BioMarin Pharmaceutical Inc. - Product Pipeline Review - 2015

Published: Apr, 2015 | Pages: 97 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

BioMarin Pharmaceutical Inc. - Product Pipeline Review - 2015

Summary 

Global Markets Direct's, 'BioMarin Pharmaceutical Inc. - Product Pipeline Review - 2015', provides an overview of the BioMarin Pharmaceutical Inc.'s pharmaceutical research and development focus.

This report provides comprehensive information on the current therapeutic developmental pipeline of BioMarin Pharmaceutical Inc.'s, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. It also reviews latest updates, and featured news and press releases, along with special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

- The report provides brief overview of BioMarin Pharmaceutical Inc. including business description, key information and facts, and its locations and subsidiaries
- The report reviews current pipeline of BioMarin Pharmaceutical Inc.'s human therapeutic division and enlists all their major and minor projects
- The report features product description and descriptive mechanism of action for key pipeline products along with the product's developmental history and major milestones 
- Special feature on out-licensed and partnered product portfolio
- The report summarizes all the dormant and discontinued pipeline projects
- Latest company statement 
- Latest news and deals relating to the BioMarin Pharmaceutical Inc.'s pipeline products

Reason To Buy 

- Evaluate BioMarin Pharmaceutical Inc.'s strategic position with total access to detailed information on its product pipeline
- Assess the growth potential of BioMarin Pharmaceutical Inc. in its therapy areas of focus
- Identify new drug targets and therapeutic classes in the BioMarin Pharmaceutical Inc.'s R&D portfolio and develop key strategic initiatives to reinforce pipeline in those areas
- Exploit in-licensing opportunities by identifying windows of opportunity to fill portfolio gaps
- Develop strategic initiatives by understanding the focus areas of BioMarin Pharmaceutical Inc. and exploit collaboration and partnership opportunities
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Plan mergers and acquisitions effectively by identifying the most promising pipeline of BioMarin Pharmaceutical Inc.
- 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
- Explore the dormant and discontinued projects of BioMarin Pharmaceutical Inc. and identify potential opportunities in those areas
- Avoid Intellectual Property Rights related issues

 Table of Contents

Table of Contents 2
List of Tables 6
List of Figures 6
BioMarin Pharmaceutical Inc. Snapshot 7
BioMarin Pharmaceutical Inc. Overview 7
Key Information 7
Key Facts 7
BioMarin Pharmaceutical Inc. - Research and Development Overview 8
Key Therapeutic Areas 8
BioMarin Pharmaceutical Inc. - Pipeline Review 11
Pipeline Products by Stage of Development 11
Pipeline Products - Monotherapy 12
BioMarin Pharmaceutical Inc. - Pipeline Products Glance 13
BioMarin Pharmaceutical Inc. - Late Stage Pipeline Products 13
Pre-Registration Products/Combination Treatment Modalities 13
Phase III Products/Combination Treatment Modalities 14
BioMarin Pharmaceutical Inc. - Clinical Stage Pipeline Products 15
Phase II Products/Combination Treatment Modalities 15
Phase I Products/Combination Treatment Modalities 16
Phase 0 Products/Combination Treatment Modalities 17
BioMarin Pharmaceutical Inc. - Early Stage Pipeline Products 18
Preclinical Products/Combination Treatment Modalities 18
Discovery Products/Combination Treatment Modalities 19
BioMarin Pharmaceutical Inc. - Unknown Stage Pipeline Products 20
Unknown Products/Combination Treatment Modalities 20
BioMarin Pharmaceutical Inc. - Drug Profiles 21
drisapersen 21
Product Description 21
Mechanism of Action 21
R&D Progress 21
elosulfase alfa 24
Product Description 24
Mechanism of Action 24
R&D Progress 24
sapropterin dihydrochloride 26
Product Description 26
Mechanism of Action 26
R&D Progress 26
amifampridine phosphate 28
Product Description 28
Mechanism of Action 28
R&D Progress 28
pegvaliase 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
reveglucosidase alfa 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
talazoparib 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
BMN-044 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
BMN-045 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
BMN-111 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
cerliponase alfa 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
BMN-053 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
RG-2833 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
Antisense RNAi Oligonucleotides to Activate Dysferlin for Dysferlinopathies 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
BMN-250 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
BMN-270 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
PRO-052 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
PRO-055 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
PRO-135 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
PRO-289 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
PROSPECT 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
RG-3250 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Small Molecule for Sanfilippo Syndrome 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Antisense RNAi Oligonucleotide for Rheumatoid Arthritis 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
RNAi Oligonucleotide for Bone Diseases 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
RNAi Oligonucleotide for Cancer 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
RNAi Oligonucleotide for Skin Diseases 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
BioMarin Pharmaceutical Inc. - Pipeline Analysis 60
BioMarin Pharmaceutical Inc. - Pipeline Products by Target 60
BioMarin Pharmaceutical Inc. - Pipeline Products by Route of Administration 62
BioMarin Pharmaceutical Inc. - Pipeline Products by Molecule Type 63
BioMarin Pharmaceutical Inc. - Pipeline Products by Mechanism of Action 64
BioMarin Pharmaceutical Inc. - Recent Pipeline Updates 66
BioMarin Pharmaceutical Inc. - Dormant Projects 90
BioMarin Pharmaceutical Inc. - Dormant Projects 90
BioMarin Pharmaceutical Inc. - Company Statement 91
BioMarin Pharmaceutical Inc. - Locations And Subsidiaries 93
Head Office 93
Other Locations & Subsidiaries 93
BioMarin Pharmaceutical Inc. - Key Manufacturing Facilities 95
Appendix 96
Methodology 96
Coverage 96
Secondary Research 96
Primary Research 96
Expert Panel Validation 96
Contact Us 96
Disclaimer 97

List of Tables

BioMarin Pharmaceutical Inc., Key Information 7
BioMarin Pharmaceutical Inc., Key Facts 7
BioMarin Pharmaceutical Inc. - Pipeline by Indication, 2015 9
BioMarin Pharmaceutical Inc. - Pipeline by Stage of Development, 2015 11
BioMarin Pharmaceutical Inc. - Monotherapy Products in Pipeline, 2015 12
BioMarin Pharmaceutical Inc. - Pre-Registration, 2015 13
BioMarin Pharmaceutical Inc. - Phase III, 2015 14
BioMarin Pharmaceutical Inc. - Phase II, 2015 15
BioMarin Pharmaceutical Inc. - Phase I, 2015 16
BioMarin Pharmaceutical Inc. - Phase 0, 2015 17
BioMarin Pharmaceutical Inc. - Preclinical, 2015 18
BioMarin Pharmaceutical Inc. - Discovery, 2015 19
BioMarin Pharmaceutical Inc. - Unknown, 2015 20
BioMarin Pharmaceutical Inc. - Pipeline by Target, 2015 61
BioMarin Pharmaceutical Inc. - Pipeline by Route of Administration, 2015 62
BioMarin Pharmaceutical Inc. - Pipeline by Molecule Type, 2015 63
BioMarin Pharmaceutical Inc. - Pipeline Products by Mechanism of Action, 2015 65
BioMarin Pharmaceutical Inc. - Recent Pipeline Updates, 2015 66
BioMarin Pharmaceutical Inc. - Dormant Developmental Projects,2015 90
BioMarin Pharmaceutical Inc., Other Locations 93
BioMarin Pharmaceutical Inc., Subsidiaries 94
BioMarin Pharmaceutical Inc., Key Manufacturing Facilities 95