 Contact Us
• 11.7 Disclaimer