BioMarin Pharmaceutical Inc.
About BioMarin Pharmaceutical Inc.
BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. In 1997, the company was founded to make a big difference in small patient populations, focusing on giving much-needed attention to the underserved communities of those with rare diseases. BioMarin sought to help patients whose voices could not always be heard and treat conditions that were not always in mainstream medical conversations. Throughout its history, BioMarin has worked tirelessly to make a difference by pursuing bold science. The company’s first approved therapy was inspired by a father who refused to accept there was no therapy for his son’s rare diagnosis. That relentless pursuit to change the course of his son’s future through the power of science is at the core of who BioMarin is today.
Through the company’s expertise in genetics and molecular biology, BioMarin has developed eight first or best-in-class targeted therapies that address the root cause of the conditions it seeks to treat, helping to better the lives of those struggling with rare genetic disease. The company’s purposeful and scientifically precise team of physicians and scientists has honed each treatment modality to seek transformative therapies.
Now, BioMarin seeks to make an even greater impact by applying the same science-driven, patient-forward approach that propelled the last 25 plus years of drug development to larger genetic disorders, as well as genetic subsets of more common conditions. Each drug candidate pursued is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address. The company believes that applying its knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most. The more innovative solutions developed, the more lives BioMarin can impact.
803 articles with BioMarin Pharmaceutical Inc.
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European Commission Approves BioMarin's VOXZOGO® (vosoritide) for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close
8/27/2021
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Commission (EC) has granted marketing authorization for VOXZOGO® (vosoritide), a once daily injection to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height.
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BioMarin to Participate in Three Upcoming Virtual Investor Conferences
8/25/2021
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in three upcoming virtual investor conferences.
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The U.S. FDA had a very packed calendar for this week, but almost everything on it was under a Priority Review pathway.
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BioMarin Announces Second Quarter 2021 Financial Results and Corporate Updates
7/28/2021
BioMarin Pharmaceutical Inc. announced financial results for the second quarter ended June 30, 2021.
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New Trial Data Call into Question Long-Term Efficacy of BioMarin’s Hemophilia A Gene Therapy
7/23/2021
Updated trial data show patients treated with BioMarin Pharmaceutical’s severe hemophilia A gene therapy expressed substantially lower factor VIII levels after five years compared with one year, calling into question the drug’s long-term benefits. -
BioMarin Announces Oral Presentation at ISTH 2021 Virtual Congress with 5 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec in Adults with Severe Hemophilia A, Demonstrating Continued, Durable Clinical Benefit
7/21/2021
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today new data for valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A, from its open-label Phase 1/2 study during an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.
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BioMarin Announces Oral Presentation of Positive One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults
7/19/2021
BioMarin Pharmaceutical Inc. today announced new data for valoctocogene roxaparvovec in its positive pivotal study, GENEr8-1, during an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.
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BioMarin to Host Second Quarter 2021 Financial Results Conference Call and Webcast on Wednesday, July 28 at 4:30pm ET
7/14/2021
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday, July 28 , at 4:30 p.m. ET to discuss second quarter 2021 financial results and provide a general business update.
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BioMarin Announces 12 Presentations at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress
7/2/2021
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced three oral presentations and nine poster presentations related to valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress being held July 17-21, 2021
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BioMarin Resubmits Marketing Authorization Application (MAA) to European Medicines Agency for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A
6/28/2021
BioMarin Pharmaceutical Inc. announced today that the company resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec.
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BioMarin Receives Positive CHMP Opinion in Europe for Vosoritide for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close
6/25/2021
European Commission Decision expected Q3 2021 Temporary Authorization for Use (ATU) Granted in France to Allow Access and Reimbursement of Vosoritide to Begin Immediately
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BioMarin to Participate in the Virtual BofA Securities 2021 Napa Biopharma Conference
6/15/2021
BioMarin Pharmaceutical Inc. today announced that management will participate in the BofA Securities 2021 Napa Biopharma Conference on June 16, 2021 at 3:30pm ET .
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BioMarin to Participate in Two Upcoming Virtual Investor Conferences - May 27, 2021
5/27/2021
BioMarin Pharmaceutical Inc. announced that management will participate in two upcoming virtual conferences.
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BioMarin Provides Highlights of 5 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec with the Longest Duration of Clinical Experience for a Gene Therapy in Hemophilia A
5/19/2021
All Study Participants in 6e13 vg/kg and 4e13 vg/kg Dose Cohorts Remain off Factor VIII Prophylactic Therapy 95% Reductions in Mean Annualized Bleed Rate (ABR) and 96% Reduction in Mean Annualized Factor VIII Usage Through Year 5 in 6e13 vg/kg Dose Cohort
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BioMarin to Participate in Two Upcoming Virtual Investor Conferences - May 18, 2021
5/18/2021
BioMarin Pharmaceutical Inc. announced that management will participate in two upcoming virtual conferences.
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BioMarin Announces 6 Presentations at American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting
5/12/2021
BioMarin today announced three oral and three poster presentations on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting being held May 11-14.
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BioMarin to Participate in Two Upcoming Virtual Investor Conferences - May 03, 2021
5/3/2021
BioMarin Pharmaceutical Inc. announced that management will participate in two upcoming virtual conferences.
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BioMarin Announces First Quarter 2021 Financial Results and Corporate Updates
4/29/2021
FDA Review of Vosoritide Proceeding; PDUFA Target Action Date Extended to November 20, 2021 to Provide Time for Review of Recently Submitted 2-year Phase 3 Results
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BioMarin Announces New and Updated Data at 2021 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting Demonstrating Commitment to Understanding Achondroplasia and Potential Treatment Choice
4/15/2021
Normal Progression of Bone Age with Chronological Age Over 60 Months with Vosoritide for Children with Achondroplasia
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BioMarin to Host First Quarter 2021 Financial Results Conference Call and Webcast on Thursday, April 29 at 4:30pm ET
4/13/2021
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday, April 29 , at 4:30 p.m. ET to discuss first quarter 2021 financial results and provide a general business update.