BioMarin Pharmaceutical Inc.-Product Pipeline Review-2015

BioMarin Pharmaceutical Inc.-Product Pipeline Review-2015

  • Products Id :- GMDHC06998CDB
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  • Pages: 97
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Executive Summary

BioMarin Pharmaceutical Inc.-Product Pipeline Review-2015


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.


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

Reasons 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

Read More

Contact Us:
Ken Research
Ankur Gupta, Head Marketing & Communications


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


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

List of Figures

BioMarin Pharmaceutical Inc.-Pipeline by Top 10 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.-Pipeline by Top 10 Target, 2015 60

BioMarin Pharmaceutical Inc.-Pipeline by Top 10 Route of Administration, 2015 62

BioMarin Pharmaceutical Inc.-Pipeline by Top 10 Molecule Type, 2015 63

BioMarin Pharmaceutical Inc.-Pipeline Products by Top 10 Mechanism of Action, 2015 64

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Current R&D Portfolio of BioMarin Pharmaceutical Inc.; BioMarin Pharmaceutical Inc. - Key Therapeutics; BioMarin Pharmaceutical Inc. - Pipeline Overview and Promising Molecules; BioMarin Pharmaceutical Inc. - News; BioMarin Pharmaceutical Inc. - Latest Updates; BioMarin Pharmaceutical Inc. - Pipeline; BioMarin Pharmaceutical Inc. - Discontinued/Dormant Projects

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