BioMarin Pharmaceutical Inc. - Product Pipeline Review - 2016

Published: Jun, 2016 | Pages: 60 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

BioMarin Pharmaceutical Inc. - Product Pipeline Review - 2016

Summary

Global Markets Direct's, 'BioMarin Pharmaceutical Inc. - Product Pipeline Review - 2016', provides an overview of the BioMarin Pharmaceutical Inc.'s pharmaceutical research and development focus. 

The report provides comprehensive information on the therapeutics under development by BioMarin Pharmaceutical Inc., 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 the 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 pipeline therapeutic landscape of BioMarin Pharmaceutical Inc.
- The report provides overview of BioMarin Pharmaceutical Inc. including its business description, key facts, and locations and subsidiaries
- 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 covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages 
- The report assesses BioMarin Pharmaceutical Inc.'s pipeline therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type 
- The report features BioMarin Pharmaceutical Inc.'s out-licensed and partnered product portfolio and summarizes its dormant and discontinued projects

Reasons To Buy

- Evaluate BioMarin Pharmaceutical Inc.'s strategic position with total access to detailed information on its product pipeline
- 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 BioMarin Pharmaceutical Inc.
- Identify potential new clients or partners in the target demographic
- Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding BioMarin Pharmaceutical Inc.'s pipeline depth and focus of pipeline 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

 Table of Contents

Table of Contents 2
List of Tables 5
List of Figures 5
BioMarin Pharmaceutical Inc. Snapshot 6
BioMarin Pharmaceutical Inc. Overview 6
Key Information 6
Key Facts 6
BioMarin Pharmaceutical Inc. - Research and Development Overview 7
Key Therapeutic Areas 7
BioMarin Pharmaceutical Inc. - Pipeline Review 10
Pipeline Products by Stage of Development 10
Pipeline Products - Monotherapy 11
Pipeline Products - Out-Licensed Products 12
Out-Licensed Products/Combination Treatment Modalities 13
BioMarin Pharmaceutical Inc. - Pipeline Products Glance 14
BioMarin Pharmaceutical Inc. - Late Stage Pipeline Products 14
Filing rejected/Withdrawn Products/Combination Treatment Modalities 14
Phase III Products/Combination Treatment Modalities 15
BioMarin Pharmaceutical Inc. - Clinical Stage Pipeline Products 16
Phase II Products/Combination Treatment Modalities 16
Phase I Products/Combination Treatment Modalities 17
BioMarin Pharmaceutical Inc. - Early Stage Pipeline Products 18
Preclinical Products/Combination Treatment Modalities 18
BioMarin Pharmaceutical Inc. - Drug Profiles 19
amifampridine phosphate 19
Product Description 19
Mechanism of Action 19
R&D Progress 19
pegvaliase 21
Product Description 21
Mechanism of Action 21
R&D Progress 21
reveglucosidase alfa 23
Product Description 23
Mechanism of Action 23
R&D Progress 23
cerliponase alfa 24
Product Description 24
Mechanism of Action 24
R&D Progress 24
vosoritide 25
Product Description 25
Mechanism of Action 25
R&D Progress 25
BMN-250 26
Product Description 26
Mechanism of Action 26
R&D Progress 26
BMN-270 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
Antisense RNAi Oligonucleotides to Activate Dysferlin for Dysferlinopathies 28
Product Description 28
Mechanism of Action 28
R&D Progress 28
PRO-052 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
PRO-055 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
PRO-135 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
PRO-289 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
RG-3250 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
Small Molecule for Sanfilippo Syndrome 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
BioMarin Pharmaceutical Inc. - Pipeline Analysis 37
BioMarin Pharmaceutical Inc. - Pipeline Products by Target 37
BioMarin Pharmaceutical Inc. - Pipeline Products by Route of Administration 38
BioMarin Pharmaceutical Inc. - Pipeline Products by Molecule Type 39
BioMarin Pharmaceutical Inc. - Pipeline Products by Mechanism of Action 40
BioMarin Pharmaceutical Inc. - Recent Pipeline Updates 41
BioMarin Pharmaceutical Inc. - Dormant Projects 54
BioMarin Pharmaceutical Inc. - Discontinued Pipeline Products 55
Discontinued Pipeline Product Profiles 55
BMN-044 55
BMN-045 55
BMN-053 55
drisapersen 55
BioMarin Pharmaceutical Inc. - Locations And Subsidiaries 56
Head Office 56
Other Locations & Subsidiaries 56
BioMarin Pharmaceutical Inc. - Key Manufacturing Facilities 58
Appendix 59
Methodology 59
Coverage 59
Secondary Research 59
Primary Research 59
Expert Panel Validation 59
Contact Us 59
Disclaimer 60

List of Tables

BioMarin Pharmaceutical Inc., Key Information 6
BioMarin Pharmaceutical Inc., Key Facts 6
BioMarin Pharmaceutical Inc. - Pipeline by Indication, 2016 8
BioMarin Pharmaceutical Inc. - Pipeline by Stage of Development, 2016 10
BioMarin Pharmaceutical Inc. - Monotherapy Products in Pipeline, 2016 11
BioMarin Pharmaceutical Inc. - Out-Licensed Products in Pipeline, 2016 12
BioMarin Pharmaceutical Inc. - Out-Licensed Products/ Combination Treatment Modalities, 2016 13
BioMarin Pharmaceutical Inc. - Filing rejected/Withdrawn, 2016 14
BioMarin Pharmaceutical Inc. - Phase III, 2016 15
BioMarin Pharmaceutical Inc. - Phase II, 2016 16
BioMarin Pharmaceutical Inc. - Phase I, 2016 17
BioMarin Pharmaceutical Inc. - Preclinical, 2016 18
BioMarin Pharmaceutical Inc. - Pipeline by Target, 2016 37
BioMarin Pharmaceutical Inc. - Pipeline by Route of Administration, 2016 38
BioMarin Pharmaceutical Inc. - Pipeline by Molecule Type, 2016 39
BioMarin Pharmaceutical Inc. - Pipeline Products by Mechanism of Action, 2016 40
BioMarin Pharmaceutical Inc. - Recent Pipeline Updates, 2016 41
BioMarin Pharmaceutical Inc. - Dormant Developmental Projects,2016 54
BioMarin Pharmaceutical Inc. - Discontinued Pipeline Products, 2016 55
BioMarin Pharmaceutical Inc., Other Locations 56
BioMarin Pharmaceutical Inc., Subsidiaries 56
BioMarin Pharmaceutical Inc., Key Manufacturing Facilities 58