FDA approves first drug for genetic cause of dwarfism

The Food and Drug Administration approved the first treatment for the most common cause of dwarfism Friday, a drug that has proved to increase children’s height but has been polarizing among adults with short stature.

The treatment, developed by BioMarin Pharmaceutical, is a once-daily injection for children with achondroplasia, a rare genetic disorder that results in dwarfism and can lead to serious medical complications. In a pivotal clinical trial, patients who got the drug, called Voxzogo, grew 1.6 centimeters more over the course of a year than those who received placebo. That means patients who take Voxzogo throughout childhood are likely to reach heights similar to their peers who don’t have achondroplasia, according to BioMarin.

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