BioMarin Pharmaceutical Inc. announced that the European Commission (EC) 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.
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.
“Today represents an important milestone for the European achondroplasia community. For the first time medical professionals in Europe can offer a meaningful targeted therapeutic 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 vosoritide 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 vosoritide beyond height over the long term.”
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.
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