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European Commission Approves BioMarin's VOXZOGO® (vosoritide) for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close
Achondroplasia is the Most Common Cause of Dwarfism First Medicine Approved to Treat Children with Achondroplasia in Europe SAN RAFAEL, Calif., Aug. 27, 2021
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"Achondroplasia is the Most Common Cause of Dwarfism\n First Medicine Approved to Treat Children with Achondroplasia in Europe\n\n\nSAN RAFAEL, Calif., Aug. 27, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today 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 down regulating fibroblast growth factor receptor 3 (FGFR3) signaling and consequently promoting endochondral bone formation. \n\n \n \n \n \n \n \n\n \n\"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 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.\" \nIt 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 an Autorisation Temporaire d'Utilisation de cohorte (ATU cohort), or 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...