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More Than 90% of Rare Diseases Are Still Without FDA-Approved Treatment

Did you know that more than 90% of rare diseases are still without an FDA-approved treatment?

On November 19, 2021, the U.S. Food and Drug Administration approved the first drug to improve growth in children with achondroplasia.

VOXZOGO® (vosoritide) for injection helps increase linear growth in children with achondroplasia aged 5 years and up whose growth plates are still open.

Voxzogo’s safety and efficacy in improving growth were evaluated in a year-long, double-blind, placebo-controlled, phase 3 study in participants five 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 most common side effects of Voxzogo include injection site reactions, vomiting and decreased blood pressure. Voxzogo’s labeling also lists decreased blood pressure as a warning and precaution, which means it is a potentially serious side effect.

The FDA approved Voxzogo under the accelerated approval pathway, which allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need, based on a surrogate or intermediate clinical endpoint. A condition of this accelerated approval is a post-marketing study that will assess final adult height. This application also received priority review designation.

The FDA granted the approval of Voxzogo to BioMarin.


Currently, voxzogo is approved in the US, Europe, Australia, Brazil, and Japan.

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