FDA approves drug to address form of dwarfism in children

Nov. 23, 2021

The U.S. Food and Drug Administration (FDA) has approved Voxzogo (vosoritide), an injection to improve growth in children 5 years of age and older with achondroplasia and open epiphyses (growth plates), meaning these children still have the potential to grow, the agency said in a news release.

Achondroplasia is the most common form of dwarfism, according to the FDA.

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.

Voxzogo’s was evaluated in a year-long, double-blind, placebo-controlled, phase 3 study in participants 5 years and older with achondroplasia who have open epiphyses. In the study, 121 participants were randomly assigned to receive either Voxzogo injections under the skin or a placebo. Researchers measured the participants’ annualized growth velocity, or rate of height growth, at the end of the year. Participants who received Voxzogo grew an average 1.57 centimeters taller compared to those who received a placebo.

The FDA granted the approval of Voxzogo to BioMarin.

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