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Hereditary Angioedema (HAE) (C1 Esterase Inhibitor (C1-INH) Deficiency) - Pipeline Review, H2 2016

Published By :

Global Markets Direct

Published Date : Dec 2016

Category :

Pharmaceutical

No. of Pages : 87 Pages

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 Directs 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 its 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 Behrings 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

List of Figures
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 Products, H2 2016 13
Assessment by Monotherapy Products, H2 2016 29
Number of Products by Targets, H2 2016 30
Number of Products by Stage and Targets, H2 2016 30
Number of Products by Mechanism of Actions, H2 2016 32
Number of Products by Stage and Mechanism of Actions, H2 2016 32
Number of Products by Routes of Administration, H2 2016 34
Number of Products by Stage and Routes of Administration, H2 2016 34
Number of Products by Molecule Types, H2 2016 36
Number of Products by Stage and Molecule Types, H2 2016 36

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