In the past, the U.S. Food and Drug Administration approved Voxzogo (vosoritide) for children with achondroplasia who were five years old or older. Voxzogo, developed by BioMarin Pharmaceutical, is a once-daily injection of a C-type natriuretic peptide analog designed to increase linear growth.
However, shares Brian Park of MPR, the FDA recently approved the expansion of Voxzogo’s label to include children with achondroplasia of all ages. This means that children under five years old can also benefit from Voxzogo use.
Approved under Accelerated Approval, Voxzogo’s expanded label was supported using data from a Phase 2 clinical study and Phase 2 extension trial. In the study, researchers evaluated how safe and effective this treatment was in children under age five compared to a placebo. 75 participants enrolled. Researchers found that Voxzogo improved height and growth velocity. While some children experienced reactions like rashes or injection site issues, the drug was safe and well-tolerated. The benefits of Voxzogo were also highlighted in the extension study, where height improved significantly on this treatment compared to non-treated children.
What is Achondroplasia?
Gene Mutations: FGFR3
Achondroplasia is a bone growth disorder in which an overactive and over-produced protein in the body causes cartilage to ossify (turn into bone or bony tissue). Most cases of the gene mutation causing achondroplasia result from spontaneous mutations. However, in some cases, it can be inherited.
When the cartilage ossifies, it leads to disproportionate dwarfism. This means that people often have a normal-sized torso, but short stature and short limbs. Males with achondroplasia have an average height of 4 feet and 4 inches, with the average female height of 4 feet and 1 inch. Other symptoms and characteristics of achondroplasia include sleep apnea, hydrocephalus, bowed legs, low muscle tone, chronic ear infections, abnormal spinal curvature, difficulty bending elbows, and cranial or spinal stenosis.
