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血友病A:パイプライン分析

Hemophilia A - Pipeline Review, H2 2016

発行 Global Markets Direct 商品コード 232826
出版日 ページ情報 英文 159 Pages
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血友病A:パイプライン分析 Hemophilia A - Pipeline Review, H2 2016
出版日: 2016年11月23日 ページ情報: 英文 159 Pages
概要

血友病 A はX染色体連鎖劣性欠損症で、血液の凝固第VIII因子(FVIII)の不足によって引き起こされ、遺伝あるいは自然突然変異によって発症します。 FVIII活動のレベルに応じて、血友病患者は、あざができやすかったり、外傷を受けても十分に凝固しなかったりし、重度の血友病では自発性出血を伴います。 症状は、痛みと腫れを伴う関節内出血や血尿・血便、あざ、消化管と尿路からの出血、鼻血、切り傷や抜歯、手術の際の長引く出血、突発性出血などが挙げられます。

当レポートでは、血友病Aの治療薬開発パイプラインの現況と最新アップデートによる各開発段階の比較分析、企業や研究機関によって開発中の治療薬、治療薬の評価、後期段階および中止されたプロジェクトに関する情報などを最新のニュースや発表も交えて提供しています。

イントロダクション

  • 調査範囲

血友病 A 概要

治療薬の開発

  • パイプライン製品;概要
  • パイプライン製品;比較分析

企業で開発中の治療薬

大学/機関で研究中の治療薬

パイプライン製品の概況

  • 後期段階の製品
  • 臨床段階の製品
  • 初期段階の製品
  • 開発段階が不明の製品

企業で開発中の製品

大学/機関で研究中の製品

治療薬開発に従事している企業

  • Alnylam Pharmaceuticals, Inc.
  • Amarna Therapeutics B.V.
  • Apitope International NV
  • Baxalta Incorporated
  • Bayer AG
  • Biogen, Inc.
  • BioMarin Pharmaceutical Inc.
  • Catalyst Biosciences, Inc.
  • CSL Limited
  • DBV Technologies S.A.
  • Dimension Therapeutics, Inc.
  • Emergent BioSolutions Inc.
  • EpiVax, Inc.
  • Expression Therapeutics LLC
  • F. Hoffmann-La Roche Ltd.
  • Green Cross Corporation
  • Idogen AB
  • Lentigen Technology, Inc.
  • mAbxience S.A.
  • Novo Nordisk A/S
  • Octapharma AG
  • OPKO Health, Inc.
  • Pfizer Inc.
  • Pharming Group N.V.
  • rEVO Biologics, Inc.
  • Sangamo BioSciences, Inc.
  • SK Chemicals Co., Ltd.
  • Spark Therapeutics, Inc.
  • The International Biotechnology Center (IBC) Generium
  • UniQure N.V.

治療薬の評価

  • 単剤製品
  • 標的別
  • 作用機序別
  • 投与経路別
  • 分子タイプ別

薬剤プロファイル

最近のパイプライン動向

休止中のプロジェクト

開発が中止された製品

製品開発のマイルストーン

  • 主なニュースとプレスリリース

付録

図表

このページに掲載されている内容は最新版と異なる場合があります。詳細はお問い合わせください。

目次
Product Code: GMDHC8692IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Hemophilia A - Pipeline Review, H2 2016, provides an overview of the Hemophilia A (Hematological Disorders) pipeline landscape.

Hemophilia A is an X-linked, recessive disorder caused by deficiency of functional plasma clotting factor VIII (FVIII), which may be inherited or arise from spontaneous mutation. Depending on the level of FVIII activity, patients with hemophilia may present with easy bruising, inadequate clotting of traumatic injury or in the case of severe hemophilia spontaneous hemorrhage. Symptoms may include bleeding into joints, with associated pain and swelling, blood in the urine or stool, bruising, gastrointestinal tract and urinary tract hemorrhage, nosebleeds, prolonged bleeding from cuts, tooth extraction, and surgery and spontaneous bleeding.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Hemophilia A - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Hemophilia A (Hematological 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 Hemophilia A (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Hemophilia A 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 III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 5, 8, 4, 4, 21, 2 and 1 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 3 molecules, respectively.Hemophilia A.

Hemophilia A (Hematological 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 Global Markets Direct's 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 Hemophilia A (Hematological Disorders).
  • The pipeline guide reviews pipeline therapeutics for Hemophilia A (Hematological 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 Hemophilia A (Hematological Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Hemophilia A (Hematological 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 Hemophilia A (Hematological 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 Hemophilia A (Hematological 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 Hemophilia A (Hematological 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.

Table of Contents

  • Table of Contents 2
  • List of Tables 5
  • List of Figures 6
  • Introduction 7
  • Global Markets Direct Report Coverage 7
  • Hemophilia A Overview 8
  • Therapeutics Development 9
  • Pipeline Products for Hemophilia A - Overview 9
  • Pipeline Products for Hemophilia A - Comparative Analysis 10
  • Hemophilia A - Therapeutics under Development by Companies 11
  • Hemophilia A - Therapeutics under Investigation by Universities/Institutes 13
  • Hemophilia A - Pipeline Products Glance 14
  • Late Stage Products 14
  • Clinical Stage Products 15
  • Early Stage Products 16
  • Unknown Stage Products 17
  • Hemophilia A - Products under Development by Companies 18
  • Hemophilia A - Products under Investigation by Universities/Institutes 21
  • Hemophilia A - Companies Involved in Therapeutics Development 22
  • Alnylam Pharmaceuticals Inc 22
  • Amarna Therapeutics BV 23
  • Apitope International NV 24
  • Bayer AG 25
  • Biogen Inc 26
  • BioMarin Pharmaceutical Inc 27
  • Catalyst Biosciences Inc 28
  • Chugai Pharmaceutical Co Ltd 29
  • CSL Ltd 30
  • DBV Technologies SA 31
  • Dimension Therapeutics Inc 32
  • Emergent BioSolutions Inc 33
  • EpiVax Inc 34
  • Expression Therapeutics LLC 35
  • Green Cross Corp 36
  • Idogen AB 37
  • Lentigen Technology Inc 38
  • mAbxience SA 39
  • Novo Nordisk A/S 40
  • Octapharma AG 41
  • OPKO Biologics Ltd 42
  • Pfizer Inc 43
  • Pharming Group NV 44
  • rEVO Biologics Inc 45
  • Sangamo BioSciences Inc 46
  • Shire Plc 47
  • SK Chemicals Co Ltd 48
  • Spark Therapeutics Inc 49
  • UniQure NV 50
  • XL-protein GmbH 51
  • Hemophilia A - Therapeutics Assessment 52
  • Assessment by Monotherapy Products 52
  • Assessment by Target 53
  • Assessment by Mechanism of Action 55
  • Assessment by Route of Administration 57
  • Assessment by Molecule Type 59
  • Drug Profiles 61
  • antihemophilic factor - Drug Profile 61
  • antihemophilic factor (human) - Drug Profile 65
  • antihemophilic factor (recombinant) - Drug Profile 69
  • antihemophilic factor (recombinant) - Drug Profile 70
  • antihemophilic factor (recombinant) - Drug Profile 71
  • antihemophilic factor (recombinant) - Drug Profile 72
  • antihemophilic factor (recombinant) - Drug Profile 73
  • antihemophilic factor (recombinant) - Drug Profile 74
  • antihemophilic factor (recombinant) biosimilar - Drug Profile 75
  • antihemophilic factor (recombinant) biosimilar - Drug Profile 76
  • antihemophilic factor (recombinant), pegylated - Drug Profile 77
  • antihemophilic factor (recombinant), single chain - Drug Profile 79
  • ATXF-8117 - Drug Profile 82
  • BAX-888 - Drug Profile 83
  • BAY-1093884 - Drug Profile 84
  • BMN-270 - Drug Profile 85
  • Cell Therapy to Target Coagulation Factor VIII for Hemophilia A - Drug Profile 87
  • coagulation factor VIIa (recombinant) - Drug Profile 88
  • coagulation factor VIII (human) - Drug Profile 90
  • concizumab - Drug Profile 91
  • CSL-689 - Drug Profile 92
  • damoctocog alfa pegol - Drug Profile 94
  • Deimmunized FVIII - Drug Profile 96
  • DTX-201 - Drug Profile 97
  • emicizumab - Drug Profile 98
  • ET-3 - Drug Profile 101
  • fitusiran - Drug Profile 102
  • Gene Therapy for Hematological Disorders - Drug Profile 108
  • Gene Therapy for Hemophilia A - Drug Profile 109
  • Gene Therapy to Activate Factor VIII for Hemophilia A - Drug Profile 110
  • IN-3012 - Drug Profile 111
  • Kovaltry - Drug Profile 112
  • LG-889 - Drug Profile 114
  • LR-769 - Drug Profile 115
  • marzeptacog alfa - Drug Profile 117
  • MG-1113A - Drug Profile 119
  • MOD-5014 - Drug Profile 120
  • PBB-8-IN - Drug Profile 123
  • PF-06741086 - Drug Profile 124
  • Recombinant Factor VIII Replacement for Hemophilia A - Drug Profile 125
  • Recombinant Factor VIII Replacement for Hemophilia A - Drug Profile 127
  • Recombinant Protein for Hemophilia A - Drug Profile 128
  • Recombinant Proteins for Hemophilia A - Drug Profile 129
  • SB-525 - Drug Profile 130
  • SB-FVIII - Drug Profile 131
  • SHP-656 - Drug Profile 133
  • SPK-8011 - Drug Profile 135
  • SVF-VIIa - Drug Profile 136
  • Hemophilia A - Dormant Projects 137
  • Hemophilia A - Discontinued Products 139
  • Hemophilia A - Product Development Milestones 140
  • Featured News & Press Releases 140
  • Appendix 154
  • Methodology 154
  • Coverage 154
  • Secondary Research 154
  • Primary Research 154
  • Expert Panel Validation 154
  • Contact Us 154
  • Disclaimer 155

List of Tables

  • Number of Products under Development for Hemophilia A, H2 2016 13
  • Number of Products under Development for Hemophilia A - Comparative Analysis, H2 2016 14
  • Number of Products under Development by Companies, H2 2016 15
  • Number of Products under Development by Companies, H2 2016 (Contd..1) 16
  • Number of Products under Investigation by Universities/Institutes, H2 2016 17
  • Comparative Analysis by Late Stage Development, H2 2016 18
  • Comparative Analysis by Clinical Stage Development, H2 2016 19
  • Comparative Analysis by Early Stage Development, H2 2016 20
  • Comparative Analysis by Unknown Stage Development, H2 2016 21
  • Products under Development by Companies, H2 2016 22
  • Products under Development by Companies, H2 2016 (Contd..1) 23
  • Products under Development by Companies, H2 2016 (Contd..2) 24
  • Products under Investigation by Universities/Institutes, H2 2016 25
  • Hemophilia A - Pipeline by Alnylam Pharmaceuticals Inc, H2 2016 26
  • Hemophilia A - Pipeline by Amarna Therapeutics BV, H2 2016 27
  • Hemophilia A - Pipeline by Apitope International NV, H2 2016 28
  • Hemophilia A - Pipeline by Bayer AG, H2 2016 29
  • Hemophilia A - Pipeline by Biogen Inc, H2 2016 30
  • Hemophilia A - Pipeline by BioMarin Pharmaceutical Inc, H2 2016 31
  • Hemophilia A - Pipeline by Catalyst Biosciences Inc, H2 2016 32
  • Hemophilia A - Pipeline by Chugai Pharmaceutical Co Ltd, H2 2016 33
  • Hemophilia A - Pipeline by CSL Ltd, H2 2016 34
  • Hemophilia A - Pipeline by DBV Technologies SA, H2 2016 35
  • Hemophilia A - Pipeline by Dimension Therapeutics Inc, H2 2016 36
  • Hemophilia A - Pipeline by Emergent BioSolutions Inc, H2 2016 37
  • Hemophilia A - Pipeline by EpiVax Inc, H2 2016 38
  • Hemophilia A - Pipeline by Expression Therapeutics LLC, H2 2016 39
  • Hemophilia A - Pipeline by Green Cross Corp, H2 2016 40
  • Hemophilia A - Pipeline by Idogen AB, H2 2016 41
  • Hemophilia A - Pipeline by Lentigen Technology Inc, H2 2016 42
  • Hemophilia A - Pipeline by mAbxience SA, H2 2016 43
  • Hemophilia A - Pipeline by Novo Nordisk A/S, H2 2016 44
  • Hemophilia A - Pipeline by Octapharma AG, H2 2016 45
  • Hemophilia A - Pipeline by OPKO Biologics Ltd, H2 2016 46
  • Hemophilia A - Pipeline by Pfizer Inc, H2 2016 47
  • Hemophilia A - Pipeline by Pharming Group NV, H2 2016 48
  • Hemophilia A - Pipeline by rEVO Biologics Inc, H2 2016 49
  • Hemophilia A - Pipeline by Sangamo BioSciences Inc, H2 2016 50
  • Hemophilia A - Pipeline by Shire Plc, H2 2016 51
  • Hemophilia A - Pipeline by SK Chemicals Co Ltd, H2 2016 52
  • Hemophilia A - Pipeline by Spark Therapeutics Inc, H2 2016 53
  • Hemophilia A - Pipeline by UniQure NV, H2 2016 54
  • Hemophilia A - Pipeline by XL-protein GmbH, H2 2016 55
  • Assessment by Monotherapy Products, H2 2016 56
  • Number of Products by Stage and Target, H2 2016 58
  • Number of Products by Stage and Mechanism of Actions H2 2016 60
  • Number of Products by Stage and Route of Administration, H2 2016 62
  • Number of Products by Stage and Molecule Type, H2 2016 64
  • Hemophilia A - Dormant Projects, H2 2016 141
  • Hemophilia A - Dormant Projects (Contd..1), H2 2016 142
  • Hemophilia A - Discontinued Products, H2 2016 143

List of Figures

  • Number of Products under Development for Hemophilia A, H2 2016 13
  • Number of Products under Development for Hemophilia A - Comparative Analysis, H2 2016 14
  • Number of Products under Development by Companies, H2 2016 15
  • Comparative Analysis by Late Stage Development, H2 2016 18
  • Comparative Analysis by Clinical Stage Development, H2 2016 19
  • Comparative Analysis by Early Stage Products, H2 2016 20
  • Assessment by Monotherapy Products, H2 2016 56
  • Number of Products by Targets, H2 2016 57
  • Number of Products by Stage and Targets, H2 2016 57
  • Number of Products by Mechanism of Actions, H2 2016 59
  • Number of Products by Stage and Mechanism of Actions, H2 2016 59
  • Number of Products by Routes of Administration, H2 2016 61
  • Number of Products by Stage and Routes of Administration, H2 2016 61
  • Number of Products by Top 10 Molecule Types, H2 2016 63
  • Number of Products by Stage and Top 10 Molecule Types, H2 2016 63
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