世界の医療・医薬品産業レポート

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酸性スフィンゴミエリナーゼ欠乏症 (ニーマンピック病) C型 - パイプラインレビュー 2016年上半期
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Pipeline Review, H1 2016

レポートタイトル 酸性スフィンゴミエリナーゼ欠乏症 (ニーマンピック病) C型 - パイプラインレビュー 2016年上半期
出版社名 Global Markets Direct
発刊日 2016-04-20
体裁 PDF / 63 ページ
シングルユーザ価格 US$ 2,000
レポート分類 疾患別治験・パイプラインレポート
地域 グローバル
領域 遺伝子
疾患名 酸性スフィンゴミエリナーゼ欠乏症 (ニーマンピック病) C型
薬剤名 -
企業名 -
備考 ※ ご請求は、お申込日のTTSレートで円換算し、消費税を加えた金額になります。
※ 本レポートは、英文です。PDF(電子媒体)での納品となります。

レポート概要

このレポートは、世界の主要国における上記疾患の治療薬開発についての包括的情報、さまざまな開発ステージでの比較分析、薬剤ターゲット、作用機序(MoA)、投与方法(RoA)、分子タイプ別の薬剤評価、および最新情報、ニュースやプレスリリースなどを提供します。
また、上記疾患における治療薬開発に関わる主要企業概要、開発後期および中止プロジェクトの特集などを提供します。


Global Market Direct社のレポートは、20の治療領域および3000もの適応症をカバーした治験薬を特集しています。
本レポートは、Global Market Direct社独自のデータベース、企業/大学ウェブサイト、SEC書類、企業や大学のウェブサイトからのプレゼンテーションやプレスリリース、および業界特有の第三者情報源、自社分析を基にしたデータや情報を盛り込んでいます。


本レポートは、意思決定能力を高め、競争上の優位性を得るための効果的な戦略を立案するのに役立ちます。
ファーストインクラスやベストインクラス製品を作り出すため、新たなターゲットやMOAを特定することにより、R&Dパイプラインを強化します。


注)本レポートの一部のセクションは、該当疾患におけるデータの可用性や妥当性に基づいて、削除または変更される場合があります。

目次

Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Overview 7
Therapeutics Development 8
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Overview 8
Pipeline Products for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Comparative Analysis 9
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Therapeutics under Development by Companies 10
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Therapeutics under Investigation by Universities/Institutes 11
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Pipeline Products Glance 12
Clinical Stage Products 12
Early Stage Products 13
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Products under Development by Companies 14
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Products under Investigation by Universities/Institutes 15
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Companies Involved in Therapeutics Development 16
CTD Holdings, Inc. 16
La Jolla Pharmaceutical Company 17
Merck & Co., Inc. 18
Okklo Life Sciences BV 19
Orphazyme ApS 20
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? 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
Antibodies for Metabolic Disorders - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
arimoclomol - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
Bryostatin-1 - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
Delta-tocopherol - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
HPGCD - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
LJPC-0712 - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
ML-SA1 - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
OKL-1014 - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
OR-0005 - Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
PERL-101 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
Small Molecules for Niemann-Pick Type C Disease - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Trappsol Cyclo - Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
vorinostat - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
VTS-270 - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Recent Pipeline Updates 50
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C - Dormant Projects 55
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Product Development Milestones 56
Featured News & Press Releases 56
Feb 29, 2016: CTD Holdings Clinical Program to Move Forward in the U.S. 56
Feb 18, 2016: Parent Advocates Join CTD Holdings Family and Physicians Listening Circle on NPC Treatment 56
Dec 14, 2015: CTD Holdings' Clinical Trial Design Receives Positive Responses From U.K.'s Medicines and Healthcare Products Regulatory Agency 57
Jun 24, 2015: Neurotrope to Conduct Study on Niemann-Pick Type C, a Devastating Rare Pediatric Disease 58
Jun 12, 2015: Neurotrope to Present Findings of In-vitro Studies of Bryostatin's Effects on Niemann Pick Type C1 Cells at the Annual "Michael, Marcia & Christa Parseghian Scientific Conference" for Niemann-Pick Type C 58
Apr 15, 2015: Orphazyme Forms Scientific Advisory Board and Confirms the Enrolment Initiation for the Clinical Programme 59
Jan 26, 2015: FDA grants orphan drug designation for Arimoclomol 60
Jan 26, 2015: Orphazyme to present at the Gordon Research Conference on Lysosomal Diseases 15-19 March in Galveston, TX 60
Oct 20, 2014: COMP issues positive opinion on orphan drug application for Arimoclomol 60
Oct 06, 2014: Orphazyme in final preparation stages for a Phase II/III clinical trial 61
Appendix 62
Methodology 62
Coverage 62
Secondary Research 62
Primary Research 62
Expert Panel Validation 62
Contact Us 62
Disclaimer 63

List of Tables
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2016 8
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Comparative Analysis, H1 2016 9
Number of Products under Development by Companies, H1 2016 10
Number of Products under Investigation by Universities/Institutes, H1 2016 11
Comparative Analysis by Clinical Stage Development, H1 2016 12
Comparative Analysis by Early Stage Development, H1 2016 13
Products under Development by Companies, H1 2016 14
Products under Investigation by Universities/Institutes, H1 2016 15
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Pipeline by CTD Holdings, Inc., H1 2016 16
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Pipeline by La Jolla Pharmaceutical Company, H1 2016 17
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Pipeline by Merck & Co., Inc., H1 2016 18
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Pipeline by Okklo Life Sciences BV, H1 2016 19
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Pipeline by Orphazyme ApS, H1 2016 20
Assessment by Monotherapy Products, H1 2016 21
Number of Products by Stage and Target, H1 2016 23
Number of Products by Stage and Mechanism of Action, H1 2016 25
Number of Products by Stage and Route of Administration, H1 2016 27
Number of Products by Stage and Molecule Type, H1 2016 29
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C Therapeutics ? Recent Pipeline Updates, H1 2016 50
Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Dormant Projects, H1 2016 55

List of Figures
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C, H1 2016 8
Number of Products under Development for Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) Type C ? Comparative Analysis, H1 2016 9
Number of Products under Development by Companies, H1 2016 10
Number of Products under Investigation by Universities/Institutes, H1 2016 11
Comparative Analysis by Clinical Stage Development, H1 2016 12
Comparative Analysis by Early Stage Products, H1 2016 13
Assessment by Monotherapy Products, H1 2016 21
Number of Products by Top 10 Targets, H1 2016 22
Number of Products by Stage and Top 10 Targets, H1 2016 22
Number of Products by Top 10 Mechanism of Actions, H1 2016 24
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2016 24
Number of Products by Routes of Administration, H1 2016 26
Number of Products by Stage and Routes of Administration, H1 2016 26
Number of Products by Molecule Types, H1 2016 28
Number of Products by Stage and Molecule Types, H1 2016 28
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