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.
807 articles about BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc. announced that Brian Mueller, Executive Vice President, Chief Financial Officer of BioMarin will present at the Bank of America 2023 Health Care Conference on Wednesday, May 10, 2023 at 3:40pm PT, in Las Vegas, NV.
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BioMarin Announces Record Breaking First Quarter 2023 Results, Including 15% Year-over-year Growth of Total Revenues
BioMarin Pharmaceutical Inc. announced financial results for the first quarter ended March 31, 2023.
MiNA Therapeutics Enters Research Collaboration with BioMarin Pharmaceutical to Advance RNAa Platform Research in Rare Genetic Diseases
MiNA Therapeutics today announced a multi-target research collaboration and option licensing agreement with BioMarin Pharmaceutical Inc.
BioMarin to Host First Quarter 2023 Financial Results Conference Call and Webcast on Wednesday, April 26, at 4:30pm ET
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 26, at 4:30 p.m. ET to discuss first-quarter 2023 financial results and provide a general business update.
BioMarin to Share Updated Data at 2023 American College of Medical Genetics and Genomics (ACMG) Meeting Demonstrating Commitment to Understanding Long-Term Benefit of VOXZOGO® in Children with Achondroplasia
BioMarin Pharmaceutical Inc. will present updated data demonstrating the long-term benefit of treatment with VOXZOGO® (vosoritide) and new observational data on disease burden in children with achondroplasia.
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.
BioMarin Provides Update on FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) Gene Therapy for Adults with Severe Hemophilia A
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.
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
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.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will participate in three upcoming investor conferences.
BioMarin Announces Record Fourth Quarter and Full Year 2022 Total Revenues Driven by Strong Global Demand for VOXZOGO® and Steady Growth of Enzyme Business
BioMarin Pharmaceutical Inc. announced financial results for the fourth quarter and full year ended December 31, 2022.
BioMarin to Participate Virtually at SVB Securities Global Biopharma Conference on February 16, 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.
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
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.
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
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.
European Medicines Agency Validates Application for Extension of Indication for VOXZOGO® (vosoritide) for injection to Treat Children with Achondroplasia Under the Age of 2
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.
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.
BioMarin Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A
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.
A request for additional data may cause a 3-month delay for potential approval of BioMarin Pharmaceutical’s hemophilia A gene therapy.
BioMarin Announces Incremental Progress on Biologics License Application (BLA) Review for Valoctocogene Roxaparvovec AAV Gene Therapy for Adults with Severe Hemophilia A Program
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).
BioMarin Pharmaceutical Inc. announced that management will present at two upcoming investor conferences.