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.
841 articles about BioMarin Pharmaceutical Inc.
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BridgeBio posted data Monday from its small but highly anticipated Phase II study of infigratinib in achondroplasia - a drug that could give BioMarin a run for its money.
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BioMarin Provides Update on FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) Gene Therapy for Adults with Severe Hemophilia A
3/7/2023
BioMarin Pharmaceutical Inc. today announced that it received notice this afternoon from the U.S. Food and Drug Administration (FDA) that the agency has extended review of the company's Biologics License Application (BLA) for ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for adults with severe hemophilia A.
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FDA Accepts BioMarin's Supplemental New Drug Application to Expand Use of VOXZOGO® (vosoritide) for Injection to Treat Children with Achondroplasia Under the Age of 5
3/7/2023
BioMarin Pharmaceutical Inc. announced today that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection to expand treatment in the United States to include children with achondroplasia under the age of 5.
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BioMarin to Participate in Three Investor Conferences in March
3/2/2023
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in three upcoming investor conferences.
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BioMarin Announces Record Fourth Quarter and Full Year 2022 Total Revenues Driven by Strong Global Demand for VOXZOGO® and Steady Growth of Enzyme Business
2/27/2023
BioMarin Pharmaceutical Inc. announced financial results for the fourth quarter and full year ended December 31, 2022.
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BioMarin to Participate Virtually at SVB Securities Global Biopharma Conference on February 16, 2023
2/14/2023
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer and Brian Mueller, Executive Vice President, Chief Financial Officer of BioMarin will participate virtually in the SVB Securities Global Biopharma Conference on Thursday, February 16, 2023, at 11:20 a.m. ET.
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BioMarin Announces Stable and Durable Annualized Bleed Control for ROCTAVIAN™ in Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A; 134-Participant Study Met All Primary and Secondary Efficacy Endpoints at 3-Year Analysis
1/8/2023
BioMarin Pharmaceutical Inc. announced positive results from more than three years of follow up from its ongoing global Phase 3 GENEr8-1 study of ROCTAVIANTM, an investigational one-time gene therapy for the treatment of adults with severe hemophilia A.
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BioMarin to Present at 41st Annual J.P. Morgan Healthcare Conference on Monday, January 9, at 10:30 am PT /1:30 pm ET, in San Francisco, CA
1/4/2023
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé , Chairman and Chief Executive Officer, will present at the 41st Annual J.P. Morgan Healthcare Conference on Monday, January 9, 2023, at 10:30 am PT / 1:30 pm ET , in San Francisco, California.
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European Medicines Agency Validates Application for Extension of Indication for VOXZOGO® (vosoritide) for injection to Treat Children with Achondroplasia Under the Age of 2
1/3/2023
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, announced today that the European Medicines Agency (EMA) validated its Type II Variation application to extend the indication for VOXZOGO® (vosoritide) for injection to treat children with achondroplasia under the age of 2.
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BioMarin Pharmaceutical announced the FDA no longer plans to hold an advisory committee meeting to review its BLA for Roctavian an AAV gene therapy for adults with severe hemophilia A.
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BioMarin Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A
11/23/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced advancements in the U.S. Food and Drug Administration (FDA) review of the Biologics License Application (BLA) of ROCTAVIAN™ (valoctocogene roxaparvovec AAV gene therapy) for adults with severe hemophilia A.
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A request for additional data may cause a 3-month delay for potential approval of BioMarin Pharmaceutical’s hemophilia A gene therapy.
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BioMarin Announces Incremental Progress on Biologics License Application (BLA) Review for Valoctocogene Roxaparvovec AAV Gene Therapy for Adults with Severe Hemophilia A Program
11/7/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today a progress update on its Biologics License Application (BLA) for valoctocogene roxaparvovec AAV gene therapy for adults with severe Hemophilia A that is currently under review by the U.S. Food and Drug Administration (FDA).
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BioMarin to Participate in Two Upcoming November 2022 Investor Conferences
11/3/2022
BioMarin Pharmaceutical Inc. announced that management will present at two upcoming investor conferences.
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During its third-quarter earnings report, BioMarin noted that the FDA planned to hold an advisory committee meeting to discuss the company's gene therapy for hemophilia A.
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BioMarin Announces Third Quarter 2022 Year-over-Year Total Revenue Growth of 24% (31% Excluding KUVAN®)
10/26/2022
BioMarin Pharmaceutical Inc. announced financial results for the third quarter ended September 30, 2022.
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Bay Area Biotech Companies Hiring Now
10/19/2022
The Biotech Bay region is home to 3,418 life sciences companies and 96,574 employees making an average of $148,285. Here's a look at a few of the bay area biotech companies hiring now. -
FDA Accepts BioMarin's Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Adults with Severe Hemophilia A
10/12/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S. Food and Drug Administration (FDA) accepted the Company's resubmission of the Biologics License Application (BLA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.
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BioMarin Simplifies Organizational Structure to Increase Efficiency
10/7/2022
BioMarin Pharmaceutical Inc. announced its decision to redesign the organization to better focus investments that advance its R&D pipeline, maximize recent commercial launch success, prepare for a potential launch of ROCTAVIAN™ in the U.S., and drive core infrastructure optimization.
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BioMarin Pharmaceuticals announced Friday it was shedding around 4% of its global workforce, or around 120 jobs, in an effort to improve its operational efficiency.