BioMarin Pharmaceutical Inc. announced that the European Commission (EC) has granted marketing authorization for VOXZOGO (vosoritide), a once daily injection to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height. Vosoritide is the first medicine to be approved to treat children with achondroplasia in Europe. Vosoritide, 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. It is estimated that over 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia and could be eligible for treatment with vosoritide. Approximately a third of this population are in countries authorized under the EMA license. Also, the French National Agency for Medicines and Health Products Safety (ANSM) granted an Autorisation Temporaire d'Utilisation de cohorte (ATU cohort), or Temporary Authorization for Use to allow access of vosoritide to begin immediately under an authorized process. An ATU allows access to drugs not yet approved in France, when provided for rare diseases with no alternative options, and when the benefit/risk is presumed positive. The EC based its decision on the totality of data from the vosoritide clinical development program including the outcomes from the randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide. The Phase 3 Study was further supported by the ongoing long-term safety and efficacy from the Phase 2 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 vosoritide is not reducing the total duration of growth. The data package included results from an ongoing Phase 2 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 vosoritide treatment in subjects aged 2 to 5 years. In addition, the data package included data from the Phase 3 extension study and extensive natural history data. The U.S. New Drug Application (NDA) for vosoritide 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 vosoritide earlier this year. Vosoritide was generally well tolerated at all doses, and approximately 38,000 injections have been administered to children around the world. The majority of adverse events (AEs) were mild and no serious adverse events (SAEs) were reported as study drug related. Across all doses, injection site reactions and hypotension were the most common drug-related AEs. All injection site reaction events were transient. AEs of hypotension were mild and transient with the majority being asymptomatic and reported in the context of routine blood pressure measurements with minimal clinical impact. No new safety findings were observed. There were no AEs related to disproportionate bone growth or bone pathology. There has been no evidence of accelerated bone age (as assessed by radiologists blinded to the age of the subjects) or negative changes in bone mineral density.