BioMarin’s application for Voxzogo in achondroplasia validated by EMA

BioMarin announced that the European Medicines Agency – EMA – validated its Type II Variation application to extend the indication for Voxzogo for injection to treat children with achondroplasia under the age of 2. Validation confirms the submission is complete and begins the EMA’s review process. The company also submitted a supplemental New Drug Application to the FDA to treat children with achondroplasia under the age of 5. Approval of the submissions would mean Voxzogo could potentially be prescribed as early as birth with more than 1,000 additional children eligible for treatment for achondroplasia, the most common form of disproportionate short stature in humans. The company anticipates action by health authorities in the second half of 2023. The supplemental marketing applications are based on the outcomes from a Phase 2 randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of Voxzogo for injection in infants and children aged 3 months to less than five years old.