Voxzogo is being developed by BioMarin Pharmaceutical
The Food and Drug Administration (FDA) has approved the first drug to treat the most common form of dwarfism, achondroplasia. This is caused by mutations in the FGFR3 gene, which encodes instructions to build a protein involved in bone growth and development. These mutations push the gene into overdrive, impeding the process by which cartilage tissue gets replaced by bone; this leads to the development of short and abnormally shaped bones and causes people with achondroplasia to be of short stature.