After FDA Delays Hemophilia Gene Therapy, BioMarin's Dwarfism Drug Under FDA Review For Expanded Use

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  • The FDA has accepted BioMarin Pharmaceutical Inc's BMRN supplemental marketing application for Voxzogo (vosoritide) to expand treatment to include children with achondroplasia under the age of 5. 
  • Achondroplasia is the most common form of disproportionate short stature.
  • The FDA has set a PDUFA target action date of Oct. 21, 2023, for the supplemental application.
  • In January, the European Medicines Agency validated BioMarin's application for an extension of indication for Voxzogo to treat children with achondroplasia under the age of 2. 
  • Voxzogo is the first FDA and EMA-approved treatment for children with achondroplasia with open bone growth plates.
  • The positive move comes one day after the FDA said it needed more time to review a three-year analysis from BioMarin's ongoing Phase 3 GENEr8-1 study of Hemophilia A gene therapy, which BioMarin submitted earlier this year.
  • The actions date has been pushed to Jun. 30 from Mar. 31.
  • BioMarin's shares plunged on Monday after BridgeBio Pharma Inc BBIO reported positive data from the dwarfism drug.
  • RelatedBridgeBio's Dwarfism Candidate Shows Favorable Height Increase, Safety Profile
  • Price Action: BMRN shares traded lower by 0.43% at $93.02 on the last check Wednesday.
Posted In: BiotechNewsFDAGeneralBriefs
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