Table of Content


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 Intertiol Inc
APR Applied Pharma Research SA
BioMarin Pharmaceutical Inc
Evox Therapeutics Ltd
Generation Bio Co
Homology Medicines Inc
MipSalus ApS
Moder Inc
Nestle Health Science SA
Peg-Bio BioPharma Co Ltd
Pluvia AS
Prosit Sole Biotechnology (Beijing) Co Ltd
PTC Therapeutics Inc
Sangamo Therapeutics Inc
SOM Biotech SL
Synlogic Inc
Ultragenyx Pharmaceutical Inc
Vera Therapeutics Inc
Phenylketonuria (PKU) - Drug Profiles
AGT-324 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
APROD-031 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Biologic for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Biologics for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BMN-307 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CDX-6114 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Phenylketonuria and Progressive Familial Intrahepatic Cholestasis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
HMI-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
HMI-103 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
mR-3283 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides to Activate PAH for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
pegvaliase - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Phelimin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PJ-008 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PSP-007 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PTC-923 - 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
Small Molecules to Target PAH for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SOM-1311 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ST-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SYNB-1618 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
UX-501 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Phenylketonuria (PKU) - Dormant Projects
Phenylketonuria (PKU) - Discontinued Products
Phenylketonuria (PKU) - Product Development Milestones
Featured News & Press Releases
Oct 07, 2020: BioMarin, pioneer in rare disease treatments for Phenylketonuria (PKU), receives FDA approval of label expansion to allow maximum dose of 60 mg for Palynziq (pegvaliase-pqpz) injection for treatment of adults with PKU
Mar 26, 2020: FDA grants Orphan Drug Desigtion to APR-OD031 for the treatment of phenylketonuria
Mar 10, 2020: American Gene Technologies to present at 2020 Inborn Errors of Metabolism Drug Development Summit in Boston
Dec 11, 2019: Synlogic bridging study data with solid oral formulation of SYNB1618, a synthetic biotic approach to treat Phenylketonuria, demonstrate improved tolerability over early liquid formulation and guide to next stage of clinical development
Dec 03, 2019: Censa Pharmaceuticals announces CNSA- 001 met primary and secondary endpoints in phase 2 trial in patients with PKU
Sep 04, 2019: Synlogic presents data from phase 1/2a study of SYNB1618 at the Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM)
Jul 15, 2019: Synlogic reports positive top-line data from phase 1/2a study of SYNB1618 in patients with phenylketonuria and guides to next phase of development
May 06, 2019: European Commission approves Palynziq (pegvaliase injection) for treatment of phenylketonuria (PKU) in patients aged 16 years or older
Apr 30, 2019: Synlogic presents data describing a Solid Oral Formulation Process for Synthetic Biotic Medicine SYNB1618 for the treatment of PKU at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
Apr 15, 2019: Synlogic to present data from new solid oral formulation process at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
Mar 01, 2019: BioMarin Receives Positive CHMP Opinion in Europe for Palynziq (pegvaliase Injection) for Treatment of Patients with Phenylketonuria (PKU) Aged 16 and Older
Jan 07, 2019: Retrophin provides update on phenylketonuria drug CNSA-001
Jan 07, 2019: BioMarin highlights Palynziq at 37th Annual J.P. Morgan Healthcare Conference in San Francisco
Jan 07, 2019: Homology Medicines advances first gene editing development candidate into IND-ebling studies for pediatric patients with phenylketonuria
Jan 03, 2019: Synlogic provides update on synthetic biotic medicine SYNB-1618
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Number of Products under Development for Phenylketonuria (PKU), 2021
Number of Products under Development by Companies, 2021
Number of Products by Top 10 Targets, 2021
Number of Products by Stage and Top 10 Targets, 2021
Number of Products by Top 10 Mechanism of Actions, 2021
Number of Products by Stage and Top 10 Mechanism of Actions, 2021
Number of Products by Top 10 Routes of Administration, 2021
Number of Products by Stage and Top 10 Routes of Administration, 2021
Number of Products by Top 10 Molecule Types, 2021
Number of Products by Stage and Top 10 Molecule Types, 2021


List of Tables


Number of Products under Development for Phenylketonuria (PKU), 2021
Number of Products under Development by Companies, 2021
Products under Development by Companies, 2021
Products under Development by Companies, 2021 (Contd..1), 2021
Number of Products by Stage and Target, 2021
Number of Products by Stage and Mechanism of Action, 2021
Number of Products by Stage and Route of Administration, 2021
Number of Products by Stage and Molecule Type, 2021
Phenylketonuria (PKU) - Pipeline by Agios Pharmaceuticals Inc, 2021
Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, 2021
Phenylketonuria (PKU) - Pipeline by APR Applied Pharma Research SA, 2021
Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, 2021
Phenylketonuria (PKU) - Pipeline by Evox Therapeutics Ltd, 2021
Phenylketonuria (PKU) - Pipeline by Generation Bio Co, 2021
Phenylketonuria (PKU) - Pipeline by Homology Medicines Inc, 2021
Phenylketonuria (PKU) - Pipeline by MipSalus ApS, 2021
Phenylketonuria (PKU) - Pipeline by Moderna Inc, 2021
Phenylketonuria (PKU) - Pipeline by Nestle Health Science SA, 2021
Phenylketonuria (PKU) - Pipeline by Peg-Bio BioPharma Co Ltd, 2021
Phenylketonuria (PKU) - Pipeline by Pluvia AS, 2021
Phenylketonuria (PKU) - Pipeline by Prosit Sole Biotechnology (Beijing) Co Ltd, 2021
Phenylketonuria (PKU) - Pipeline by PTC Therapeutics Inc, 2021
Phenylketonuria (PKU) - Pipeline by Sangamo Therapeutics Inc, 2021
Phenylketonuria (PKU) - Pipeline by SOM Biotech SL, 2021
Phenylketonuria (PKU) - Pipeline by Synlogic Inc, 2021
Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, 2021
Phenylketonuria (PKU) - Pipeline by Vera Therapeutics Inc, 2021
Phenylketonuria (PKU) - Dormant Projects, 2021
Phenylketonuria (PKU) - Discontinued Products, 2021