The first medicine designed for children living with achondroplasia has been approved for use by the European Commission!
Voxzogo®, or as it’s more commonly referred to as Vosoritide, has been approved for use in children living with achondroplasia from the age of two until their growth plates close. This is the first approved therapy for those living with achondroplasia in Europe!
BioMarin, who developed Vosoritide, has received marketing authorization in Europe. Their therapy targets the underlying cause of achondroplasia, which is the faulty FGFR3 gene. BioMarin estimates that over 11,000 children live with achondroplasia across Europe, the Middle East and Africa. Only 1/3rd of this population fall under EMA license.
Vosoritide is a once daily injection that hinders the signaling from the FGFR3 gene, which acts as a negative regulator to bone growth. Vosoritide adds the hormone CNP (c-type natriuretic peptide) into the growth plate of achondroplasia patients to promote bone growth. A person’s growth plate tends to close after puberty when he or she reaches his or her final height. It is why Vosoritide can be used until a person’s growth plate closes.
Vosoritide was approved for use by the European Commission based off the data from the Voxzogo® clinical development program. This program demonstrated the positive benefits to achondroplasia patients and highlighted the safety profile of the drug. Vosoritide did increase a patient’s growth velocity and all doses were generally well tolerated during clinical trials. To date, around 38,000 injections have been administered to achondroplasia children around the world.
Where does the rest of the world stand with access to Vosoritide?
In France, Vosoritide was granted Temporary Emergency Use. This means that French achondroplasia patients can now access Vosoritide without having to wait for approval from a regulatory body.
The U.S. New Drug Application (NDA) for Voxzogo® is under review by the United States Food and Drug Administration (FDA). There is a Prescription Drug User Fee Act (PDUFA) target action date for November 20, 2021.
In the US, Latin America, Middle East and most of the Asia Pacific, there is currently no licensed medicines for achondroplasia.
The Middle East and Africa are waiting on EMA approval of Vosoritide.
Did you know?
Voxzogo® received Orphan Drug Designation from the FDA and EMA for the treatment of children living with achondroplasia. This unique designation advances the evaluation and development of drugs that demonstrate promise for the diagnosis and/or treatment of rare diseases and conditions.
BioMarin has published a press release on the European Commission’s approval of Vosoritide on its website. View the press release now and help share the news!
