BioMarin Pharmaceutical (BMRN) said Monday that new clinical data showed its Voxzogo drug had a "meaningful impact" on tibial bowing in children under five years old with achondroplasia.
New studies showed that the drug had a significant reduction in the magnitude of tibial bowing compared to children who received a placebo, and the improvement was sustained in children who received the treatment for several years, the company said in a statement.
Voxzogo also showed early investigational efficacy in other skeletal conditions, according to the company.
BioMarin said it continues to advance its clinical program for Voxzogo in hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency.
The company recently completed enrollment in its phase 3 trial for hypochondroplasia and said it expects to report topline data in 2026, with a potential launch targeted for 2027.
Shares of BioMarin were up 4.4% in recent trading.
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