市場調査レポート
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1148934

フェニルケトン尿症市場:上市済みおよびパイプライン薬剤評価、臨床試験、競合情勢

Phenylketonuria Marketed and Pipeline Drugs Assessment, Clinical Trials and Competitive Landscape

出版日: | 発行: GlobalData | ページ情報: 英文 69 Pages | 納期: 即納可能 即納可能とは

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フェニルケトン尿症市場:上市済みおよびパイプライン薬剤評価、臨床試験、競合情勢
出版日: 2022年09月28日
発行: GlobalData
ページ情報: 英文 69 Pages
納期: 即納可能 即納可能とは
ご注意事項 :
本レポートは最新情報反映のため適宜更新し、内容構成変更を行う場合があります。ご検討の際はお問い合わせください。
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  • 概要
  • 目次
概要

当レポートでは、フェニルケトン尿症市場について調査し、疾患の概要とともに、上市済みおよびパイプライン薬剤の評価、臨床試験動向、および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

  • 疾患の概要
  • 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 パイプライン薬剤評価

  • 第III相パイプライン薬
  • 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
  • 薬剤固有の相移行成功率(PTSR)および承認の可能性(LoA)
  • 治療領域および適応症別PTSRおよびLoA

第7章 臨床試験の評価

第8章 取引情勢

第9章 商業的評価

  • 主な市場参入企業

第10章 将来の市場促進要因

第11章 付録

目次
Product Code: GDHC081CL

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

Synopsis

  • In 2022, there will be more than 500,000 diagnosed prevalent cases of PKU across 16 pharmaceutical markets.
  • There are only two leading marketed drugs for the treatment of PKU, both from BioMarin Pharmaceutical.
  • R&D activity in PKU is relatively low, with two products in Phase III development.
  • Of the 56 trials investigating PKU that were initiated over the past 10 years, nearly 18% were Phase III trials.
  • In deals involving companies with PKU assets, partnerships and licensing agreements are the most common type of deal globally.
  • Commercial launch of sepiapterin from PTC Therapeutics is expected in the US and EU in Q4 2023.

Scope

GlobalData's Phenylketonuria: 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, Molecule Type, Route of Administration
  • Product Profiles with Sales Forecast
  • Pricing and Reimbursement Assessment
  • Retail or Manufacturer Price of Products
  • 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 happening and driving the Phenylketonuria market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global Phenylketonuria 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 Manufacturer Price
  • 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