Gaucher Disease Type III – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Gaucher Disease Type III – Pipeline Review, H2 2016’, provides an overview of the Gaucher Disease Type III pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Gaucher Disease Type III, 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 Gaucher Disease Type III 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.

Scope

The report provides a snapshot of the global therapeutic landscape of Gaucher Disease Type III

The report reviews pipeline therapeutics for Gaucher Disease Type III 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 Gaucher Disease Type III therapeutics and enlists all their major and minor projects

The report assesses Gaucher Disease Type III 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 Gaucher Disease Type III

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 Gaucher Disease Type III

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 Gaucher Disease Type III 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

Companies mentioned

Actelion Ltd

Belrose Pharma Inc

Erad Therapeutics Inc

Genzyme Corp

Shire Plc

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 3

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Gaucher Disease Type III Overview 6

Therapeutics Development 7

Pipeline Products for Gaucher Disease Type III - Overview 7

Gaucher Disease Type III - Therapeutics under Development by Companies 8

Gaucher Disease Type III - Pipeline Products Glance 9

Clinical Stage Products 9

Early Stage Products 10

Gaucher Disease Type III - Products under Development by Companies 11

Gaucher Disease Type III - Companies Involved in Therapeutics Development 12

Actelion Ltd 12

Belrose Pharma Inc 13

Erad Therapeutics Inc 14

Genzyme Corp 15

Shire Plc 16

Gaucher Disease Type III - Therapeutics Assessment 17

Assessment by Monotherapy Products 17

Assessment by Target 18

Assessment by Mechanism of Action 20

Assessment by Route of Administration 22

Assessment by Molecule Type 24

Drug Profiles 26

ambroxol - Drug Profile 26

Product Description 26

Mechanism Of Action 26

R&D Progress 26

ibiglustat - Drug Profile 28

Product Description 28

Mechanism Of Action 28

R&D Progress 28

miglustat - Drug Profile 30

Product Description 30

Mechanism Of Action 30

R&D Progress 30

Recombinant Protein for Type I and Type III Gaucher's Disease - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

velaglucerase alfa - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

Gaucher Disease Type III - Dormant Projects 34

Appendix 35

Methodology 35

Coverage 35

Secondary Research 35

Primary Research 35

Expert Panel Validation 35

Contact Us 35

Disclaimer 36

List of Tables

List of Tables

Number of Products under Development for Gaucher Disease Type III, H2 2016 7

Number of Products under Development by Companies, H2 2016 8

Comparative Analysis by Clinical Stage Development, H2 2016 9

Comparative Analysis by Early Stage Development, H2 2016 10

Products under Development by Companies, H2 2016 11

Gaucher Disease Type III – Pipeline by Actelion Ltd, H2 2016 12

Gaucher Disease Type III – Pipeline by Belrose Pharma Inc, H2 2016 13

Gaucher Disease Type III – Pipeline by Erad Therapeutics Inc, H2 2016 14

Gaucher Disease Type III – Pipeline by Genzyme Corp, H2 2016 15

Gaucher Disease Type III – Pipeline by Shire Plc, H2 2016 16

Assessment by Monotherapy Products, H2 2016 17

Number of Products by Stage and Target, H2 2016 19

Number of Products by Stage and Mechanism of Action, H2 2016 21

Number of Products by Stage and Route of Administration, H2 2016 23

Number of Products by Stage and Molecule Type, H2 2016 25

Gaucher Disease Type III – Dormant Projects, H2 2016 34

List of Figures

List of Figures

Number of Products under Development for Gaucher Disease Type III, H2 2016 7

Number of Products under Development by Companies, H2 2016 8

Comparative Analysis by Clinical Stage Development, H2 2016 9

Assessment by Monotherapy Products, H2 2016 17

Number of Products by Targets, H2 2016 18

Number of Products by Stage and Targets, H2 2016 18

Number of Products by Mechanism of Actions, H2 2016 20

Number of Products by Stage and Mechanism of Actions, H2 2016 20

Number of Products by Routes of Administration, H2 2016 22

Number of Products by Stage and Routes of Administration, H2 2016 22

Number of Products by Molecule Types, H2 2016 24

Number of Products by Stage and Molecule Types, H2 2016 24

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