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Hemophilia A - Pipeline Review, H1 2016

Published: May 25, 2016 | Pages: 159 | Publisher: Global Markets Direct | Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Hemophilia A - Pipeline Review, H1 2016


Global Markets Direct's, 'Hemophilia A - Pipeline Review, H1 2016', provides an overview of the Hemophilia A pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Hemophilia A, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Hemophilia A and features dormant and discontinued projects. 

Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.


- The report provides a snapshot of the global therapeutic landscape of Hemophilia A
- The report reviews pipeline therapeutics for Hemophilia A by companies and universities/research institutes based on information derived from company and industry-specific sources 
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages 
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved Hemophilia A therapeutics and enlists all their major and minor projects
- The report assesses Hemophilia A therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type 
- The report summarizes all the dormant and discontinued pipeline projects 
- The report reviews latest news related to pipeline therapeutics for Hemophilia A

Reasons To Buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage 
- Identify and understand important and diverse types of therapeutics under development for Hemophilia A
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies 
- Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Hemophilia A 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
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
 Table of Contents
Table of Contents 2 List of Tables 8 List of Figures 10 Introduction 11 Global Markets Direct Report Coverage 11 Hemophilia A Overview 12 Therapeutics Development 13 Pipeline Products for Hemophilia A - Overview 13 Pipeline Products for Hemophilia A - Comparative Analysis 14 Hemophilia A - Therapeutics under Development by Companies 15 Hemophilia A - Therapeutics under Investigation by Universities/Institutes 17 Hemophilia A - Pipeline Products Glance 18 Late Stage Products 18 Clinical Stage Products 19 Early Stage Products 20 Unknown Stage Products 21 Hemophilia A - Products under Development by Companies 22 Hemophilia A - Products under Investigation by Universities/Institutes 25 Hemophilia A - Companies Involved in Therapeutics Development 26 Alnylam Pharmaceuticals, Inc. 26 Amarna Therapeutics B.V. 27 Apitope International NV 28 Baxalta Incorporated 29 Bayer AG 30 Biogen, Inc. 31 BioMarin Pharmaceutical Inc. 32 Catalyst Biosciences, Inc. 33 CSL Limited 34 DBV Technologies S.A. 35 Dimension Therapeutics, Inc. 36 Emergent BioSolutions Inc. 37 EpiVax, Inc. 38 Expression Therapeutics LLC 39 F. Hoffmann-La Roche Ltd. 40 Green Cross Corporation 41 Idogen AB 42 Lentigen Technology, Inc. 43 mAbxience S.A. 44 Novo Nordisk A/S 45 Octapharma AG 46 OPKO Health, Inc. 47 Pfizer Inc. 48 Pharming Group N.V. 49 rEVO Biologics, Inc. 50 Sangamo BioSciences, Inc. 51 SK Chemicals Co., Ltd. 52 Spark Therapeutics, Inc. 53 The International Biotechnology Center (IBC) Generium 54 UniQure N.V. 55 Hemophilia A - Therapeutics Assessment 56 Assessment by Monotherapy Products 56 Assessment by Target 57 Assessment by Mechanism of Action 59 Assessment by Route of Administration 61 Assessment by Molecule Type 63 Drug Profiles 65 antihemophilic factor - Drug Profile 65 Product Description 65 Mechanism of Action 65 R&D Progress 65 antihemophilic factor (human) - Drug Profile 67 Product Description 67 Mechanism of Action 67 R&D Progress 67 antihemophilic factor (recombinant) - Drug Profile 69 Product Description 69 Mechanism of Action 69 R&D Progress 69 antihemophilic factor (recombinant) - Drug Profile 70 Product Description 70 Mechanism of Action 70 R&D Progress 70 antihemophilic factor (recombinant) - Drug Profile 72 Product Description 72 Mechanism of Action 72 R&D Progress 72 antihemophilic factor (recombinant) - Drug Profile 73 Product Description 73 Mechanism of Action 73 R&D Progress 73 antihemophilic factor (recombinant) - Drug Profile 74 Product Description 74 Mechanism of Action 74 R&D Progress 74 antihemophilic factor (recombinant) biosimilar - Drug Profile 75 Product Description 75 Mechanism of Action 75 R&D Progress 75 ATXF-8117 - Drug Profile 76 Product Description 76 Mechanism of Action 76 R&D Progress 76 BAX-826 - Drug Profile 77 Product Description 77 Mechanism of Action 77 R&D Progress 77 BAX-888 - Drug Profile 78 Product Description 78 Mechanism of Action 78 R&D Progress 78 BAY-1093884 - Drug Profile 79 Product Description 79 Mechanism of Action 79 R&D Progress 79 BMN-270 - Drug Profile 80 Product Description 80 Mechanism of Action 80 R&D Progress 80 CB-813d - Drug Profile 81 Product Description 81 Mechanism of Action 81 R&D Progress 81 Cell Therapy to Target Coagulation Factor VIII for Hemophilia A - Drug Profile 83 Product Description 83 Mechanism of Action 83 R&D Progress 83 coagulation factor VIIa (recombinant) - Drug Profile 84 Product Description 84 Mechanism of Action 84 R&D Progress 84 coagulation factor VIII (human) - Drug Profile 86 Product Description 86 Mechanism of Action 86 R&D Progress 86 concizumab - Drug Profile 87 Product Description 87 Mechanism of Action 87 R&D Progress 87 CSL-689 - Drug Profile 88 Product Description 88 Mechanism of Action 88 R&D Progress 88 damoctocog alfa pegol - Drug Profile 90 Product Description 90 Mechanism of Action 90 R&D Progress 90 Deimmunized FVIII - Drug Profile 91 Product Description 91 Mechanism of Action 91 R&D Progress 91 DTX-201 - Drug Profile 93 Product Description 93 Mechanism of Action 93 R&D Progress 93 emicizumab - Drug Profile 94 Product Description 94 Mechanism of Action 94 R&D Progress 94 ET-3 - Drug Profile 95 Product Description 95 Mechanism of Action 95 R&D Progress 95 factor VIII (recombinant) biosimilar - Drug Profile 96 Product Description 96 Mechanism of Action 96 R&D Progress 96 fitusiran - Drug Profile 97 Product Description 97 Mechanism of Action 97 R&D Progress 97 Gene Therapy for Bleeding and Clotting Disorders - Drug Profile 98 Product Description 98 Mechanism of Action 98 R&D Progress 98 Gene Therapy for Hemophilia A - Drug Profile 99 Product Description 99 Mechanism of Action 99 R&D Progress 99 Gene Therapy to Activate Factor VIII for Hemophilia A - Drug Profile 100 Product Description 100 Mechanism of Action 100 R&D Progress 100 IN-3012 - Drug Profile 101 Product Description 101 Mechanism of Action 101 R&D Progress 101 Kovaltry - Drug Profile 102 Product Description 102 Mechanism of Action 102 R&D Progress 102 LG-889 - Drug Profile 104 Product Description 104 Mechanism of Action 104 R&D Progress 104 LR-769 - Drug Profile 105 Product Description 105 Mechanism of Action 105 R&D Progress 105 MOD-5014 - Drug Profile 107 Product Description 107 Mechanism of Action 107 R&D Progress 107 PF-06741086 - Drug Profile 108 Product Description 108 Mechanism of Action 108 R&D Progress 108 Recombinant Factor VIII Replacement for Hemophilia A - Drug Profile 109 Product Description 109 Mechanism of Action 109 R&D Progress 109 Recombinant Factor VIII Replacement for Hemophilia A - Drug Profile 110 Product Description 110 Mechanism of Action 110 R&D Progress 110 Recombinant Protein for Hemophilia A - Drug Profile 111 Product Description 111 Mechanism of Action 111 R&D Progress 111 Recombinant Proteins for Hemophilia A - Drug Profile 112 Product Description 112 Mechanism of Action 112 R&D Progress 112 SB-FVIII - Drug Profile 113 Product Description 113 Mechanism of Action 113 R&D Progress 113 SPK-FVIII - Drug Profile 114 Product Description 114 Mechanism of Action 114 R&D Progress 114 SVF-VIIa - Drug Profile 115 Product Description 115 Mechanism of Action 115 R&D Progress 115 turoctocog alfa pegol - Drug Profile 116 Product Description 116 Mechanism of Action 116 R&D Progress 116 Hemophilia A - Recent Pipeline Updates 118 Hemophilia A - Dormant Projects 147 Hemophilia A - Discontinued Products 149 Hemophilia A - Product Development Milestones 150 Featured News & Press Releases 150 May 07, 2016: Dimension Therapeutics Announces Preclinical Data From Bayer-Partnered Hemophilia A Program At American Society For Gene And Cell Therapy (ASGCT) Annual Meeting 150 Apr 18, 2016: Dimension Therapeutics announces preclinical data on hemophilia A program at upcoming American Society For Gene And Cell Therapy (ASGCT) annual meeting 150 Apr 04, 2016: Baxalta Expands Global Reach of ADYNOVATE for Hemophilia A Patients with Approval in Japan 152 Mar 17, 2016: Bayer Receives FDA Approval for Kovaltry for the Treatment of Children and Adults with Hemophilia A 152 Mar 04, 2016: Baxalta Commences Phase 1 Clinical Trial of BAX 826, the Company's Second Extended Half-Life Factor VIII Treatment for Hemophilia A 153 Mar 02, 2016: Baxalta Submits Marketing Authorization Application to the European Medicines Agency for ADYNOVI for Treatment of Hemophilia A Patients 154 Feb 25, 2016: Baxalta Announces Submission of Supplemental BLAs to Expand Use of ADYNOVATE to Pediatric Patients and Surgical Settings 154 Feb 23, 2016: OPKO Health Announces Dosing of First Patient in Phase 2a Clinical Study of a Long-Acting Factor VIIa for the Treatment of Hemophilia 155 Feb 22, 2016: Bayer Receives EU Approval of Kovaltry for Treatment of Hemophilia A 156 Feb 03, 2016: Health Canada Approves New Treatment Option For Canadian Adults and Children Living with Hemophilia A 156 Appendix 158 Methodology 158 Coverage 158 Secondary Research 158 Primary Research 158 Expert Panel Validation 158 Contact Us 158 Disclaimer 159
List of Tables
Number of Products under Development for Hemophilia A, H1 2016 13 Number of Products under Development for Hemophilia A - Comparative Analysis, H1 2016 14 Number of Products under Development by Companies, H1 2016 15 Number of Products under Development by Companies, H1 2016 (Contd..1) 16 Number of Products under Investigation by Universities/Institutes, H1 2016 17 Comparative Analysis by Late Stage Development, H1 2016 18 Comparative Analysis by Clinical Stage Development, H1 2016 19 Comparative Analysis by Early Stage Development, H1 2016 20 Comparative Analysis by Unknown Stage Development, H1 2016 21 Products under Development by Companies, H1 2016 22 Products under Development by Companies, H1 2016 (Contd..1) 23 Products under Development by Companies, H1 2016 (Contd..2) 24 Products under Investigation by Universities/Institutes, H1 2016 25 Hemophilia A - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2016 26 Hemophilia A - Pipeline by Amarna Therapeutics B.V., H1 2016 27 Hemophilia A - Pipeline by Apitope International NV, H1 2016 28 Hemophilia A - Pipeline by Baxalta Incorporated, H1 2016 29 Hemophilia A - Pipeline by Bayer AG, H1 2016 30 Hemophilia A - Pipeline by Biogen, Inc., H1 2016 31 Hemophilia A - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 32 Hemophilia A - Pipeline by Catalyst Biosciences, Inc., H1 2016 33 Hemophilia A - Pipeline by CSL Limited, H1 2016 34 Hemophilia A - Pipeline by DBV Technologies S.A., H1 2016 35 Hemophilia A - Pipeline by Dimension Therapeutics, Inc., H1 2016 36 Hemophilia A - Pipeline by Emergent BioSolutions Inc., H1 2016 37 Hemophilia A - Pipeline by EpiVax, Inc., H1 2016 38 Hemophilia A - Pipeline by Expression Therapeutics LLC, H1 2016 39 Hemophilia A - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016 40 Hemophilia A - Pipeline by Green Cross Corporation, H1 2016 41 Hemophilia A - Pipeline by Idogen AB, H1 2016 42 Hemophilia A - Pipeline by Lentigen Technology, Inc., H1 2016 43 Hemophilia A - Pipeline by mAbxience S.A., H1 2016 44 Hemophilia A - Pipeline by Novo Nordisk A/S, H1 2016 45 Hemophilia A - Pipeline by Octapharma AG, H1 2016 46 Hemophilia A - Pipeline by OPKO Health, Inc., H1 2016 47 Hemophilia A - Pipeline by Pfizer Inc., H1 2016 48 Hemophilia A - Pipeline by Pharming Group N.V., H1 2016 49 Hemophilia A - Pipeline by rEVO Biologics, Inc., H1 2016 50 Hemophilia A - Pipeline by Sangamo BioSciences, Inc., H1 2016 51 Hemophilia A - Pipeline by SK Chemicals Co., Ltd., H1 2016 52 Hemophilia A - Pipeline by Spark Therapeutics, Inc., H1 2016 53 Hemophilia A - Pipeline by The International Biotechnology Center (IBC) Generium, H1 2016 54 Hemophilia A - Pipeline by UniQure N.V., H1 2016 55 Assessment by Monotherapy Products, H1 2016 56 Number of Products by Stage and Target, H1 2016 58 Number of Products by Stage and Mechanism of Action, H1 2016 60 Number of Products by Stage and Route of Administration, H1 2016 62 Number of Products by Stage and Molecule Type, H1 2016 64 Hemophilia A Therapeutics - Recent Pipeline Updates, H1 2016 118 Hemophilia A - Dormant Projects, H1 2016 147 Hemophilia A - Dormant Projects (Contd..1), H1 2016 148 Hemophilia A - Discontinued Products, H1 2016 149

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