バッテン病（BD）の市場・パイプライン薬剤評価、臨床試験、競合情勢

16の主要医薬品市場全体において2023年に診断されたBDの有病者数は8,537人であり、2028年には8,717人に増加すると予測されています。BD治療薬として上市されているのは、バイオマリン社のブリヌーラ（セルリポナーゼ・アルファ）のみです。

BDの後期パイプライン薬剤は不足しており、現在世界でフェーズII段階にあるのは7剤のみです。

BDを対象とした15件の臨床試験が開始または計画されており、うち5件は完了したが、7件は現在進行中です。進行中の7つの試験のうち、約71.4%が第I/II相試験、28.6%が第I相試験であり、第III相試験はありません。

当レポートは、バッテン病（BD）治療薬の現在および将来の競合情勢について、データに基づいた概要を提供しています。

目次

第1章 序文

第2章 主な調査結果

第3章 病気の情勢

• 病気の概要
• 疫学の概要
• 治療の概要

第4章 上市済み薬剤評価

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

第5章 価格設定と償還の評価

• 年間治療費
• 価格設定と償還までの時間

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
• 開発段階別の概要
• 分子タイプ別の概要
• 作用機序別の概要
• 投与経路別の概要
• 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

• 歴史的概要
• フェーズ別の概要
• ステータス別の概要
• 進行中および計画中の試験のフェーズ別の概要
• 仮想コンポーネントを使用した臨床試験
• 地理的概要
• 地域別の単一国および多国籍試験
• 上位20のスポンサーのフェーズ別内訳
• 上位20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の概要
• 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 取引の情勢

• 合併、買収、戦略的提携、地域別
• 最近の合併、買収、戦略的提携

第9章 商業的評価

• 主要な市場参入企業

第10章 将来の市場触媒

第11章 付録

Abstract

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

Across the 16 major pharmaceutical markets, GlobalData estimates that there are 8,537 diagnosed prevalent cases of BD in 2023 and the number is expected to increase to 8,717 cases by 2028.

The marketed space for BD treatment is limited only to BioMarin's Brineura (cerliponase alfa).

There is a lack of late-stage pipeline drugs for BD; only seven drugs are currently in Phase II globally.

15 trials have been initiated or planned in BD, five of which have been completed, while seven trials are currently ongoing. Of the seven ongoing trials, approximately 71.4% are in Phase I/II, and 28.6% are in Phase I of development, while no trials are in Phase III development.

Partnership is the most prevalent deal type in Europe (71.4%).

Scope

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