異染性白質ジストロフィー（MLD）の世界市場：上市済み薬品とパイプライン薬品の評価、臨床試験、競合情勢

概要

GlobalDataによるMLDの疫学予測では、対象16ヶ国において2023年に5,500を超えるMLD患者が診断されると予測されています。

MLD治療薬の市場参入はOrchard TherapeuticsのLibmeldyのみに限られています。

MLDのフェーズ3パイプライン薬品は不足しています。現在、米国で登録前の段階にある薬剤は1種類のみです。

過去10年間に実施された臨床試験はわずか15件です。北米はMLDの臨床試験を行う主要国として浮上しており、合計27の臨床試験施設があります。

北米のMLD分野では、パートナーシップがもっとも顕著な取引形態となっています。

当レポートでは、世界の糖尿病性腎症（DN）市場について調査分析し、疾患の情勢、上市済み薬品とパイプライン薬品の評価、現在と将来の競合情勢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 疾患情勢

• 疾患の概要
• 疫学の概要
• 治療の概要

第4章 上市済み薬品の評価

• 主な上市済み薬品
• 概要：作用機序別
• 概要：投与経路別
• 上市済み薬品のプロファイルと売上の予測

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 パイプライン薬品の評価

• フェーズ3パイプライン薬品
• 概要：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
• 薬品固有のフェーズ移行成功率（PTSR）と承認可能性（LoA）
• 治療領域と適応症固有のPTSRとLoA

第7章 臨床試験の評価

• 過去の概要
• 概要：フェーズ別
• 概要：ステータス別
• 概要：進行中/計画済みの試験のフェーズ別
• 仮想コンポーネントを使用した試験
• 地理的概要
• 単一国・多国間試験：地域別
• 上位20のスポンサーと内訳：フェーズ別
• 上位20のスポンサーと内訳：ステータス別
• 概要：エンドポイントステータス別
• 概要：人種/民族別
• 登録データ
• 試験施設の上位20ヶ国
• 世界の上位20施設
• 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

• 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

• 主な市場企業

第10章 将来の市場カタリスト

第11章 付録

Abstract

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

More than 5,500 diagnosed prevalent cases of MLD are anticipated in 2023 in the 16 countries covered in GlobalData's epidemiology forecast for MLD.

The marketed space for MLD treatment is limited only to Orchard Therapeutics' Libmeldy.

There is a lack of Phase III pipeline drugs for MLD. Only a single drug is currently available in pre-registration stage in the US.

Over the past decade, only 15 clinical trials have been conducted. North America has emerged as the primary country for conducting MLD trials, with a total of 27 trial sites.

Partnerships were the most prominent type of deal ventured in the MLD space in North America.

Scope

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