한글목차

프리드리히 운동실조증(FRDA)에 대한 역학 예측에 따르면, 2024년 16개국에서 54,000명 이상의 FRDA 환자가 진단될 것으로 예상되며, FRDA의 시판 중인 약물 영역은 두 가지 승인된 약물로 제한되어 있으며, Biogen의 Skyclarys( omaveloxolone)이 FRDA 영역의 주요 약물입니다.

FRDA 영역의 연구 개발 노력은 제한적이며, 현재 등록 전 단계의 약물은 없으며, 임상 3상 단계에 있는 분자는 단 하나뿐입니다. 미국은 FRDA 시험을 수행하는 주요 국가로 부상하고 있습니다. 북미에서는 FRDA 치료제를 개발하는 회사들 사이에서 파트너십이 가장 일반적인 계약 형태였습니다. 유럽에서는 라이선스 계약이 주류를 이루고 있습니다.

이 보고서는 전 세계 프리드리히 운동실조증(FRDA) 시장을 조사하여 질환 개요와 함께 임상시험 동향, 파이프라인 개요, 향후 전망 등을 제공합니다.

목차

제1장 서문

제2장 주요 조사 결과

제3장 질환 상황

• 질병 개요
• 역학 개요
• 치료 개요

제4장 출시 약제 평가

• 주요 출시 약제
• 작용기서별 개요
• 투여 경로별 개요
• 출시 약제 프로파일과 판매량 예측

제5장 약가 설정과 상환 평가

• 연간 치료비
• 약가 설정과 상환 기간

제6장 파이프라인 약제 평가

• 제III상 파이프라인 약제
• 개발 단계별 개요
• 분자 유형별 개요
• 작용기서별 개요
• 투여 경로별 개요
• 의약품 특이적 상전이 성공률(PTSR)과 승인 가능성(LoA)
• 치료 분야 및 적응증 고유의 PTSR 및 LoA

제7장 임상시험 평가

• 과거 개요
• 단계별 개요
• 상황별 개요
• 진행중 및 계획중인 시험 단계별 개요
• 가상 컴포넌트를 사용한 임상시험
• 지역적 개요
• 지역별 단일국 및 다국적 시험
• 상위 20개사 스폰서와 단계별 내역
• 상위 20개사 스폰서 상황별 내역
• 엔드포인트 상황별 개요
• 인종 및 민족별 개요
• 등록 데이터
• 임상시험 사이트 상위 20개국
• 세계의 상위 20개 사이트
• 실현 가능성 분석 - 지역적 개요
• 실현 가능성 분석 - 벤치마크 모델

제8장 거래 상황

제9장 상업적 평가

• 주요 시장 진출 기업

제10장 향후 시장 성장 촉진요인

제11장 부록

영문목차

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

• More than 54,000 diagnosed prevalent cases of FRDA are anticipated in 2024 in the 16 countries covered in GlobalData's epidemiology forecast for FRDA.
• The marketed drugs space for FRDA is limited to just two approved drugs, Biogen's Skyclarys (omaveloxolone) is the leading drug in FRDA space.
• R&D efforts in the FRDA space are limited, with no pre-registration drugs currently in the pipeline and only one molecule in Phase III.
• The US is emerging as the key country for conducting FRDA trials.
• In North America, partnerships were the most common type of deal among companies developing treatments for FRDA. In Europe, licensing agreements were prevalent.

Scope

GlobalData's FRDA: 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 FRDA market.
• Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global FRDA market in the future.
• Formulate effective sales and marketing strategies by understanding the competitive landscape and by 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 and segments that present 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 Route of Administration
• 4.4 Marketed Drugs Profiles and Sales Forecasts

5 Pricing and Reimbursement Assessment

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

6 Pipeline Drugs Assessment

• 6.1 Phase III Pipeline Drugs
• 6.2 Overview by Development Stage
• 6.3 Overview by Molecule Type
• 6.4 Overview by Mechanism of Action
• 6.5 Overview by Route of Administration
• 6.6 Drug Specific Phase Transition Success Rate (PTSR) and Likelihood of Approval (LoA)
• 6.7 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 Geographic Overview
• 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