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フェニルケトン尿症 - パイプライン製品の分析

Phenylketonuria (PKU) - Pipeline Review, H1 2017

発行 Global Markets Direct 商品コード 321900
出版日 ページ情報 英文 47 Pages
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フェニルケトン尿症 - パイプライン製品の分析 Phenylketonuria (PKU) - Pipeline Review, H1 2017
出版日: 2017年02月28日 ページ情報: 英文 47 Pages
概要

フェニルケトン尿症は希少な遺伝性疾患で、必須アミノ酸であるフェニルアラニンによって引き起こされます。症状は知能障害、精神遅滞、多動、発疹、骨強度の不足、異常に小さな頭部といったものです。

当レポートでは、フェニルケトン尿症に対する治療薬の開発状況について調査分析し、パイプライン製品の概要、治験の段階別の製品の概要、主要企業プロファイル、薬剤のプロファイル、パイプライン製品の最新動向、最新ニュースとプレスリリースなどについて、体系的な情報を提供しています。

目次

イントロダクション

フェニルケトン尿症の概要

治療薬の開発

  • パイプライン製品:概要
  • パイプライン製品:比較分析

開発中の治療薬:企業別

パイプライン製品の概要

  • 後期段階の製品
  • 初期段階の製品

開発中の製品:企業別

治療薬の開発に従事している企業

  • BioMarin Pharmaceutical Inc.
  • Erytech Pharma SA
  • Promethera Biosciences S.A.
  • SOM Innovation Biotech SL

治療薬の評価

  • 単独療法の製品
  • 投与経路別
  • 分子タイプ別

薬剤のプロファイル

  • BMN-165
  • Cell therapy for Phenylketonuria
  • HepaStem
  • sapropterin dihydrochloride
  • SOM-7400

最新のパイプライン情報

開発休止中のプロジェクト

製品開発のマイルストーン

  • 注目のニュースとプレスリリース

付録

図表

このページに掲載されている内容は最新版と異なる場合があります。詳細はお問い合わせください。

目次
Product Code: GMDHC9009IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) - Pipeline Review, H1 2017, provides an overview of the Phenylketonuria (PKU) (Metabolic Disorders) pipeline landscape.

Phenylketonuria (also called PKU), is a rare inherited disorder that causes an amino acid called phenylalanine to build up in body. Symptoms include intellectual disability, psychiatric disorders, hyperactivity, skin rashes, poor bone strength and abnormally small head.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Phenylketonuria (PKU) - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Phenylketonuria (PKU) (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Phenylketonuria (PKU) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Phenylketonuria (PKU) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Preclinical and Discovery stages are 1, 9 and 1 respectively.

Phenylketonuria (PKU) (Metabolic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Phenylketonuria (PKU) (Metabolic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Phenylketonuria (PKU) (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Phenylketonuria (PKU) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Phenylketonuria (PKU) (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Phenylketonuria (PKU) (Metabolic Disorders)

Reasons to buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Phenylketonuria (PKU) (Metabolic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Phenylketonuria (PKU) (Metabolic Disorders) pipeline depth and focus of Indication therapeutics.
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Contents

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Phenylketonuria (PKU) - Overview
  • Phenylketonuria (PKU) - Therapeutics Development
    • Pipeline Overview
    • Pipeline by Companies
    • Products under Development by Companies
  • Phenylketonuria (PKU) - Therapeutics Assessment
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Phenylketonuria (PKU) - Companies Involved in Therapeutics Development
    • American Gene Technologies International Inc
    • BioMarin Pharmaceutical Inc
    • Codexis Inc
    • Dimension Therapeutics Inc
    • Erytech Pharma SA
    • MipSalus ApS
    • SOM Biotech SL
    • Synlogic Inc
    • Synthetic Biologics Inc
  • Phenylketonuria (PKU) - Drug Profiles
    • DTX-501 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • pegvaliase - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Phelimine - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Enzyme for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SOM-7400 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SYN-200 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SYNB-2010 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Phenylketonuria (PKU) - Dormant Projects
  • Phenylketonuria (PKU) - Product Development Milestones
    • Featured News & Press Releases
      • Mar 31, 2016: Promising results with breakthrough treatment of orphan disease PKU
      • Mar 21, 2016: BioMarin Phase 3 Study of Pegvaliase for Phenylketonuria Meets Primary Endpoint of Blood Phenylalanine Reduction (p0.0001)
      • Jun 05, 2013: BioMarin Initiates Phase III Trial For PEG-PAL For Treatment Of Phenylketonuria
      • Sep 26, 2012: BioMarin Pharma To Initiate Phase III Program For PEG-PAL In Second Quarter Of 2013
      • Aug 02, 2010: BioMarin Reports Encouraging Preliminary Safety And Efficacy Trends In Phase II Clinical Study Of PEG-PAL In Phenylketonuria
      • Sep 22, 2009: BioMarin Initiates Phase II Clinical Study Of PEG-PAL In PKU
      • Jun 09, 2009: Results From Phase 1 Clinical Study Of PEG-PAL In PKU And Update On Phase 2 Clinical Study
      • May 20, 2008: BioMarin Initiates Phase 1 Clinical Study Of PEG-PAL In PKU
      • Nov 27, 2007: BioMarin Files Investigational New Drug Application For PEG-PAL For The Treatment Of PKU
      • May 16, 2005: BioMarin And Serono Form Strategic Alliance For The Development And Commercialization Of Phenoptin And Phenylase
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Phenylketonuria (PKU), H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Products under Development by Companies, H1 2017
  • Number of Products by Stage and Target, H1 2017
  • Number of Products by Stage and Mechanism of Action, H1 2017
  • Number of Products by Stage and Route of Administration, H1 2017
  • Number of Products by Stage and Molecule Type, H1 2017
  • Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H1 2017
  • Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H1 2017
  • Phenylketonuria (PKU) - Pipeline by Codexis Inc, H1 2017
  • Phenylketonuria (PKU) - Pipeline by Dimension Therapeutics Inc, H1 2017
  • Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H1 2017
  • Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H1 2017
  • Phenylketonuria (PKU) - Pipeline by SOM Biotech SL, H1 2017
  • Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H1 2017
  • Phenylketonuria (PKU) - Pipeline by Synthetic Biologics Inc, H1 2017
  • Phenylketonuria (PKU) - Dormant Projects, H1 2017

List of Figures

  • Number of Products under Development for Phenylketonuria (PKU), H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products by Targets, H1 2017
  • Number of Products by Stage and Targets, H1 2017
  • Number of Products by Mechanism of Actions, H1 2017
  • Number of Products by Stage and Mechanism of Actions, H1 2017
  • Number of Products by Routes of Administration, H1 2017
  • Number of Products by Stage and Routes of Administration, H1 2017
  • Number of Products by Molecule Types, H1 2017
  • Number of Products by Stage and Molecule Types, H1 2017
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