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Market Research Report

Phenylketonuria (PKU) - Pipeline Review, H2 2018

Published by Global Markets Direct Product code 321900
Published Content info 64 Pages
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Phenylketonuria (PKU) - Pipeline Review, H2 2018
Published: July 17, 2018 Content info: 64 Pages
Description

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) - Pipeline Review, H2 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, H2 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 II, Phase I, Preclinical and Discovery stages are 1, 1, 2, 9 and 3 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
Product Code: GMDHC10647IDB

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
    • Agios Pharmaceuticals Inc
    • American Gene Technologies International Inc
    • BioMarin Pharmaceutical Inc
    • Censa Pharmaceuticals Inc
    • Codexis Inc
    • Erytech Pharma SA
    • Homology Medicines, Inc.
    • MipSalus ApS
    • Rubius Therapeutics Inc
    • Synlogic Inc
    • Synthetic Biologics Inc
    • Ultragenyx Pharmaceutical Inc
  • Phenylketonuria (PKU) - Drug Profiles
    • BMN-307 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • 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
    • Gene Therapy for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • HMI-102 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • pegvaliase - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Peptides for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Phelimin - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Enzyme for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RTX-134 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules 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
      • Jul 09, 2018: Codexis Doses First Subjects in Phase 1a Trial of CDX-6114
      • May 24, 2018: BioMarin Receives Standard Approval for Palynziq (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease
      • May 18, 2018: Homology Medicines Presents Data from PKU Gene Therapy Program Demonstrating Long-Term Durability of Treatment in IND-Enabling Studies
      • Apr 30, 2018: Homology Medicines Announces Upcoming Presentations on its Genetic Medicines Platform at the American Society of Gene & Cell Therapy Meeting
      • Mar 28, 2018: European Medicines Agency Accepts BioMarin's Marketing Application for Pegvaliase MAA for Treatment of Phenylketonuria
      • Dec 22, 2017: BioMarin Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28, 2018
      • 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
      • Jul 31, 2017: MipSalus: Promising Results from Preliminary Safety Study
      • Jun 30, 2017: BioMarin Submits Pegvaliase Biologics License Application to the U.S. FDA for Treatment of Phenylketonuria
      • Apr 30, 2017: MipSalus: Multi-dose studies with Phelimin show high efficacy and flexibility
      • 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
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
  • Disclaimer

List of Tables

  • Number of Products under Development for Phenylketonuria (PKU), H2 2018
  • Number of Products under Development by Companies, H2 2018
  • Products under Development by Companies, H2 2018
  • Number of Products by Stage and Target, H2 2018
  • Number of Products by Stage and Mechanism of Action, H2 2018
  • Number of Products by Stage and Route of Administration, H2 2018
  • Number of Products by Stage and Molecule Type, H2 2018
  • Phenylketonuria (PKU) - Pipeline by Agios Pharmaceuticals Inc, H2 2018
  • Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H2 2018
  • Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H2 2018
  • Phenylketonuria (PKU) - Pipeline by Censa Pharmaceuticals Inc, H2 2018
  • Phenylketonuria (PKU) - Pipeline by Codexis Inc, H2 2018
  • Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H2 2018
  • Phenylketonuria (PKU) - Pipeline by Homology Medicines, Inc., H2 2018
  • Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H2 2018
  • Phenylketonuria (PKU) - Pipeline by Rubius Therapeutics Inc, H2 2018
  • Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H2 2018
  • Phenylketonuria (PKU) - Pipeline by Synthetic Biologics Inc, H2 2018
  • Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, H2 2018
  • Phenylketonuria (PKU) - Dormant Projects, H2 2018

List of Figures

  • Number of Products under Development for Phenylketonuria (PKU), H2 2018
  • Number of Products under Development by Companies, H2 2018
  • Number of Products by Targets, H2 2018
  • Number of Products by Stage and Targets, H2 2018
  • Number of Products by Mechanism of Actions, H2 2018
  • Number of Products by Stage and Mechanism of Actions, H2 2018
  • Number of Products by Routes of Administration, H2 2018
  • Number of Products by Stage and Routes of Administration, H2 2018
  • Number of Products by Molecule Types, H2 2018
  • Number of Products by Stage and Molecule Types, H2 2018
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