한글목차

2024년에는 16개국에서 111,000명 이상이 지대형 근이영양증(LGMD) 유병자로 진단받을 것으로 예상됩니다.

지금까지 LGMD의 진행을 변화시키는 것으로 입증된 질병 변형 치료법은 없기 때문에 증상 관리가 치료의 주축을 이루고 있습니다.

LGMD 파이프라인은 21개의 분자를 보유하고 있으며, 등록 전 단계의 자산은 없으며, 3상 개발 중인 자산은 2분자에 불과하고, 2상 자산은 4분자입니다.

지난 10년간 LGMD는 22건의 임상시험을 진행했습니다. 가장 많은 임상이 진행된 해는 2016년(4건)이며, 2023년, 2022년, 2019년(각 3건)이 그 뒤를 이었습니다.

지난 10년간 북미와 아시아태평양에서 가장 많은 파트너십이 체결됐습니다. 반면 유럽에서는 합병과 라이선스 계약이 같은 수치를 기록했습니다.

이 보고서는 전 세계 지대형 근이영양증(LGMD) 시장에 대해 조사했으며, 질환 개요, 임상시험 동향, 파이프라인 개요, 향후 전망 등의 정보를 제공합니다.

목차

제1장 서문

제2장 주요 조사 결과

제3장 병 상황

• 질환 개요
• 역학 개요
• 치료 개요

제4장 출시 약제 평가

• 주요 시판약
• 작용기전별 개요
• 분자 유형별 개요
• 제품 개요와 판매 예측

제5장 가격 결정과 상환 평가

제6장 파이프라인 의약품 평가

제7장 임상시험 평가

• 역사적 개요
• 상별 개요
• 현황별 개요
• 진행중 및 계획중인 임상시험 상별 개요
• 가상 컴포넌트 시험
• 지역 시험 개요
• 지역별 단일국 및 다국간 시험
• 상별 스폰서 상위 20개사
• 현황별 스폰서 상위 20개사
• 엔드포인트 현황별 개요
• 인종과 민족별 개요
• 등록 데이터
• 임상시험 시행 시설 주요 20개국
• 세계의 주요 20개 시설
• 실현 가능성 분석 - 지역 개요
• 실현 가능성 분석 - 벤치마크 모델

제8장 거래 상황

제9장 상업적 평가

• 주요 시장 진출기업

제10장 향후 시장 성장 촉진요인

제11장 부록

영문목차

This reports provides a data-driven overview of the current and future competitive landscape in Limb-Girdle Muscular Dystrophy therapeutics.

In 2024, more than 111,000 diagnosed prevalent cases of LGMD are anticipated in the 16 countries covered in GlobalData's epidemiology forecast.

No disease-modifying treatment has proven to alter LGMD progression so far, and thus symptom management remains the mainstay of care.

The LGMD pipeline holds 21 molecules with no assets in the pre-registration stage, only two molecules in Phase III development, and four assets in Phase II.

Over the past decade, 22 clinical trials have been conducted in LGMD. The largest number of studies was conducted in 2016 (four trials) followed by 2023, 2022, and 2019 (three trials each).

During the past decade, partnerships were the most frequent type of deal in North America and Asia-Pacific. Meanwhile, in Europe, mergers and licensing agreements contributed to an equal number of deals.

Scope

GlobalData's Limb-Girdle Muscular Dystrophy: 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 Limb-Girdle Muscular Dystrophy market.
• Drive revenues by understanding the key trends, innovative products and technologies, and companies likely to impact the global Limb-Girdle Muscular Dystrophy 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