Voxzogo demonstrates positive results for infants with dwarfism

Biomarin’s achondroplasia treatment has been approved for children as young as five in the US, and the firm is now hoping to address patients who have yet to turn one.


In November 2021, the US Food and Drug Administration (FDA) market approved Biomarin’s drug, Voxzogo (vosoritide), for five-year-old children and up who have achondroplasia, also known as dwarfism.

The company has now publicized results from a phase II study that included patients with the disease younger than one year of age, according to a press release.

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