결절성 경화증(TSC) : 시장 전망

This report provides a data-driven overview of the current and future competitive landscape in Tuberous Sclerosis Complex (TSC) Therapeutics.

In 2025, there were 198,627 diagnosed prevalent cases of TSC-associated epilepsy in the 16 countries covered in GlobalData's epidemiology forecast.

In 2021, the most recent updated guidance for physicians for the diagnosis and treatment of TSC was established by the International TSC Consensus Group. In addition to the international consensus, country- or region-specific guidelines are available for selected TSC manifestations.

TSC treatment is multidisciplinary and symptom-driven, centered on seizure control, management of TSC-associated tumors, and organ-specific care (brain, kidneys, lungs, skin, heart). Standard care includes anti-seizure medications plus dietary/surgical options for refractory epilepsy, surveillance imaging, and supportive interventions (dermatologic, behavioral, developmental). Marketed mTOR therapies are key: everolimus (Afinitor) for subependymal giant cell astrocytoma (SEGA) and renal angiomyolipoma, and topical sirolimus (Hyftor) for facial angiofibromas in some markets. For seizures, vigabatrin is commonly used first-line for TSC-associated infantile spasms (with vision-toxicity monitoring), and cannabidiol (Epidiolex) is approved for TSC-associated seizures in certain age groups.

The TSC pipeline holds 18 molecules, with one pre-registration stage, one asset in Phase III development, and three assets in Phase II development.

Over the past decade, 85 clinical trials have been conducted in TSC. The years with the most studies initiated were 2022, with 14 trials, followed by 2021, with 13 trials. In 2022, everolimus was the most frequently studied drug, accounting for six of the 14 initiated trials by multiple sponsors.

Over the past decade, in deals involving companies developing TSC products, licensing agreements accounted for the largest share of deals in South America (50.0%), followed by the Middle East and Africa (40%), Asia-Pacific (33.3%), Europe (30.0%), and North America (22.7%). By comparison, partnerships were most prevalent in Europe (40.0%), followed by Asia-Pacific (33.3%), South America (25.0%), North America (22.7%) and the Middle East and Africa region (20.0%). Mergers were more prevalent in Europe (10.0%) and North America (9.1%), while no mergers were recorded in Asia-Pacific, South America, and the Middle East & Africa.

Scope

GlobalData's Tuberous Sclerosis Complex (TSC): Market View 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
• 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 Tuberous Sclerosis Complex (TSC) Therapeutics market.
• Drive revenues by understanding the key trends, innovative products and technologies, and companies likely to impact the global Tuberous Sclerosis Complex (TSC)Therapeutics 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

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 Product Profile and Sales Forecast

5 Pricing and Reimbursement Assessment

6 Pipeline Drugs Assessment

• 7.1 Pipeline Drugs
• 7.2 Overview by Development Stage
• 7.3 Overview by Mechanism of Action
• 7.4 Overview by Molecule Type
• 7.5 Overview by Route of Administration
• 7.6 Product Profile and Sales Forecast
• 7.7 Drug-specific PTSR and LoA
• 7.8 Therapy Area-specific PTSR and LoA

8 Clinical Trials Assessment

• 8.1 Historical Overview
• 8.2 Overview by Phase
• 8.3 Overview by Status
• 8.4 Overview by Phase for Ongoing and Planned Trials
• 8.5 Trials with Virtual Components
• 8.6 Top 20 Biomarkers by Trial Phase
• 8.7 Overview of Trials by Geography
• 8.8 Single-Country and Multinational Trials by Region
• 8.9 Top 20 Sponsors with Breakdown by Phase
• 8.10 Top 20 Sponsors with Breakdown by Status
• 8.11 Overview by Endpoint Status
• 8.12 Overview by Race and Ethnicity
• 8.13 Enrollment Data
• 8.14 Top 20 countries for Trial Sites
• 8.15 Top 20 Sites Globally
• 8.16 Feasibility Analysis - Geographic Overview
• 8.17 Feasibility Analysis - Benchmark Models

9 Deals Landscape

• 9.1 Mergers, Acquisitions, and Strategic Alliances by Region
• 9.2 Recent Mergers, Acquisitions, and Strategic Alliances

10 Commercial Assessment

• 10.1 Key Market Players

11 Future Market Catalysts

12 Appendix

• 12.1 Methodology
• 12.2 Methodology - Sales Forecast
• 12.3 Methodology - Pricing and Reimbursement
• 12.4 Methodology - PTSR and LoA Analysis
• 12.5 About the Authors
• 12.6 Contact Us
• 12.7 Disclaimer