Gaucher Disease – Pipeline Review, H1 2017

Global Markets Direct’s, ‘Gaucher Disease – Pipeline Review, H1 2017’, provides an overview of the Gaucher Disease pipeline landscape.

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

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

The report assesses Gaucher Disease 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

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

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 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

Genzyme Corp

greenovation Biotech GmbH

JCR Pharmaceuticals Co Ltd

Lixte Biotechnology Holdings Inc

Neuraltus Pharmaceuticals Inc

Orphazyme ApS

Pharming Group NV

Protalix BioTherapeutics Inc

Shire Plc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Gaucher Disease - Overview

Gaucher Disease - Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Gaucher Disease - Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Gaucher Disease - Companies Involved in Therapeutics Development

Actelion Ltd

Genzyme Corp

greenovation Biotech GmbH

JCR Pharmaceuticals Co Ltd

Lixte Biotechnology Holdings Inc

Neuraltus Pharmaceuticals Inc

Orphazyme ApS

Pharming Group NV

Protalix BioTherapeutics Inc

Shire Plc

Gaucher Disease - Drug Profiles

ADN-LYS - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ambroxol - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

arimoclomol - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AVRRD-02 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy for Gaucher Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy for Gaucher Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ibiglustat - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

imiglucerase biosimilar - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LB-201 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LB-205 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LTI-291 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

miglustat - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

MOSS-GBA - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NCGC-607 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NP-003 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Pcgin - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PRX-112 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

RND-001 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

taliglucerase alfa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

velaglucerase alfa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gaucher Disease - Dormant Projects

Gaucher Disease - Discontinued Products

Gaucher Disease - Product Development Milestones

Featured News & Press Releases

Mar 22, 2017: AVROBIO Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease

Oct 25, 2016: Asthma research unexpectedly yields new treatment approach for inherited enzyme disease

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

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

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

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

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

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

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

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

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

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

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

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

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Gaucher Disease, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Gaucher Disease – Pipeline by Actelion Ltd, H1 2017

Gaucher Disease – Pipeline by Genzyme Corp, H1 2017

Gaucher Disease – Pipeline by greenovation Biotech GmbH, H1 2017

Gaucher Disease – Pipeline by JCR Pharmaceuticals Co Ltd, H1 2017

Gaucher Disease – Pipeline by Lixte Biotechnology Holdings Inc, H1 2017

Gaucher Disease – Pipeline by Neuraltus Pharmaceuticals Inc, H1 2017

Gaucher Disease – Pipeline by Orphazyme ApS, H1 2017

Gaucher Disease – Pipeline by Pharming Group NV, H1 2017

Gaucher Disease – Pipeline by Protalix BioTherapeutics Inc, H1 2017

Gaucher Disease – Pipeline by Shire Plc, H1 2017

Gaucher Disease – Dormant Projects, H1 2017

Gaucher Disease – Discontinued Products, H1 2017

List of Figures

List of Figures

Number of Products under Development for Gaucher Disease, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Number of Products by Targets, H1 2017

Number of Products by Stage and Targets, H1 2017

Number of Products by Top 10 Mechanism of Actions, H1 2017

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

Number of Products by Stage and Routes of Administration, H1 2017

Number of Products by Molecule Types, H1 2017

Number of Products by Stage and Molecule Types, H1 2017

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