脊髄性筋萎縮症（SMA）市場：上市済み薬剤およびパイプライン薬剤の評価、臨床試験、競合情勢

世界の主要16カ国において、脊髄性筋萎縮症（SMA）は2023年に138,844例以上の5q SMA（i-Ⅳ型）の有病者が診断されると予測されています。現在、SMAの治療薬として承認されているのはほんの一握りで、いずれも生存運動ニューロン（SMN）タンパク質のアップレギュレーションという単一の重要なメカニズムに属しています。SMAの後期パイプラインには、登録前の薬剤が1つ、第III相段階にある薬剤が5つあります。SMAの臨床試験スペースは、商業的スポンサーと学術的スポンサーがほぼ同程度に混在しており、日本が第III相試験を実施する主要国として浮上しています。ライセンシング契約は、SMA領域において世界的に最も顕著な契約形態です。

当レポートでは、脊髄性筋萎縮症（SMA）市場について調査し、疾患の概要、上市済み薬剤およびパイプライン薬剤の評価、臨床試験動向、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 病気の情勢

• 病気の概要
• 疫学の概要
• 治療の概要

第4章 上市済み薬剤の評価

• 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

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トライアル
• 地理的概要
• 地域別の単一国および多国籍試験
• 上位20のスポンサーとフェーズ別の内訳
• 上位20スポンサーのステータス別の内訳
• 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
• 世界のトップ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

• 地域別の合併、買収、戦略的提携
• 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 将来の市場促進要因

第11章 付録

Abstract

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

• More than 138,844 diagnosed prevalent cases of 5q SMA (types I-IV) are anticipated in 2023 in the 16 countries covered in GlobalData's epidemiology forecast for SMA.
• Only a handful of approved therapeutic drugs are currently available for the treatment of SMA, all belonging to a single key mechanism of survival motor neuron (SMN) protein upregulation.
• The late-stage pipeline for SMA includes one drug in pre-registration and five drugs in Phase III.
• The clinical trial space in SMA consists of almost an equal mix of commercial as well as academic sponsors, with Japan emerging as the key country for conducting Phase III trials.
• Licensing agreements were the most prominent deals type ventured in the SMA space globally.

Scope

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