FDA Approves First Drug for Boosting Growth in Kids With Dwarfism

The FDA has approved BioMarin Pharmaceutical’s Voxzogo (vosoritide), an injectable drug for increasing growth in children five years and up with achondroplasia, the most common form of dwarfism.

The FDA green light was supported by a 121-person phase 3 study showing children receiving Voxzogo grew 1.57 centimeters on average vs. placebo.  

This is the first approval of its kind for this pediatric population, but since Voxzogo was cleared under an accelerated pathway, the drug must still be evaluated in a confirmatory trial assessing final adult height.

The approval “fulfills an unmet medical need for more than 10,000 children in the United States,” said Theresa Kehoe, the FDA’s director of the Division of General Endocrinology within the Center for Drug Evaluation and Research.