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.
828 articles about BioMarin Pharmaceutical Inc.
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BioMarin Announces Favorable Results from Global Phase 2 Study of VOXZOGO™ (vosoritide) for Injection in Infants and Young Children with Achondroplasia at The Endocrine Society Annual Meeting, ENDO 2022 (June 11-14), in Atlanta
6/13/2022
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) presented data at The Endocrine Society Annual Meeting, ENDO 2022, demonstrating the Company's ongoing commitment to understanding the lifetime impact of achondroplasia, the most common form of disproportionate short stature.
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It was a busy, busy week for clinical trial news and updates, largely driven by the annual ASCO meeting taking place June 3-7 in Chicago.
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BioMarin Delays Hemophilia A Therapy Valoctocogene Roxaparvovec Filing as FDA Calls for More Data
5/31/2022
The U.S. FDA has requested some additional data from BioMarin Pharmaceuticals for the BLA it filed for the hemophilia A therapy valoctocogene roxaparvovec. -
BioMarin Announces Durable Hemostatic Efficacy Maintained Over 6 years in Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec, Investigational Gene Therapy for Hemophilia A
5/31/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today updated results from its ongoing open-label Phase 1/2 study, which represents the longest duration of clinical observation for valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A.
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BioMarin to Present Findings from Ongoing Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the World Federation of Hemophilia 2022 World Congress, May 8-11, 2022, Including Five Platform Presentations
5/5/2022
BioMarin Pharmaceutical Inc. today announced five platform presentations and one poster presentation on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the World Federation of Hemophilia (WFH) 2022 World Congress from May 8-11 in Montreal, Canada.
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BioMarin Hires Pharmaceutical Industry Leaders in Marketing and Accounting to Fill Strategic Roles Supporting Company's Growth Plans
5/5/2022
BioMarin Pharmaceutical Inc. announced that the Company hired two pharmaceutical industry leaders in their respective areas of expertise.
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BioMarin to Participate in the BofA Securities 2022 Healthcare Conference, Thursday, May 12 at 8:40amPT/11:40amET
5/4/2022
BioMarin Pharmaceutical Inc., announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will present at the BofA Securities Healthcare Conference on May 12 at 8:40am PT/11:40am ET in Las Vegas.
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The Q1 results for Voxzogo gave BioMarin the confidence to raise its 2022 full-year guidance to between $100 million to $125 million (net product revenue) for the drug.
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BioMarin Announces Record Revenues in First Quarter 2022
4/27/2022
BioMarin Pharmaceutical Inc. announced financial results for the first quarter ended March 31, 2022.
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In honor of World Hemophilia Day, BioSpace takes a look at some of the facts relating to the disease, and some of the highlights in recent hemophilia research and development.
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BioMarin to Host First Quarter 2022 Financial Results Conference Call and Webcast on Wednesday, April 27, at 4:30pm ET
4/13/2022
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, April 27th, at 4:30 p.m. ET to discuss first quarter 2022 financial results and provide a general business update.
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It was a busy week for clinical trial announcements. Here's a look.
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BioMarin Pharmaceuticals Hemophilia A Data Indicate Potential for "Sustained Bleed Control"
3/18/2022
BioMarin Pharmaceuticals has published positive results in the New England Journal of Medicine from its Phase III clinical trial of valoctocogene roxaparvovec. -
BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A
3/17/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced publication of results from the Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, in the New England Journal of Medicine (NEJM).
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BioMarin Announces Fourth Quarter and Full Year 2021 Financial Results and Corporate Updates
2/23/2022
BioMarin Pharmaceutical Inc. announced financial results for the fourth quarter and full-year ended December 31, 2021.
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Silence Therapeutics, Totus Medicines and Poseida Therapeutics all adjust as they welcome new members to their leadership teams.
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Biomarin indicated that the FDA had requested more data from non-clinical studies to evaluate the theoretical cancer risk to human trial participants. This is likely to take several quarters.
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BioMarin Provides Updates on Progress in Gene Therapy Programs
2/17/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced updates on its investigational gene therapy programs in clinical development.
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BioMarin to Participate in Two Upcoming Virtual Investor Conferences - Feb 16, 2022
2/16/2022
BioMarin Pharmaceutical Inc. announced that management will participate in two upcoming virtual conferences.
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BioMarin to Host Fourth Quarter and Full Year 2021 Financial Results Conference Call and Webcast on Wednesday, February 23 at 4:30pm ET
2/10/2022
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, February 23rd, at 4:30 p.m. ET to discuss fourth quarter and full year 2021 financial results and provide a general business update.