BioMarin Pharmaceutical (NSDQ:BMRN) announced today that the FDA granted accelerated approval for its Voxzogo injection.
San Rafael, California-based BioMarin’s Voxzogo (vosoritide) injection has an indication to increase linear growth in pediatric patients with achondroplasia (a form of dwarfism) five years of age and older with open epiphysis (growth plates). Continued approval may be contingent upon verification and description of clinical benefit in confirmatory studies, with BioMarin intending to use the ongoing open-label extension studies compared to available natural history.
According to a news release, Voxzogo represents the first FDA-approved treatment for children with achondroplasia. The treatment, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy that acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth, the company said.
FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV) that confers priority review to a subsequent drug application that would otherwise not qualify for such review. BioMarin expects Voxzogo to be available in the U.S. by mid- to late-December, with promotion beginning immediately.
Voxzogo has been approved in Europe since August, while authorization reviews are in progress in Japan, Brazil and Australia. BioMarin has eyes on approvals in those countries in 2022.
“Voxzogo is a medical first that is rooted in BioMarin’s focus on molecular genetics and targets the underlying cause of the condition. More than a decade of scientific research underpins the medical advance that Voxzogo represents. We thank the FDA for recognizing its value as the first therapeutic treatment option for children with achondroplasia,” BioMarin Chair & CEO Jean-Jacques Bienaimé said in the release. “We extend our gratitude to the community, clinical investigators and the children and their families, who participated and continue to participate in our comprehensive clinical research program as we continue to investigate the full potential of vosoritide.”