한글목차

GlobalData의 레트 증후군(RS)의 역학 예측에 포함된 16개국에서 2024년 RS 환자 수는 9만 7,327명으로 2029년까지 9만 3,614명으로 감소할 것으로 예측됩니다.

RS의 치료 가이드라인은 아직 개발 도상이지만, 증상 관리 및 집학적 치료에 대해서는 대략 명확하게 정의되어 있습니다.

RS의 치료제로서 개발중인 분자는 51유형이 있지만, 등록 전 단계에 있는 것은 1유형만으로 미국에서는 이미 승인되고 있으며 EU에서는 3단계에서 개발중인 트로피네타이드 뿐입니다.

지난 10년간 RS를 대상으로 시작된 임상시험은 총 42건이었습니다. RS의 임상시험이 가장 많이 시작된 것은 2019년의 9건으로, 이어 2023년의 6건이 되었습니다.

RS 자산을 가진 기업이 관여하는 거래에서는 아시아태평양에서는 라이선스 계약이 북미와 아프리카에서는 파트너십이 우세했습니다. 유럽에서는 인수, 제휴 및 라이선스 계약이 균등하게 배포되었습니다.

이 보고서는 세계 레트 증후군 시장에 대한 조사 분석을 통해 질병 상황, 시상한 약물 및 파이프라인 약물 평가, 현재 및 미래경쟁 구도 등을 제공합니다.

목차

제1장 서문

제2장 주요 조사 결과

제3장 질병 상황

• 질병의 개요
• 역학의 개요
• 치료 개요

제4장 출시가 끝난 약품의 평가

• 주요 출시가 끝난 약품
• 개요 : 작용기전별
• 개요 : 분자 유형별
• 제품 프로파일, 매출 예측

제5장 가격 설정과 상환 평가

• 연간 치료비
• 가격 설정과 상환까지의 시간

제6장 파이프라인 의약품 평가

• 중기-후기의 파이프라인 의약품
• 개요 : 개발 단계별
• 개요 : 작용기전별
• 개요 : 분자 유형별
• 약품 고유의 위상 전환 성공률(PTSR)과 승인 가능성(LoA)
• 치료 영역과 적응증 고유의 PTSR과 LoA

제7장 임상시험 평가

• 과거의 개요
• 개요 : 페이즈별
• 개요 : 스테이터스별
• 개요: 페이즈별(진행중/계획이 끝난 시험)
• 가상 컴포넌트에 의한 시험
• 시험의 개요 : 지역별
• 단일국/다국간 시험:지역별
• 스폰서 상위 20개사와 내역: 페이즈별
• 스폰서 상위 20사와 내역 : 스테이터스별
• 개요 : 엔드포인트 스테이터스별
• 개요: 인종/민족별
• 등록 데이터
• 시험 시설의 상위 20개국
• 세계의 주요 20개 시설
• 실현 가능성 분석 - 지리적 개요
• 실현 가능성 분석 - 벤치마크 모델

제8장 거래 상황

• 합병, 인수, 전략적 제휴:지역별
• 최근의 합병, 인수, 전략적 제휴

제9장 상업적 평가

• 주요 시장 기업

제10장 미래 시장 카탈리스트

제11장 부록

영문목차

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