한글목차

2024년에는 GlobalData의 역학 예측이 적용되는 16개국에서 2,000건 이상의 GSD 발병이 진단될 것으로 예상됩니다.

시판된 GSD 치료제의 대부분은 효소 보충 요법입니다.

3상 파이프라인은 2개, 2상 파이프라인은 7개이며, 등록 전 단계의 약물은 없습니다.

지난 10년간 GSD와 PD를 대상으로 총 67건의 임상시험이 시작되었으며, GSD의 경우 2021년에 가장 많은 임상시험이 시작되었습니다(11건).

GSD 개발에서 남미에서는 파트너십, 아프리카에서는 라이선싱 계약이 주요 거래 유형이었습니다.

이 보고서는 세계 글리코겐 저장병(GSD) 시장을 조사 분석하여 질병 현황, 시판 중인 약물 및 파이프라인 의약품 평가, 현재와 미래경쟁 구도 등을 제공합니다.

목차

제1장 서문

제2장 주요 조사 결과

제3장 질환 상황

• 질환 개요
• 역학 개요
• 치료 개요

제4장 출시 약품 평가

• 주요 출시 약품
• 개요 : 작용기전별
• 개요 : 분자 유형별
• 제품 개요와 판매 예측

제5장 가격 결정과 상환 평가

• 연간 치료비
• 가격 결정과 상환까지 시간

제6장 파이프라인 의약품 평가

• 중기부터 후기 파이프라인 의약품
• 개요 : 개발 단계별
• 개요 : 작용기전별
• 개요 : 분자 유형별
• 약품 고유 단계 이동 성공률(PTSR)과 승인 가능성(LoA)
• 치료 영역과 적응증 고유의 PTSR 및 LoA

제7장 임상시험 평가

• 과거 개요
• 개요 : 단계별
• 개요 : 현황별
• 개요 : 단계별(진행중/계획된 임상시험)
• 가상 컴포넌트에 의한 시험
• 임상시험 개요 : 지역별
• 단일국/다국간 시험 : 지역별
• 스폰서 주요 20개사와 내역 : 단계별
• 스폰서 주요 20개사와 내역 : 현황별
• 개요 : 엔드포인트 현황별
• 개요 : 인종/민족별
• 등록 데이터
• 임상시험 시설 주요 20개국
• 세계의 주요 20개 시설
• 실현 가능성 분석 - 지역 개요
• 실현 가능성 분석 - 벤치마크 모델

제8장 거래 상황

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

제9장 상업적 평가

• 주요 시장 기업

제10장 향후 시장 촉매

제11장 부록

영문목차

This reports provides a data-driven overview of the current and future competitive landscape in Glycogen Storage Disorders therapeutics.

In 2024, more than 2,000 diagnosed incident cases of GSD are anticipated in the 16 countries covered in GlobalData's epidemiology forecast.

Most of the marketed drugs for GSD are enzyme replacement therapies.

The Phase III pipeline consists of two drugs, with seven drugs in Phase II; there are no drugs in the pre-registrational stage.

A total of 67 clinical trials were initiated for GSD and PD in the last 10 years. The most trials initiated in GSD were conducted in 2021 (11 trials).

In the development of GSD, the predominant deal type in South America was partnership and Africa was licensing agreement.

Scope

GlobalData's Glycogen Storage Disorders: 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 Glycogen Storage Disorders market.
• Drive revenues by understanding the key trends, innovative products and technologies, and companies likely to impact the global Glycogen Storage Disorders 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 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