European Commission approves Voxzogo to treat achondroplasia.- BioMarin.

Voxzogo, a modified C-type natriuretic peptide (CNP), directly targets the underlying pathophysiology of achondroplasia by down regulating fibroblast growth factor receptor 3 (FGFR3) signaling and consequently promoting endochondral bone formation.

The EC based its decision on the totality of data from the Voxzogo clinical development program including the outcomes from the randomized, double-blind, placebo-controlled Phase III study evaluating the efficacy and safety of Voxzogo. The Phase III Study was further supported by the ongoing long-term safety and efficacy from the Phase II dose-finding study, which showed that growth rates have been sustained above participants' baseline rates and above the expected annualized growth velocity for untreated children with achondroplasia throughout the five-year observation period for which data are currently available. No acceleration of bone age was observed, suggesting that Voxzogo is not reducing the total duration of growth. The data package included results from an ongoing Phase II randomized double-blind study in infants and young children, including extensive pharmacokinetic and biomarker data, as well as preliminary growth data from participants in the 2 to 5-year age cohort. Data in sentinel study participants showed a positive effect on growth following two years of Voxzogo treatment in subjects aged 2 to 5 years. In addition, the data package included data from the Phase III extension study and extensive natural history data. .

The U.S. New Drug Application (NDA) for Voxzogo is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of November 20, 2021. The Company successfully closed out the in-person FDA pre-approval inspection of its manufacturing facilities for Voxzogo earlier this year.