India, July 13 -- BioMarin Pharmaceutical Inc. (BMRN) announced that the U.S. FDA has accepted its supplemental New Drug Application (sNDA) for VOXZOGO (vosoritide) to secure full approval for use in children with achondroplasia. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.
Achondroplasia is the most common form of skeletal dysplasia leading to disproportionate short stature caused by changes in the FGFR3 gene, a regulator of bone growth. VOXZOGO, which received accelerated approval in 2021, is intended to fulfill postmarketing requirements by demonstrating long-term clinical benefit and converting to traditional approval.
The sNDA submission is supported by data from three ongoing stud...