FDA Approves Vosoritide Injection for achondroplasia in children
The US food and drug administration has approved Vosoritide Injection for Treatment of Children With Achondroplasia. Vosoritide is the first therapeutic treatment indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses.Achondroplasia is the most common form of dwarfism.
The FDA granted the approval of Voxzogo to BioMarin.
"Today's approval fulfills an unmet medical need for more than 10,000 children in the United States and underscores the FDA's commitment to help make new therapies available for rare diseases," said Theresa Kehoe, M.D., director of the Division of General Endocrinology in the FDA's Center for Drug Evaluation and Research. "With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature."
Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth. The average height of an adult with achondroplasia is approximately four feet. People with achondroplasia have a genetic mutation that causes a certain growth regulation gene called fibroblast growth factor receptor 3 to be overly active, which prevents normal bone growth. Voxzogo works by binding to a specific receptor called natriuretic peptide receptor-B that reduces the growth regulation gene's activity and stimulates bone growth.
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