EC approves BioMarin drug as first treatment for children with common form of dwarfism; Servier sNDA wins approval
The European Commission just approved its first drug for use in children with achondroplasia, a common form of dwarfism.
BioMarin received marketing authorization today for Voxzogo, also known as vosoritide. The drug is a once-daily injection to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty.
An estimated 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia — and a third of this population are in countries authorized under the EMA license. The French National Agency for Medicines and Health Products Safety also granted Voxzogo a “Temporary Authorization for Use” to allow immediate access.
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