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市場調查報告書

苯酮尿症 - 開發中產品分析

Phenylketonuria (PKU) - Pipeline Review, H1 2018

出版商 Global Markets Direct 商品編碼 321900
出版日期 內容資訊 英文 53 Pages
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苯酮尿症 - 開發中產品分析 Phenylketonuria (PKU) - Pipeline Review, H1 2018
出版日期: 2018年01月30日 內容資訊: 英文 53 Pages
簡介

苯酮尿症是罕見的遺傳疾病,是由於必需氨基酸的苯丙胺酸所引起。症狀有智力障礙、精神遲緩、過動、皮疹、骨骼強度不足,頭部異常小等。

本報告提供苯酮尿症治療藥的開發情形調查分析,提供您開發中產品的概要,臨床實驗的各階段的產品概要,主要企業簡介,藥物簡介,開發中產品的最新趨勢,最新消息和新聞稿等相關的系統性資訊。

目錄

簡介

苯酮尿症概要

治療藥的開發

  • 開發中產品:概要
  • 開發中產品:比較分析

開發中的治療藥:各企業

開發中產品的概要

  • 後期階段的產品
  • 初期階段的產品

開發中的產品:各企業

治療藥的開發企業

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

治療藥的評估

  • 單獨療法的產品
  • 各給藥途徑
  • 各分子類型

藥物簡介

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

最新的開發平台資訊

開發暫停中的計劃

產品開發的里程碑

  • 熱門的新聞和新聞稿

附錄

圖表

本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

目錄
Product Code: GMDHC10117IDB

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) - Pipeline Review, H1 2018, 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 2018, 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 Pre-Registration, Phase I and Preclinical stages are 1, 1 and 9 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

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
  • Censa Pharmaceuticals Inc
  • Codexis Inc
  • EryDel SPA
  • Erytech Pharma SA
  • MipSalus ApS
  • Synlogic Inc
  • Synthetic Biologics Inc
  • Ultragenyx Pharmaceutical Inc
  • Phenylketonuria (PKU) - Drug Profiles
  • CDX-6114 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • CNSA-001 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • 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 Ammonia Lyase 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
  • SYN-200 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SYNB-1618 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Phenylketonuria (PKU) - Dormant Projects
  • Phenylketonuria (PKU) - Product Development Milestones
  • Featured News & Press Releases
  • Jan 05, 2018: Synlogic Provides Update on its Phenylketonuria Drug candidate SYNB1618
  • Dec 22, 2017: BioMarin Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28, 2018
  • Oct 24, 2017: Synlogic Receives Orphan Drug Designation for SYNB1618, a Synthetic Biotic Medicine for the Treatment of Phenylketonuria
  • Sep 14, 2017: FDA Not Currently Planning to Hold Advisory Committee Meeting for BioMarins Pegvaliase Biologics License Application (BLA)
  • Sep 05, 2017: BioMarin To Present Data On Pegvaliase At 13th International Congress of Inborn Errors of Metabolism 2017
  • Aug 29, 2017: FDA Accepts BioMarins Pegvaliase Biologics License Application and Grants Priority Review Designation
  • Jun 30, 2017: BioMarin Submits Pegvaliase Biologics License Application to the U.S. FDA for Treatment of Phenylketonuria
  • 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)
  • Feb 28, 2014: Grant for development of breakthrough treatment of PKU
  • 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
  • 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 2018
  • Number of Products under Development by Companies, H1 2018
  • Products under Development by Companies, H1 2018
  • Number of Products by Stage and Target, H1 2018
  • Number of Products by Stage and Mechanism of Action, H1 2018
  • Number of Products by Stage and Route of Administration, H1 2018
  • Number of Products by Stage and Molecule Type, H1 2018
  • Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H1 2018
  • Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H1 2018
  • Phenylketonuria (PKU) - Pipeline by Censa Pharmaceuticals Inc, H1 2018
  • Phenylketonuria (PKU) - Pipeline by Codexis Inc, H1 2018
  • Phenylketonuria (PKU) - Pipeline by EryDel SPA, H1 2018
  • Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H1 2018
  • Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H1 2018
  • Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H1 2018
  • Phenylketonuria (PKU) - Pipeline by Synthetic Biologics Inc, H1 2018
  • Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, H1 2018
  • Phenylketonuria (PKU) - Dormant Projects, H1 2018

List of Figures

  • Number of Products under Development for Phenylketonuria (PKU), H1 2018
  • Number of Products under Development by Companies, H1 2018
  • Number of Products by Targets, H1 2018
  • Number of Products by Stage and Targets, H1 2018
  • Number of Products by Mechanism of Actions, H1 2018
  • Number of Products by Stage and Mechanism of Actions, H1 2018
  • Number of Products by Routes of Administration, H1 2018
  • Number of Products by Stage and Routes of Administration, H1 2018
  • Number of Products by Molecule Types, H1 2018
  • Number of Products by Stage and Molecule Types, H1 2018
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