BioMarin to present data showing LT benefit of Voxzogo on children’s growth

BioMarin Pharmaceutical announced that new data on VOXZOGO in children with achondroplasia will be presented at the 61st Annual European Society for Paediatric Endocrinology Meeting in The Hague, Netherlands. In an open-label, long-term Phase 2 extension study, VOXZOGO demonstrated consistent and durable growth benefits in young children with achondroplasia when initiated before the age of 5. Over a four-year period, children aged 2 years and above who received VOXZOGO exhibited a mean height Z-score improvement exceeding 1 standard deviation compared to control groups and a height gain of more than 6 centimeters during this time period. Additionally, children under the age of 2 years, treated with VOXZOGO for three years, demonstrated substantial height gains, reflected in a mean height Z-score improvement of 0.79 standard deviations compared to controls and a height gain of more than 3 cm during this time period. Both groups of treated children demonstrated substantial restoration of height when compared to untreated children with achondroplasia. VOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates. Last week, the European Medicines Agency’s Committee for Medicinal Products for Human Use adopted a positive opinion recommending marketing authorization to expand the indication for VOXZOGO for injection to treat children with achondroplasia aged 4 months and older whose epiphyses are not closed. VOXZOGO is also currently approved in the United States in children with achondroplasia who are 5 years of age and older with open growth plates. The U.S. FDA has set a PDUFA Target Action Date of Oct. 21, 2023, for the company’s Supplemental New Drug Application for VOXZOGO to expand treatment in the U.S. to include children with achondroplasia under 5 years of age.