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BioMarin Investors 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 , Nov. 19 at 11:30 AM Eastern SAN 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 VOXZOGOTM ( 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 . Voxzogo 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 . " 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 . " " Achondroplasia is a lifelong genetic condition resulting from the disordered skeletal