Phenylketonuria (PKU) - Pipeline Review, H1 2018 - Product Image

Phenylketonuria (PKU) - Pipeline Review, H1 2018

  • ID: 4457546
  • Report
  • 53 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Censa Pharmaceuticals Inc
  • Codexis Inc
  • EryDel SPA
  • Erytech Pharma SA
  • MipSalus ApS
  • Synlogic Inc
  • MORE
Phenylketonuria (PKU) - Pipeline Review, H1 2018

Summary

The 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

The latest 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 our 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.
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Note: Product cover images may vary from those shown
2 of 5

FEATURED COMPANIES

  • Censa Pharmaceuticals Inc
  • Codexis Inc
  • EryDel SPA
  • Erytech Pharma SA
  • MipSalus ApS
  • Synlogic Inc
  • MORE
Introduction

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,

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

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

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

Number of Products under Development by Companies, H1

Products under Development by Companies, H1

Number of Products by Stage and Target, H1

Number of Products by Stage and Mechanism of Action, H1

Number of Products by Stage and Route of Administration, H1

Number of Products by Stage and Molecule Type, H1

Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H1

Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H1

Phenylketonuria (PKU) - Pipeline by Censa Pharmaceuticals Inc, H1

Phenylketonuria (PKU) - Pipeline by Codexis Inc, H1

Phenylketonuria (PKU) - Pipeline by EryDel SPA, H1

Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H1

Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H1

Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H1

Phenylketonuria (PKU) - Pipeline by Synthetic Biologics Inc, H1

Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, H1

Phenylketonuria (PKU) - Dormant Projects, H1

List of Figures

Number of Products under Development for Phenylketonuria (PKU), H1

Number of Products under Development by Companies, H1

Number of Products by Targets, H1

Number of Products by Stage and Targets, H1

Number of Products by Mechanism of Actions, H1

Number of Products by Stage and Mechanism of Actions, H1

Number of Products by Routes of Administration, H1

Number of Products by Stage and Routes of Administration, H1

Number of Products by Molecule Types, H1

Number of Products by Stage and Molecule Types, H
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  • 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
Note: Product cover images may vary from those shown
5 of 5
Note: Product cover images may vary from those shown
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