Shots:

• The approval was based on the P-III study and the open-label extension to evaluate Voxzogo vs PBO in 121 children aged 5 to 14.9yrs. with achondroplasia. Following the completion of the 52wks. P-III study, 58 patients were enrolled into an open-label extension
• The results showed an improvement in AGV @2yrs of follow-up, 8 (13%) of 60 patients treated with Voxzogo had a total of 11 events of transient decreases in blood pressure compared to 3 (5%) of 61 patients on PBO @52wks. treatment period
• The therapy is expected to be available in the US in December while BioMarin will initiate the promotion of Voxzogo imminently. In Aug 2021, Voxzogo received the EC approval for the same indication

Ref: BioMarin | Image: BioMarin