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Voxzogo was developed by BioMarin and represents over a decade of scientific research that targets the underlying cause of achondroplasia.

On 19 November 2021, the U.S. Food and Drug Administration (FDA) approved Voxzogo (vosoritide), developed by BioMarin, for children with achondroplasia from the age of five, when children still have growth potential as their growth plates (epiphyses) are open.

This is an accelerated approval, a pathway that allows early access to medicines for serious conditions and fulfills an unmet medical need, but requires a post-marketing assessment study.

"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. With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature.”

Theresa Kehoe, M.D.
Dir. of the Division of General Endocrinology in the FDA’s Center for Drug Evaluation and Research

What is Voxzogo?

Achondroplasia is caused by a mutation in the FGFR3 gene that regulates bone growth. Voxzogo has shown efficacy in reducing this regulatory activity, leading to an improvement in short stature in children with this condition by stimulating bone growth.

The safety and efficacy of this drug were evaluated in a one-year, double-blind, placebo-controlled Phase 3 study in participants with achondroplasia (121) aged five years and older. At the end of the study, the researchers measured the growth rate in height, concluding that those who received Voxzogo grew an average of 1.57 cm more compared to those who received a placebo.

What are the side effects?

Injection site reactions, vomiting and decreased blood pressure have been reported during the study and will be listed in the drug's label. Voxzogo’s labeling also lists decreased blood pressure as a warning and precaution, which means it is a potentially serious side effect.

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