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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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.
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BioMarin to Host Third Quarter 2022 Financial Results Conference Call and Webcast on Wednesday, October 26, at 4:30pm ET
10/4/2022
BioMarin Pharmaceutical Inc. announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday, October 26th, at 4:30 p.m. ET to discuss third quarter 2022 financial results and provide a general business update.
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Two years after receiving a Complete Response Letter for its hemophilia gene therapy, BioMarin is heading back to the FDA with stronger and more robust data for potential approval.
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BioMarin Resubmits Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Severe Hemophilia A to the FDA
9/29/2022
BioMarin Pharmaceutical Inc. announced that the Company resubmitted a Biologics License Application to the U.S. Food and Drug Administration for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.
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BioMarin's Gene Therapy for Adults with Severe Hemophilia A, ROCTAVIAN™ (valoctocogene roxaparvovec), Assessed to Provide Substantial Cost Savings Per Patient in a Preliminary Independent Report
9/15/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced hat the Institute for Clinical and Economic Review (ICER) released a Draft Evidence Report updating the previous Hemophilia A assessment of the comparative clinical effectiveness and value of ROCTAVIAN ™ (valoctocogene roxaparvovec).
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The Institute for Clinical and Economic Review (ICER) concluded that even at a hefty lifetime cost of $2.5 million, gene therapies for hemophilia A and B are worth it.
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BioMarin’s Roctavian was granted conditional authorization in the European Union, making it the first gene therapy approved for severe hemophilia A. Now, the company is shooting for U.S. approval.
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First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec), Approved by European Commission (EC)
8/24/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the European Commission (EC) has granted conditional marketing authorization (CMA) to ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).
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BioMarin Announces Record Revenues in Second Quarter 2022; Increases Full-year 2022 Top and Bottom-line Guidance
8/3/2022
BioMarin Pharmaceutical Inc. announced financial results for the second quarter ended June 30, 2022.
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BioMarin to Host Second Quarter 2022 Financial Results Conference Call and Webcast on Wednesday, August 3, at 4:30pm ET
7/20/2022
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, August 3rd, at 4:30 p.m. ET to discuss second quarter 2022 financial results and provide a general business update.
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Sanofi announced positive data for its therapeutic, fitusiran, for treating patients with hemophilia A and B, as well as efanesoctocog alfa therapy for treating hemophilia A.
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BioMarin Presents Findings from Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 Congress July 9-12, Including 4 Oral and 2 Poster Presentations
7/11/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that it presented four oral presentations and two poster presentations on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 World Congress from July 9-12 in London.
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BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A
6/24/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, valoctocogene roxaparvovec.
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BioMarin Announces the Ministry of Health, Labor and Welfare (MHLW) in Japan Granted Approval for VOXZOGO® (vosoritide) for Injection for the Treatment of Children with Achondroplasia, Whose Growth Plates are Not Closed
6/21/2022
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan granted approval of the registration of VOXZOGO® (vosoritide) for injection, indicated for the treatment of achondroplasia in children of all ages, whose growth plates are not closed.
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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.