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Published: Dec, 2016 | Pages:
87 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline Review, H2 2016 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline Review, H2 2016, provides an overview of the Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology) pipeline landscape. Hereditary angioedema (HAE) is a rare inherited disease that causes considerable swelling in various body tissues, such as the abdomen or face. Symptoms include severe abdominal pain and cramping, dehydration, diarrhea and shock, hoarse voice, difficulty swallowing and difficulty breathing. Treatment includes medications, such as epinephrine, antihistamines, and corticosteroids. Report Highlights Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology), 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 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology) pipeline guide also reviews of key players involved in therapeutic development for Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) 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 and Discovery stages are 1, 3, 2, 4, 9 and 1 respectively. Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology) 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 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology). - The pipeline guide reviews pipeline therapeutics for Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology) 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 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology) therapeutics and enlists all their major and minor projects. - The pipeline guide evaluates Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology) 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 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology) 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 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology). - 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 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) (Immunology) 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 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) Overview 8 Therapeutics Development 9 Pipeline Products for Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Overview 9 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Therapeutics under Development by Companies 10 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline Products Glance 11 Late Stage Products 11 Clinical Stage Products 12 Early Stage Products 13 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Products under Development by Companies 14 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Companies Involved in Therapeutics Development 16 ActiveSite Pharmaceuticals Inc 16 Adverum Biotechnologies Inc 17 Arrowhead Pharmaceuticals Inc 18 BioCryst Pharmaceuticals Inc 19 Cevec Pharmaceuticals GmbH 20 CSL Ltd 21 Global Blood Therapeutics Inc 22 iBio Inc 23 Ionis Pharmaceuticals Inc 24 Kalvista Pharmaceuticals Ltd 25 Pharming Group NV 26 ProMetic Life Sciences Inc 27 Shire Plc 28 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Therapeutics Assessment 29 Assessment by Monotherapy Products 29 Assessment by Target 30 Assessment by Mechanism of Action 32 Assessment by Route of Administration 34 Assessment by Molecule Type 36 Drug Profiles 38 ADVM-053 - Drug Profile 38 Product Description 38 Mechanism Of Action 38 R&D Progress 38 ARCF-12 - Drug Profile 39 Product Description 39 Mechanism Of Action 39 R&D Progress 39 BCX-7353 - Drug Profile 40 Product Description 40 Mechanism Of Action 40 R&D Progress 40 BEL-0215 - Drug Profile 42 Product Description 42 Mechanism Of Action 42 R&D Progress 42 C1 esterase inhibitor (human) - Drug Profile 43 Product Description 43 Mechanism Of Action 43 R&D Progress 43 C1 esterase inhibitor (human) - Drug Profile 48 Product Description 48 Mechanism Of Action 48 R&D Progress 48 C1 esterase inhibitor (human) - Drug Profile 50 Product Description 50 Mechanism Of Action 50 R&D Progress 50 C1 esterase inhibitor (recombinant) - Drug Profile 51 Product Description 51 Mechanism Of Action 51 R&D Progress 51 C1 esterase inhibitor (recombinant) - Drug Profile 58 Product Description 58 Mechanism Of Action 58 R&D Progress 58 CSL-312 - Drug Profile 59 Product Description 59 Mechanism Of Action 59 R&D Progress 59 GBT-18713 - Drug Profile 60 Product Description 60 Mechanism Of Action 60 R&D Progress 60 icatibant acetate - Drug Profile 61 Product Description 61 Mechanism Of Action 61 R&D Progress 61 IONIS-PKKRx - Drug Profile 64 Product Description 64 Mechanism Of Action 64 R&D Progress 64 KVD-818 - Drug Profile 65 Product Description 65 Mechanism Of Action 65 R&D Progress 65 lanadelumab - Drug Profile 66 Product Description 66 Mechanism Of Action 66 R&D Progress 66 Recombinant Protein to Inhibit C1-esterase for Hereditary Angioedema - Drug Profile 70 Product Description 70 Mechanism Of Action 70 R&D Progress 70 SHP-623 - Drug Profile 71 Product Description 71 Mechanism Of Action 71 R&D Progress 71 Small Molecules to Inhibit Plasma Kallikrein for Hereditary Angioedema - Drug Profile 72 Product Description 72 Mechanism Of Action 72 R&D Progress 72 Small Molecules to Inhibit Plasma Kallikrein for Hereditary Angioedema - Drug Profile 73 Product Description 73 Mechanism Of Action 73 R&D Progress 73 Small Molecules to Inhibit Plasma Kallikrein for Hereditary Angioedema - Drug Profile 74 Product Description 74 Mechanism Of Action 74 R&D Progress 74 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Dormant Projects 75 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Discontinued Products 76 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Product Development Milestones 77 Featured News & Press Releases 77 Dec 01, 2016: CSL Commits to New Phase I Clinical Trials of CSL312 in Australia 77 Nov 14, 2016: Pharming Announces the Presentation of the Results of the RUCONEST Phase II study for Prophylaxis of Hereditary Angioedema Attacks 78 Nov 10, 2016: Pharming Group Receives CHMP Positive Opinion recommending change to the terms of the marketing authorisation for Ruconest 78 Aug 30, 2016: FDA Accepts CSL Behring's BLA for First Subcutaneous Prophylactic Therapy to Prevent Hereditary Angioedema Attacks 78 Aug 11, 2016: BioCryst Announces Initiation of the APeX-1 Clinical Trial of BCX7353 for Hereditary Angioedema 79 Jul 18, 2016: U.S. FDA Approves CSL Behrings Berinert as the First and Only Pediatric Treatment for Hereditary Angioedema (HAE) 79 Jul 18, 2016: Pharming Announces Positive Results from Randomized Controlled Trial with RUCONEST for HAE Prophylaxis 79 May 19, 2016: KalVista Pharmaceuticals to Support Global Conference Focused on Hereditary Angioedema 81 Apr 07, 2016: Pharming Announces Adoption By The European Commission Of The CHMP Recommendation To Change The Terms Of The Marketing Authorisation For Ruconest 81 Mar 23, 2016: CINRYZE (C1 inhibitor [human]) now available in Canada 82 Feb 26, 2016: Pharming Announces Positive CHMP Opinion To Change The Terms Of The Marketing Authorisation For Ruconest 82 Feb 09, 2016: Pharming And Cytobioteck Announce Extension Of Distribution Agreement For Ruconest 83 Jan 06, 2016: Pharming Announces Completion Of Patient Enrolment In Clinical Study Of Ruconest For Prophylaxis Of Hereditary Angioedema 84 Dec 21, 2015: Pharming And Hyupjin Announce Receipt Of The South Korean Marketing Authorisation For Ruconest 84 Dec 07, 2015: European Medicines Agency Reaffirms Important Health Benefits and Safety of RUCONEST for Patients With Hereditary Angio-edema 85 Appendix 86 Methodology 86 Coverage 86 Secondary Research 86 Primary Research 86 Expert Panel Validation 86 Contact Us 86 Disclaimer 87
List of Tables
Number of Products under Development for Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency), H2 2016 9 Number of Products under Development by Companies, H2 2016 10 Comparative Analysis by Late Stage Development, H2 2016 11 Comparative Analysis by Clinical Stage Development, H2 2016 12 Comparative Analysis by Early Stage Development, H2 2016 13 Products under Development by Companies, H2 2016 14 Products under Development by Companies, H2 2016 (Contd..1) 15 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by ActiveSite Pharmaceuticals Inc, H2 2016 16 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by Adverum Biotechnologies Inc, H2 2016 17 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by Arrowhead Pharmaceuticals Inc, H2 2016 18 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by BioCryst Pharmaceuticals Inc, H2 2016 19 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by Cevec Pharmaceuticals GmbH, H2 2016 20 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by CSL Ltd, H2 2016 21 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by Global Blood Therapeutics Inc, H2 2016 22 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by iBio Inc, H2 2016 23 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by Ionis Pharmaceuticals Inc, H2 2016 24 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by Kalvista Pharmaceuticals Ltd, H2 2016 25 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by Pharming Group NV, H2 2016 26 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by ProMetic Life Sciences Inc, H2 2016 27 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Pipeline by Shire Plc, H2 2016 28 Assessment by Monotherapy Products, H2 2016 29 Number of Products by Stage and Target, H2 2016 31 Number of Products by Stage and Mechanism of Action, H2 2016 33 Number of Products by Stage and Route of Administration, H2 2016 35 Number of Products by Stage and Molecule Type, H2 2016 37 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Dormant Projects, H2 2016 75 Hereditary Angioedema (HAE) (C1 Esterase Inhibitor [C1-INH] Deficiency) - Discontinued Products, H2 2016 76
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