Table of Contents
Table of Contents 3
List of Tables 6
List of Figures 6
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 7
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare Deals By Type, 2012 to YTD 2018 8
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 9
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 10
BioMarin Pharmaceutical Inc, Medical Devices Deals, 2012 to YTD 2018 12
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals Summary, 2012 to YTD 2018 13
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deal Details 15
Venture Financing 15
ProSensa Raises US$30 Million In Series C Financing Round 15
Partnerships 17
Zacharon Pharma Enters Into Research Agreement With Acorda Therapeutics 17
Licensing Agreements 18
Avrobio Enters into Licensing Agreement with BioMarin Pharma 18
Sarepta Therapeutics Enters into Licensing Agreement with BioMarin Pharma 19
BioMarin Pharma Enters into Sub-license Agreement with Chugai Pharma 21
BioMarin Enters Into Licensing Agreement With Repligen For Histone Deacetylase Inhibitor Portfolio 22
Shire Human Genetic Enters into Licensing Agreement with Biomarin Pharma 23
Catalyst Pharma Amends Licensing Agreement with BioMarin Pharmaceutical 24
BioMarin Pharma Enters Into Licensing Agreement With University College London And St. Jude Children’s Research Hospital For Hemophilia A Gene Therapy Program 26
Equity Offering 28
BioMarin Pharma Raises USD720 Million in Public Offering of Shares 28
BioMarin Pharma Raises USD911.5 Million in Public Offering of Shares 29
BioMarin Pharma Completes Public Offering Of Shares For US$119.3 Million 30
BioMarin Completes Public Offering Of Common Stock For US$236 Million 31
Debt Offering 32
BioMarin Pharma Completes Underwriters Exercise of Over-Allotment Option of 0.599% Notes Due 2024 for USD45 Million 32
BioMarin Pharma Completes Public Offering Of Notes Due 2020 For US$375 Million 33
BioMarin Pharma Completes Public Offering Of Notes Due 2018 For US$375 Million 35
Asset Transactions 37
Medivation Acquires Rights to Talazoparib from BioMarin Pharma 37
Acquisition 38
Sanofi May Acquire BioMarin Pharma 38
Roche May Acquire BioMarin Pharma 39
BioMarin Pharmaceutical (BioMarin Pharma) Acquires 100% stake in Prosensa for USD751.5 Million 40
Roche Rumored to Acquire BioMarin Pharma for USD13.5 Billion 42
BioMarin Pharma Acquires Zacharon Pharma For USD9.7 Million 43
GlaxoSmithKline Plans To Acquire BioMarin Pharma 44
BioMarin Pharmaceutical Inc – Key Competitors 45
BioMarin Pharmaceutical Inc – Key Employees 46
BioMarin Pharmaceutical Inc – Locations And Subsidiaries 47
Head Office 47
Other Locations & Subsidiaries 47
Joint Venture 48
Recent Developments 49
Financial Announcements 49
Oct 25, 2018: BioMarin Pharmaceutical reports third quarter 2018 results 49
Aug 02, 2018: BioMarin announces second quarter 2018 results 52
Apr 25, 2018: BioMarin Announces First Quarter 2018 Financial Results 55
Feb 22, 2018: BioMarin Reports Fourth Quarter 2017 Financial Results 58
Oct 26, 2017: BioMarin Announces Third Quarter 2017 Financial Results 61
Aug 02, 2017: BioMarin Announces Second Quarter 2017 Financial Results 65
May 04, 2017: BioMarin Announces First Quarter 2017 Financial Results 67
Feb 23, 2017: BioMarin Announces Fourth Quarter and Full Year 2016 Financial Results 69
Jan 10, 2017: BioMarin Transfers Duchenne Natural History Database to CureDuchenne To Improve Knowledge of Duchenne Patients and Improve Clinical Outcomes 71
Corporate Communications 72
Oct 03, 2017: BioMarin Appoints Robert J. Hombach, Former Baxalta CFO and COO, to Board of Directors 72
Product News 73
Dec 14, 2017: Gene therapy success could lead to cure for haemophilia 73
12/11/2017: BioMarin Highlights New Results for Gene Therapy Valoctocogene Roxaparvovec at the 2017 American Society of Hemophilia (ASH) Meeting 74
12/06/2017: BioMarin Announces Presentations at 59th American Society of Hematology Annual Meeting & Exposition 76
09/06/2017: BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome at 13th International Congress of Inborn Errors of Metabolism 2017 77
09/05/2017: BioMarin To Present Data On ICV BMN 250 At 13th International Congress of Inborn Errors of Metabolism 2017 79
07/11/2017: BioMarins Investigational Gene Therapy for Hemophilia A at 6e13 vg/kg Dose Maintains Average Factor VIII Levels within Normal Range for over One Year 81
06/16/2017: BioMarin Announces Acceptance of Late Breaking Abstract at the International Society on Thrombosis and Haemostasis 2017 Congress 83
05/22/2018: BioMarin Provides 2 Years of Clinical Data in 6e13 vg/kg Dose from Ongoing Phase 1/2 Study in Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at World Federation of Hemophilia 2018 World Congress 84
05/17/2018: BioMarin Announces 1 Platform and 3 Poster Presentations at World Federation of Hemophilia 2018 World Congress 86
05/15/2018: BioMarin Announces First Patient Dosed in Phase 1/2 Study Evaluating Valoctocogene Roxaparvovec Gene Therapy in Severe Hemophilia A Patients with Pre-existing AAV5 Antibodies 88
04/27/2017: FDA approves first treatment for a form of Batten disease 89
04/21/2017: New medicine for rare neurodegenerative disorder in children 91
04/21/2017: BioMarin Receives Positive CHMP Opinion in Europe for Brineura (cerliponase alfa) for First Treatment of CLN2 Disease, a Form of Batten Disease and Ultra-Rare and Fatal Brain Disorder in Children 92
03/21/2018: BioMarin’s Gene Therapy Manufacturing Facility Recognized with Industry Award 94
02/07/2018: BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at WORLDSymposium 2018 95
01/08/2017: BioMarin to Provide Update to Proof-of-Concept Data for BMN 270 Gene Therapy in Hemophilia A at 35th Annual J.P. Morgan Healthcare Conference 97
Product Approvals 99
Feb 01, 2017: BioMarin Receives Access to Priority Medicines Regulatory Support from EMA for BMN 270 Gene Therapy in Hemophilia A 99
Clinical Trials 100
Jan 23, 2018: Local researcher leads breakthrough for haemophilia patients 100
Dec 19, 2017: BioMarin Doses First Patient in Global GENEr8-1 Phase 3 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A 101
Dec 09, 2017: BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at 59th American Society of Hematology Annual Meeting Concurrent with NEJM Publication 102
Oct 26, 2017: FDA Grants Breakthrough Therapy Designation for BioMarin’s Valoctocogene Roxaparvovec (formerly BMN 270), an Investigational Gene Therapy for Hemophilia A 104
Aug 02, 2017: BioMarin Announces Plans to Progress Both the 6e13vg/kg and 4e13 vg/kg Doses of BMN 270, its Investigational Gene Therapy for Hemophilia A, into Phase 3 Studies 105
Appendix 107
Methodology 107
About GlobalData 107
Contact Us 107
Disclaimer 107
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Key Facts, 2017 2
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 7
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare Deals By Type, 2012 to YTD 2018 8
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 9
BioMarin Pharmaceutical Inc, Deals By Therapy Area, 2012 to YTD 2018 10
BioMarin Pharmaceutical Inc, Medical Devices Deals, 2012 to YTD 2018 12
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals Summary, 2012 to YTD 2018 13
ProSensa Raises US$30 Million In Series C Financing Round 15
Zacharon Pharma Enters Into Research Agreement With Acorda Therapeutics 17
Avrobio Enters into Licensing Agreement with BioMarin Pharma 18
Sarepta Therapeutics Enters into Licensing Agreement with BioMarin Pharma 19
BioMarin Pharma Enters into Sub-license Agreement with Chugai Pharma 21
BioMarin Enters Into Licensing Agreement With Repligen For Histone Deacetylase Inhibitor Portfolio 22
Shire Human Genetic Enters into Licensing Agreement with Biomarin Pharma 23
Catalyst Pharma Amends Licensing Agreement with BioMarin Pharmaceutical 24
BioMarin Pharma Enters Into Licensing Agreement With University College London And St. Jude Children's Research Hospital For Hemophilia A Gene Therapy Program 26
BioMarin Pharma Raises USD720 Million in Public Offering of Shares 28
BioMarin Pharma Raises USD911.5 Million in Public Offering of Shares 29
BioMarin Pharma Completes Public Offering Of Shares For US$119.3 Million 30
BioMarin Completes Public Offering Of Common Stock For US$236 Million 31
BioMarin Pharma Completes Underwriters Exercise of Over-Allotment Option of 0.599% Notes Due 2024 for USD45 Million 32
BioMarin Pharma Completes Public Offering Of Notes Due 2020 For US$375 Million 33
BioMarin Pharma Completes Public Offering Of Notes Due 2018 For US$375 Million 35
Medivation Acquires Rights to Talazoparib from BioMarin Pharma 37
Sanofi May Acquire BioMarin Pharma 38
Roche May Acquire BioMarin Pharma 39
BioMarin Pharmaceutical (BioMarin Pharma) Acquires 100% stake in Prosensa for USD751.5 Million 40
Roche Rumored to Acquire BioMarin Pharma for USD13.5 Billion 42
BioMarin Pharma Acquires Zacharon Pharma For USD9.7 Million 43
GlaxoSmithKline Plans To Acquire BioMarin Pharma 44
BioMarin Pharmaceutical Inc, Key Competitors 45
BioMarin Pharmaceutical Inc, Key Employees 46
BioMarin Pharmaceutical Inc, Subsidiaries 47
BioMarin Pharmaceutical Inc, Joint Venture 48
List of Figures
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals by Type, 2012 to YTD 2018 2
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 2
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 2
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 2
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 7
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals by Type, 2012 to YTD 2018 8
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 9
BioMarin Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 10
BioMarin Pharmaceutical Inc, Medical Devices Deals, 2012 to YTD 2018 12