BioMarin Pharmaceutical 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. Voxzogo is the first medicine to be approved to treat children with achondroplasia in Europe. Voxzogo, a modified C-type natriuretic peptide (CNP), directly targets the underlying pathophysiology of achondroplasia by downregulating fibroblast growth factor receptor 3 (FGFR3) signalling and consequently promoting endochondral bone formation. 

"Today represents an important milestone for the European achondroplasia community. For the first time medical professionals in Europe can offer a meaningful targeted treatment option for children and families affected by achondroplasia," said Klaus Mohnike, Professor of Paediatrics at Magdeburg University Hospital in Germany and investigator for the Voxzogo clinical program. "Achondroplasia is a serious, progressive, and lifelong condition, which can cause multi-system complications that in some cases require surgical intervention.  This regulatory approval is based on improved height gain, one important determinant of day-to-day function for people with achondroplasia, and is a first step to understand the potential benefits of Voxzogo beyond height over the long term." 

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 Voxzogo.  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 Temporary Authorization for Use to allow access of Voxzogo 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.

"We are committed to advancing the care of children affected by achondroplasia and are pleased to be able to offer a genetically targeted medicine that when administered in children over the age of two only while skeletal growth plates are open but could potentially offer benefit over a lifetime," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We are grateful to the families and study investigators and their teams, who dedicated their time to the clinical program to make this treatment option a reality for this community in the EU.  Voxzogo is the most widely studied therapeutic option for achondroplasia with an ongoing robust clinical program built on more than a decade of research and development."

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 3 study evaluating the efficacy and safety of Voxzogo.  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 Voxzogo 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 Voxzogo 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 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.