【レポートの概要（一部）】
Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Phenylketonuria (PKU) Overview 6
Therapeutics Development 7
Pipeline Products for Phenylketonuria (PKU) – Overview 7
Pipeline Products for Phenylketonuria (PKU) – Comparative Analysis 8
Phenylketonuria (PKU) – Therapeutics under Development by Companies 9
Phenylketonuria (PKU) – Pipeline Products Glance 10
Late Stage Products 10
Early Stage Products 11
Phenylketonuria (PKU) – Products under Development by Companies 12
Phenylketonuria (PKU) – Companies Involved in Therapeutics Development 13
BioMarin Pharmaceutical Inc. 13
Codexis, Inc. 14
Erytech Pharma SA 15
Promethera Biosciences S.A. 16
SOM Innovation Biotech SL 17
Phenylketonuria (PKU) – Therapeutics Assessment 18
Assessment by Monotherapy Products 18
Assessment by Target 19
Assessment by Route of Administration 20
Assessment by Molecule Type 22
Drug Profiles 24
BMN-165 – Drug Profile 24
Product Description 24
Mechanism of Action 24
R&D Progress 24
Cell therapy for Phenylketonuria – Drug Profile 25
Product Description 25
Mechanism of Action 25
R&D Progress 25
HepaStem – Drug Profile 26
Product Description 26
Mechanism of Action 26
R&D Progress 26
Recombinant Enzyme to Replace Phenylalanine Hydroxylase for Phenyketonuria – Drug Profile 28
Product Description 28
Mechanism of Action 28
R&D Progress 28
sapropterin dihydrochloride – Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
SOM-7400 – Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
Phenylketonuria (PKU) – Recent Pipeline Updates 32
Phenylketonuria (PKU) – Dormant Projects 35
Phenylketonuria (PKU) – Product Development Milestones 36
Featured News & Press Releases 36
Sep 24, 2014: FDA Receives Paragraph IV Notice Letter for KUVAN (sapropterin dihydrochloride) Tablets 36
Sep 03, 2014: Merck Announces Detailed 26-Week Results from Phase IIIb Study with Kuvan in Children with PKU Below 4 Years of Age 36
Apr 14, 2014: FDA Extends Market Exclusivity Six Months for BioMarin’s Rare Disease Therapy KUVAN (sapropterin dihydrochloride) Powder for Oral Solution and Tablets 38
Jun 05, 2013: BioMarin Initiates Phase III Trial For PEG-PAL For Treatment Of Phenylketonuria 39
Feb 19, 2013: BioMarin Announces Kuvan Significantly Improves Inattentiveness In Phenylketonuria Patients 39
Sep 26, 2012: BioMarin Pharma To Initiate Phase III Program For PEG-PAL In Second Quarter Of 2013 40
Jun 30, 2011: Merck Serono Initiates Phase IIIb European Study SPARK In Children Younger Than Four Years, Suffering From Phenylketonuria 42
Mar 18, 2011: BioMarin Announces Preliminary Results From ADAPT Study 42
Sep 01, 2010: BioMarin Presents Results From Investigator-Sponsored Trials On Kuvan At SSIEM Annual Conference 44
Aug 17, 2010: BioMarin Initiates Phase IIIb Study To Evaluate Effects Of Kuvan On Neurophychiatric Symptoms In Subjects With Phenylketonuria 45
Appendix 46
Methodology 46
Coverage 46
Secondary Research 46
Primary Research 46
Expert Panel Validation 46
Contact Us 47
Disclaimer 47
【レポート販売概要】
■ タイトル：フェニルケトン尿症(Phenylketonuria (PKU))の治療薬パイプライン動向（2015年上半期版）■ 英文：Phenylketonuria (PKU) - Pipeline Review, H1 2015
■ 発行日：2015年1月22日
■ 調査会社：Global Markets Direct
■ 商品コード：GMDHC6110IDB
■ 調査対象地域：グローバル
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