Summary
Global Markets Direct’s, ‘Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Pipeline Review, H1 2016’, provides in depth analysis on Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) targeted pipeline therapeutics.
The report provides comprehensive information on the Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45), targeted therapeutics, complete with analysis by indications, stage of development, 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 Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) targeted therapeutics development 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 for Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45)
- The report reviews Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) targeted therapeutics under development 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 in Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) targeted therapeutics and enlists all their major and minor projects
- The report assesses Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) targeted therapeutics based on 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 and deals related to Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) targeted therapeutics
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 the targeted therapy areas and indications for Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45)
- Identify the use of drugs for target identification and drug repurposing
- 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 Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) development landscape
- 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
Companies Mentioned
Amicus Therapeutics, Inc.
Bioorganic Research and Services S.A.
greenovation Biotech GmbH
JCR Pharmaceuticals Co., Ltd.
Pharming Group N.V.
Protalix BioTherapeutics, Inc.
Shire Plc
The International Biotechnology Center (IBC) Generium
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Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) Overview 7
Therapeutics Development 8
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Products under Development by Stage of Development 8
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Products under Development by Therapy Area 9
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Products under Development by Indication 10
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Pipeline Products Glance 11
Late Stage Products 11
Early Stage Products 12
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Products under Development by Companies 13
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Products under Development by Universities/Institutes 15
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Therapeutics Assessment 17
Assessment by Monotherapy/Combination Products 17
Assessment by Mechanism of Action 18
Assessment by Route of Administration 20
Assessment by Molecule Type 22
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Companies Involved in Therapeutics Development 24
Amicus Therapeutics, Inc. 24
Bioorganic Research and Services S.A. 25
greenovation Biotech GmbH 26
JCR Pharmaceuticals Co., Ltd. 27
Pharming Group N.V. 28
Protalix BioTherapeutics, Inc. 29
Shire Plc 30
The International Biotechnology Center (IBC) Generium 31
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Drug Profiles 32
ambroxol - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
AT-3375 - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
BNT-001 - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
imiglucerase biosimilar - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
MOSS-GBA - Drug Profile 37
Product Description 37
Mechanism Of Action 37
R&D Progress 37
PRX-112 - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
Recombinant Beta-Cerebrosidase Replacement for Gaucher's Disease - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
Small Molecule to Activate Glucocerebrosidase for Parkinson's, Gaucher's and Neurodegenerative Diseases - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
Small Molecules to Activate GCase for Parkinson's Disease - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
Small Molecules to Activate Glucocerebrosidase for Gaucher and Parkinson Disease - Drug Profile 43
Product Description 43
Mechanism Of Action 43
R&D Progress 43
taliglucerase alfa - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
taliglucerase alfa - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
velaglucerase alfa - Drug Profile 51
Product Description 51
Mechanism Of Action 51
R&D Progress 51
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Dormant Projects 52
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Discontinued Products 54
Glucosylceramidase (Acid Beta-Glucosidase or Alglucerase or D-Glucosyl-N-Acylsphingosine Glucohydrolase or EC 3.2.1.45) - Featured News & Press Releases 55
Feb 12, 2015: Safety and Efficacy of ELELYSO (taliglucerase alfa) for Injection in Pediatric Patients with Type 1 Gaucher Disease in Long-term Outcome Study Presented at the WORLDSymposium 2015 55
Sep 02, 2014: Shire’s VPRIV400 units/vial launched in Japan for the improvement of symptoms of Gaucher disease 55
Aug 28, 2014: Pfizer and Protalix BioTherapeutics Announce FDA Approval of Pediatric Indication for ELELYSO (taliglucerase alfa) for Injection, for Intravenous Use for the Treatment of Type 1 Gaucher Disease 56
Jul 04, 2014: Shire’s VPRIV approved in Japan for the improvement of symptoms of Gaucher disease 57
Jul 01, 2014: Pfizer Receives Kosher Certification for Elelyso (taliglucerase alfa) for Injection, for the Treatment of Type 1 Gaucher Disease 58
Jun 27, 2014: Protalix Announces New Data on ELELYSO (taliglucerase alfa) Presented at the European Working Group on Gaucher Disease 2014 11th Meeting 58
Jun 23, 2014: Protalix BioTherapeutics Announces New Data on ELELYSO (taliglucerase alfa) and Oral GCD to be Presented at the European Working Group on Gaucher Disease 2014 11th Meeting 59
Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease 59
May 30, 2014: Protalix Announces ELELYSO (taliglucerase alfa) Approved in Canada for the Treatment of Gaucher Disease in Both Adult and Pediatric Patients 60
May 22, 2014: Protalix Announces ELELYSO Approved in Australia for the Treatment of Gaucher Disease in both Adult and Pediatric Patients 60
Mar 14, 2014: Shire Pharmaceuticals Initiates Voluntary Nationwide Recall Of One Batch, Packaged into Three Lots, of VPRIV (velaglucerase alfa for injection) 61
Feb 14, 2014: Shire Announces FDA Approval of Manufacturing Facility for VPRIV (Velaglucerase Alfa for Injection) Drug Substance 62
Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014 62
Feb 06, 2014: Protalix BioTherapeutics' Technology Transfer Agreement for UPLYSO (alfataliglicerase) With Brazil's Ministry of Health Approved by the Brazilian National Institute of Industrial Property 63
Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients 64
Appendix 65
Methodology 65
Coverage 65
Secondary Research 65
Primary Research 65
Expert Panel Validation 65
Contact Us 65
Disclaimer 66
List of Tables
Number of Products under Development for, H1 2016 8
Number of Products under Development by Therapy Area, H1 2016 9
Number of Products under Development by Indication, H1 2016 10
Comparative Analysis by Late Stage Development, H1 2016 11
Comparative Analysis by Early Stage Products, H1 2016 12
Number of Products under Development by Companies, H1 2016 13
Products under Development by Companies, H1 2016 14
Number of Products under Investigation by Universities/Institutes, H1 2016 15
Products under Investigation by Universities/Institutes, H1 2016 16
Assessment by Monotherapy/Combination Products, H1 2016 17
Number of Products by Stage and Mechanism of Action, H1 2016 19
Number of Products by Stage and Route of Administration, H1 2016 21
Number of Products by Stage and Molecule Type, H1 2016 23
Pipeline by Amicus Therapeutics, Inc., H1 2016 24
Pipeline by Bioorganic Research and Services S.A., H1 2016 25
Pipeline by greenovation Biotech GmbH, H1 2016 26
Pipeline by JCR Pharmaceuticals Co., Ltd., H1 2016 27
Pipeline by Pharming Group N.V., H1 2016 28
Pipeline by Protalix BioTherapeutics, Inc., H1 2016 29
Pipeline by Shire Plc, H1 2016 30
Pipeline by The International Biotechnology Center (IBC) Generium, H1 2016 31
Dormant Projects, H1 2016 52
Dormant Projects (Contd..1), H1 2016 53
Discontinued Products, H1 2016 54
List of Figures
Number of Products under Development for, H1 2016 8
Number of Products under Development by Therapy Area, H1 2016 9
Number of Products under Development by Top 10 Indication, H1 2016 10
Comparative Analysis by Early Stage Products, H1 2016 12
Assessment by Monotherapy/Combination Products, H1 2016 17
Number of Products by Mechanism of Actions, H1 2016 18
Number of Products by Stage and Mechanism of Actions, H1 2016 18
Number of Products by Routes of Administration, H1 2016 20
Number of Products by Stage and Routes of Administration, H1 2016 20
Number of Products by Molecule Types, H1 2016 22
Number of Products by Stage and Molecule Type, H1 2016 22
