BioMarin seeks full approval for VOXZOGO

• BioMarin’s supplemental application for full VOXZOGO approval in achondroplasia children was accepted by the FDA.
• The FDA set a PDUFA target action date of February 28, 2027.
• BioMarin said the submission includes long-term safety and efficacy data from three ongoing studies.

BioMarin Pharmaceutical (NASDAQ:BMRN) announced that the U.S. Food and Drug Administration accepted its supplemental New Drug Application for full approval of VOXZOGO (vosoritide) in children with achondroplasia, with an FDA decision target date of February 28, 2027.

The application seeks to convert VOXZOGO’s 2021 accelerated approval into traditional approval based on additional long-term safety and efficacy data collected from ongoing clinical studies.

The submission includes data from studies 111-205, 111-208 and 111-302, covering adult height measurements and additional clinical outcomes including body proportionality and arm span.

BioMarin said the FDA acceptance does not represent approval and that the agency will review the submitted data before making a decision.

VOXZOGO is a therapy developed by BioMarin for children with achondroplasia, the most common form of disproportionate short stature caused by genetic changes affecting bone growth.