Drug For Short-Limbed Dwarfism From Biomarin Wins European Approval

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  • The European Commission (EC) has approved BioMarin Pharmaceutical Inc's BMRN Voxzogo (vosoritide) once-daily injection to treat achondroplasia.
  • The approval covers children from 2 years until growth plates are closed, which occurs after puberty when children reach final adult height.
  • Voxzogo is a modified C-type natriuretic peptide (CNP) that directly downregulates fibroblast growth factor receptor 3 (FGFR3) signaling and promoting endochondral bone formation. 
  • The FDA's US application for Voxzogo is under review with a target action date of November 20.
  • Related content: Benzinga's Full FDA Calendar
  • Price Action: BMRN stock is up 6.48% at $83.98 on the last check Friday.
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