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BioMarin Receives FDA Approval for VOXZOGO™ (vosoritide) for Injection, Indicated to Increase Linear Growth in Children with Achondroplasia Aged 5 and Up with Open Growth Plates
1st Therapeutic Treatment for Achondroplasia, Most Common Form of Disproportionate Short Stature Investor Conference Call and Webinar to be Held on Friday,
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"1st Therapeutic Treatment for Achondroplasia, Most Common Form of Disproportionate Short Stature\n Investor Conference Call and Webinar to be Held on Friday, Nov. 19 at 11:30 AM Eastern\n\n\nSAN RAFAEL, Calif., Nov. 19, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to VOXZOGO™ (vosoritide) for Injection, indicated to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates). This indication is approved under accelerated approval based on an improvement in annualized growth velocity (AGV). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory studies. To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.\n\n \n \n \n \n \n \n\n \nVoxzogo is the first FDA approved treatment for children with achondroplasia. In patients with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 gene (FGFR3). Voxzogo, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.\n\"Voxzogo is a medical first that is rooted in BioMarin's focus on molecular genetics and targets the underlying cause of the condition. More than a decade of scientific research underpins the medical advance that Voxzogo represents. We thank the FDA for recognizing its value as the first therapeutic treatment option for children with achondroplasia,\" said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. \"We extend our gratitude to the community, clinical investigators and the children and their families, who participated and continue to participate in our comprehensive clinical research program as we continue to investigate the full potential of vosoritide.\" \n\"Achondroplasia is a lifelong genetic condition resulting from the disordered skeletal architecture caused by impaired endochondral bone growth throughout childhood,\" said...