The European Medicines Agency has given a positive scientific review to a new treatment for achondroplasia, a condition that impairs bone growth and causes dwarfism. It is recommending that a marketing authorisation be issued to BioMarin Pharmaceutical Inc, the developer, for use of Voxzogo (vosoritide) in patients two years and older whose epiphyses, or growth plates of the bones, are not yet closed. This condition must be confirmed by genetic testing. The medicine was evaluated in 121 patients with achondroplasia between the ages of five and 18 years.
