BioMarin Pharmaceutical Inc.
ABOUT
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with multiple commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
770 Lindaro Street
San Rafael, CA 94901
105 Digital Drive
Novato, CA 94949
San Rafael, CA 94901
105 Digital Drive
Novato, CA 94949
NEWS
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.
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.
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.
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.
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.
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.
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.
The U.S. FDA has requested some additional data from BioMarin Pharmaceuticals for the BLA it filed for the hemophilia A therapy valoctocogene roxaparvovec.
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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