Update to the Vosoritide (Voxzogo TM) indication in USA
On October 20, 2023 US Food and Drug Administration (FDA) has extended approval of Voxzogo to children with Achondroplasia of all age groups with open epiphyses under accelerated approval program (VOXZOGO.COM). The results from an open-label, long-term phase 2 extension study were presented by BioMarin at the 2023 European Society for Pediatric Endocrinology Meeting.
Previously, in 2021, the FDA approved Voxzogo for children with Achondroplasia over 5 years of age with open epiphyses.
Vosoritide original approval 2021:
New Meds and Tech from PES Drugs and Therapeutics committee
VOXZOGOTM
Anna Ryabets-Lienhard, DO
On November 19, 2021, the FDA approved VOXZOGOTM (vosoritide) for the treatment of children over five years of age with achondroplasia and open epiphyses to increase linear growth. VOXZOGOTM was approved under an accelerated program based on improved annualized growth velocity (AGV); continued approval is contingent upon continued review and verification of clinical benefit in ongoing extension clinical trials. Vosoritide is a biological analog of C-type natriuretic peptide (CNP), which stimulates cartilaginous bone growth via endochondral ossification. The approved dose is approximately 15 mcg/kg administered subcutaneously once daily; a weight-based dosing chart is available in the FDA published prescribing information (https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214938s000lbl.pdf). VOXZOGOTM is provided as a lyophilized powder in a single-dose vial at 0.4 mg, 0.56 mg, or 1.2 mg. It requires reconstitution with a prefilled co-packed diluent (sterile water) delivered in a syringe prior to administration. Due to transient decrease in blood pressure that has been observed with VOXZOGO,TM the patient needs to be well fed and hydrated (approximately 240-300 mL of fluid) an hour prior to administration of the medication to reduce the risk of hypotension.