VOXZOGO™ (vosoritide) earned U.S. Food and Drug Administration (FDA) approval on November 19, 2021, as the first therapeutic treatment for Achondroplasia. Pediatric orthopaedic surgeon Daniel Hoernschemeyer, MD began working with BioMarin Pharmaceutical in 2015 by participating in the Natural History study.
Study participants were screened for inclusion into the blinded, placebo randomized study for daily subcutaneous injection. Participation in this study lasted one year at which point the subjects were given the option to continue into an open-label study where the subject was given access to the study medication. It is now year three of the open-label study. Dr. Hoernschemeyer and his team will continue to follow their subjects to support the ongoing clinical benefit of Voxzogo.
Voxzogo, a C-type natriuretic peptide (CNP) analog, is indicated to increase linear growth in children 5 and up with open growth plates. BioMarin has been conducting research in molecular genetics for over a decade to develop Voxzogo. Their research has focused on the underlying cause of Achondroplasia, a gain of function mutation in fibroblast growth factor receptor 3 gene (FGFR3). In a progressive class of therapy, Voxzogo functions as a positive regulator downstream of FGFR3, promoting endochondral bone growth, the process of replacing growing cartilage with bone.
Achondroplasia, the most common form of short-limbed dwarfism, is a lifelong genetic condition. Before Voxzogo, treatments only focused on complications of the condition, rather than treating the cause of the condition. The milestone approval of this medication finally offers patients and their families a clinically proven treatment to increase growth. Voxzogo now has approval from the European Commission (EC) and the FDA, with reviews pending in Japan, Brazil, and Australia.
Congratulations to Dr. Hoernschemeyer and his research team!
