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
List of Figures
BioMarin Pharmaceutical Inc. - Pipeline by Top 10 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. - Pipeline by Top 10 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 Top 10 Mechanism of Action, 2016 40
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