酸性スフィンゴミエリナーゼ欠乏症(Acid Sphingomyelinase Deficiency/ニーマンピック病)：治療薬開発パイプライン動向（2015年上半期版）...市場調査レポートについてご紹介

Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Overview 7
Therapeutics Development 8
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Overview 8
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Comparative Analysis 9
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Therapeutics under Development by Companies 10
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Therapeutics under Investigation by Universities/Institutes 11
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Pipeline Products Glance 12
Clinical Stage Products 12
Early Stage Products 13
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Products under Development by Companies 14
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Products under Investigation by Universities/Institutes 15
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Companies Involved in Therapeutics Development 16
Genzyme Corporation 16
La Jolla Pharmaceutical Company 17
Merck & Co., Inc. 18
Okklo Life Sciences BV 19
Orphazyme ApS 20
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Therapeutics Assessment 21
Assessment by Monotherapy Products 21
Assessment by Target 22
Assessment by Mechanism of Action 24
Assessment by Route of Administration 26
Assessment by Molecule Type 28
Drug Profiles 30
arimoclomol – Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
Delta-tocopherol – Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
LJPC-0712 – Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
ML-SA1 – Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
OKL-1014 – Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
olipudase alfa – Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
OR-0005 – Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
Stem Cell Therapy for Cancer, Pain, Retinal Degeneration and Niemann-Pick Disease – Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
vorinostat – Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
VTS-270 – Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Recent Pipeline Updates 44
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Dormant Projects 46
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) – Product Development Milestones 47
Featured News & Press Releases 47
Jan 26, 2015: FDA grants orphan drug designation for Arimoclomol 47
Oct 20, 2014: COMP issues positive opinion on orphan drug application for Arimoclomol 47
Oct 06, 2014: Orphazyme in final preparation stages for a Phase II/III clinical trial 47
Jul 17, 2013: La Jolla Pharmaceutical Company Receives Orphan Designation From FDA 48
Apr 03, 2013: Old Drug Offers New Treatment For Niemann-Pick Type C, Study Finds 49
Feb 21, 2013: Orphazyme Receives Orphan Drug Designation For OR0005 In EU 50
Jan 23, 2013: NIH Clinical Trial Begins For Treatment Of Niemann-Pick Disease Type C1 51
Mar 13, 2012: U-M biologists Find Potential Drug That Speeds Cellular Recycling 52
Appendix 54
Methodology 54
Coverage 54
Secondary Research 54
Primary Research 54
Expert Panel Validation 54
Contact Us 54
Disclaimer 55