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

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.
Related: BridgeBio'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.