Marktanalyse - Gaucher Disease - Pipeline Review, H2 2016

Global Markets Direct
12.2016
77 Seiten

 
Typ:
Marktanalyse
Verfüg­barkeit:
verfügbar
Regionen/­Länder:
  • Europa
  • Asien / Pazifik
  • Mittlerer Osten / Afrika
  • Nordamerika / USA
  • Australien
  • Mittel- / Südamerika
Sprache:
Englisch

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Gaucher Disease - Pipeline Review, H2 2016


Summary


Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Gaucher Disease — Pipeline Review, H2 2016, provides an overview of the Gaucher Disease (Genetic Disorders) pipeline landscape.

Gaucher disease is a rare genetic disorder occurs due to lacks an enzyme called glucocerebrosidase. Symptoms include cognitive impairment, enlarged spleen, severe swelling, lung disease, fatigue and seizures. Treatment includes enzyme replacement therapy.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Gaucher Disease — Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Gaucher Disease (Genetic 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 Gaucher Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Gaucher Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I, Preclinical and Discovery stages are 4, 2, 9 and 1 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 1 and 5 molecules, respectively.

Gaucher Disease (Genetic 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 Gaucher Disease (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Gaucher Disease (Genetic 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 Gaucher Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Gaucher Disease (Genetic 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 Gaucher Disease (Genetic 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 Gaucher Disease (Genetic 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 Gaucher Disease (Genetic 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

Gaucher Disease Overview 8

Therapeutics Development 9

Pipeline Products for Gaucher Disease - Overview 9

Pipeline Products for Gaucher Disease - Comparative Analysis 10

Gaucher Disease - Therapeutics under Development by Companies 11

Gaucher Disease - Therapeutics under Investigation by Universities/Institutes 12

Gaucher Disease - Pipeline Products Glance 13

Clinical Stage Products 13

Early Stage Products 14

Gaucher Disease - Products under Development by Companies 15

Gaucher Disease - Products under Investigation by Universities/Institutes 16

Gaucher Disease - Companies Involved in Therapeutics Development 17

Actelion Ltd 17

Genzyme Corp 18

greenovation Biotech GmbH 19

JCR Pharmaceuticals Co Ltd 20

Lixte Biotechnology Holdings Inc 21

Lysosomal Therapeutics Inc 22

Neuraltus Pharmaceuticals Inc 23

Pharming Group NV 24

Protalix BioTherapeutics Inc 25

Sangamo BioSciences Inc 26

Shire Plc 27

The International Biotechnology Center (IBC) Generium 28

Gaucher Disease - 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

ADN-LYS - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

ambroxol - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

Gene Therapy for Gaucher Disease - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

ibiglustat - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

imiglucerase biosimilar - Drug Profile 43

Product Description 43

Mechanism Of Action 43

R&D Progress 43

LB-201 - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

LB-205 - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

miglustat - Drug Profile 46

Product Description 46

Mechanism Of Action 46

R&D Progress 46

MOSS-GBA - Drug Profile 48

Product Description 48

Mechanism Of Action 48

R&D Progress 48

NCGC-607 - Drug Profile 49

Product Description 49

Mechanism Of Action 49

R&D Progress 49

NP-003 - Drug Profile 50

Product Description 50

Mechanism Of Action 50

R&D Progress 50

PRX-112 - Drug Profile 51

Product Description 51

Mechanism Of Action 51

R&D Progress 51

Recombinant Beta Glucosidase 2 Replacement for Gaucher's Disease and Male Infertility - Drug Profile 53

Product Description 53

Mechanism Of Action 53

R&D Progress 53

Recombinant Beta-Cerebrosidase Replacement for Gaucher's Disease - Drug Profile 54

Product Description 54

Mechanism Of Action 54

R&D Progress 54

Recombinant Protein for Type I and Type III Gaucher's Disease - Drug Profile 55

Product Description 55

Mechanism Of Action 55

R&D Progress 55

SBLSD-3 - Drug Profile 56

Product Description 56

Mechanism Of Action 56

R&D Progress 56

Small Molecule to Activate Beta Galactosidase for Gaucher Disease and GM1 Gangliosidosis - Drug Profile 57

Product Description 57

Mechanism Of Action 57

R&D Progress 57

Small Molecule to Activate Glucocerebrosidase for Parkinson's, Gaucher's and Neurodegenerative Diseases - Drug Profile 58

Product Description 58

Mechanism Of Action 58

R&D Progress 58

Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease - Drug Profile 59

Product Description 59

Mechanism Of Action 59

R&D Progress 59

Small Molecules to Inhibit Glucosidase for Diabetes and Gaucher's Disease - Drug Profile 60

Product Description 60

Mechanism Of Action 60

R&D Progress 60

taliglucerase alfa - Drug Profile 61

Product Description 61

Mechanism Of Action 61

R&D Progress 61

velaglucerase alfa - Drug Profile 62

Product Description 62

Mechanism Of Action 62

R&D Progress 62

Gaucher Disease - Dormant Projects 63

Gaucher Disease - Discontinued Products 65

Gaucher Disease - Product Development Milestones 66

Featured News & Press Releases 66

Sep 02, 2014: Shire’s VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease 66

Jul 04, 2014: Shire’s VPRIV approved in Japan for the improvement of symptoms of Gaucher disease 66

Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease 67

Mar 14, 2014: Shire Pharmaceuticals Initiates Voluntary Nationwide Recall Of One Batch, Packaged into Three Lots, of VPRIV (velaglucerase alfa for injection) 67

Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance 68

Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014 69

Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients 70

Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112 70

Mar 07, 2013: Protalix BioTherapeutics Receives Approval From Israeli Ministry Of Health To Initiate Phase I Clinical Trial Of PRX-112 For Treatment Of Gaucher Disease 71

Jun 28, 2012: Shire’s Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease 72

Jun 28, 2012: Shire’s Vpriv Shows Significant Improvement In Gaucher-Related Bone Disease 72

Feb 22, 2012: Shire Announces European Approval Of Manufacturing Facility For VPRIV 73

Dec 19, 2011: Lixte's LB-205 Shows Therapeutic Potential For Gaucher Disease 74

Nov 22, 2011: Shire Announces Regulatory Filings In US And EU For New Manufacturing Facility 74

Mar 21, 2011: Shire Presents Data For VPRIV In Gaucher Disease At 2011 ACMG Annual Meeting 75

Appendix 76

Methodology 76

Coverage 76

Secondary Research 76

Primary Research 76

Expert Panel Validation 76

Contact Us 76

Disclaimer 77





List of Tables

Number of Products under Development for Gaucher Disease, H2 2016 9

Number of Products under Development for Gaucher Disease - Comparative Analysis, H2 2016 10

Number of Products under Development by Companies, H2 2016 11

Number of Products under Investigation by Universities/Institutes, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 13

Comparative Analysis by Early Stage Development, H2 2016 14

Products under Development by Companies, H2 2016 15

Products under Investigation by Universities/Institutes, H2 2016 16

Gaucher Disease - Pipeline by Actelion Ltd, H2 2016 17

Gaucher Disease - Pipeline by Genzyme Corp, H2 2016 18

Gaucher Disease - Pipeline by greenovation Biotech GmbH, H2 2016 19

Gaucher Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H2 2016 20

Gaucher Disease - Pipeline by Lixte Biotechnology Holdings Inc, H2 2016 21

Gaucher Disease - Pipeline by Lysosomal Therapeutics Inc, H2 2016 22

Gaucher Disease - Pipeline by Neuraltus Pharmaceuticals Inc, H2 2016 23

Gaucher Disease - Pipeline by Pharming Group NV, H2 2016 24

Gaucher Disease - Pipeline by Protalix BioTherapeutics Inc, H2 2016 25

Gaucher Disease - Pipeline by Sangamo BioSciences Inc, H2 2016 26

Gaucher Disease - Pipeline by Shire Plc, H2 2016 27

Gaucher Disease - Pipeline by The International Biotechnology Center (IBC) Generium, 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

Gaucher Disease - Dormant Projects, H2 2016 63

Gaucher Disease - Dormant Projects (Contd..1), H2 2016 64

Gaucher Disease - Discontinued Products, H2 2016 65





List of Figures

Number of Products under Development for Gaucher Disease, H2 2016 9

Number of Products under Development for Gaucher Disease - Comparative Analysis, H2 2016 10

Number of Products under Development by Companies, H2 2016 11

Number of Products under Investigation by Universities/Institutes, H2 2016 12

Comparative Analysis by Clinical Stage Development, H2 2016 13

Comparative Analysis by Early Stage Products, H2 2016 14

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 Top 10 Mechanism of Actions, H2 2016 32

Number of Products by Stage and Top 10 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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