フリードライヒ失調症（FRDA）-競合情勢

フリードライヒ失調症（FRDA）に関する疫学予測では、16カ国において2024年に54,000例以上のFRDAの有病者が診断されると予測されています。FRDAの上市済み薬剤領域は2つの承認薬に限られており、BiogenのSkyclarys（omaveloxolone）がFRDA領域の主要薬剤です。

FRDA領域の研究開発努力は限られており、現在パイプラインにある登録前の薬剤はなく、第III相にある分子は1つだけです。米国はFRDA試験を実施する主要国として台頭しています。北米では、FRDAの治療法を開発する企業の間で、パートナーシップが最も一般的な契約形態でした。欧州ではライセンス契約が主流となっています。

当レポートでは、世界のフリードライヒ失調症（FRDA）市場について調査し、疾患の概要とともに、治験動向、パイプライン概要、将来の見通しなどを提供しています。

目次

第1章 序文

第2章 主な調査結果

第3章 疾患の情勢

• 病気の概要
• 疫学の概要
• 治療の概要

第4章 上市済み薬剤の評価

• 主要な上市済み薬剤
• 作用機序別概要
• 投与経路別概要
• 上市済み薬剤のプロファイルと売上予測

第5章 薬価設定と償還の評価

• 年間治療費
• 薬価設定と償還期間

第6章 パイプライン薬剤の評価

• 第Ⅲ相パイプライン薬剤
• 開発段階別概要
• 分子タイプ別概要
• 作用機序別概要
• 投与経路別概要
• 医薬品特異的相転移成功率（PTSR）と承認の可能性（LoA）
• 治療領域および適応症固有のPTSRおよびLoA

第7章 臨床試験の評価

• 過去の概要
• フェーズ別概要
• ステータス別概要
• 進行中および計画中の試験のフェーズ別概要
• 仮想コンポーネントを使用した治験
• 地理的概要
• 地域別の単一国および多国籍試験
• 上位20社のスポンサーとフェーズ別内訳
• 上位20社のスポンサーのステータス別内訳
• エンドポイントステータス別概要
• 人種および民族別の概要
• 登録データ
• 治験サイトの上位20か国
• 世界の上位20サイト
• 実現可能性分析- 地理的概要
• 実現可能性分析- ベンチマークモデル

第8章 取引情勢

第9章 商業的評価

• 主要な市場参入企業

第10章 将来の市場促進要因

第11章 付録

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