BioMarin has reported phase 3 results for its dwarfism drug Voxzogo in children with hypochondroplasia (HChttps://lifescivoice.com/H), a rare genetic condition that affects bone growth. According to the company, the CANOPY-HCH-3 study met its primary endpoint, with Voxzogo showing statistically significant improvements in growth measurements compared with placebo.
The study enrolled 80 children aged 3 to 17 with hypochondroplasia. At week 52, children treated with Voxzogo recorded an annualized growth velocity (AGV) that was 2.33 centimeters greater than those who received a placebo. BioMarin described the result as “highly statistically significant.”
The company also reported statistically significant improvements in additional growth-related measurements, including standing height, height Z-score, and arm span. According to BioMarin, increased arm span may help improve reach and support independence for patients. Detailed findings from the CANOPY-HCH-3 study are expected to be presented at a medical conference later this year.
Hypochondroplasia is a rare genetic disorder caused by mutations in the FGFR3 gene, which slows the conversion of cartilage into bone. The condition results in short-limbed dwarfism, with affected individuals typically having an average-sized torso but disproportionately shorter arms, legs, hands, and feet. It may also lead to ear, nose, and throat complications as well as neurological issues. Hypochondroplasia is considered a milder form of achondroplasia (ACH).
Voxzogo, a CNP analogue, was approved by the FDA in 2021 as a treatment for achondroplasia. Two years later, the approval was expanded to include children aged 5 years and younger. BioMarin has stated that there are currently no approved therapies for hypochondroplasia.
Greg Friberg, M.D., BioMarin’s head of research and development, said the phase 3 findings had exceeded the company’s expectations. Andrew Dauber, lead investigator of the study and chief of endocrinology at Children’s National Hospital in Washington, D.C., described the results as encouraging. “Seeing these improvements in growth is a milestone we have hoped for after so many years without treatment options,” Dauber said in a release. He added that the findings suggest the possibility of a new approach to caring for children with hypochondroplasia.
Based on the study results, BioMarin said it expects to submit an application for approval of Voxzogo in hypochondroplasia during the third quarter of this year. The company also plans submissions to the European Medicines Agency and other regulators.
The results come as other companies advance treatments for achondroplasia. Two months ago, Ascendis received FDA approval for Yuviwel for children aged 2 and older with open growth plates. Unlike Voxzogo, which is administered daily, Yuviwel is dosed weekly. In February, BridgeBio reported successful phase 3 results for infigratinib in children with achondroplasia, and the company and partner Kyowa Kirin plan to seek approval in the second half of this year.
Voxzogo recorded $927 million in sales in 2025, an increase of 26%, and accounted for 29% of BioMarin’s revenue. In March, BioMarin also announced it had discontinued phase 2 trials of Voxzogo in Turner syndrome, SHOX-deficiency, and aggrecan-deficiency, while studies in Noonan syndrome and idiopathic short stature without ACAN-deficiency remain ongoing.
BioMarin Shares Positive Clinical Trial Findings
BioMarin has announced encouraging Phase 3 clinical trial results for Voxzogo in children diagnosed with hypochondroplasia. The latest update from BioMarin demonstrated measurable growth improvements, strengthening confidence in the therapy’s long-term potential for treating rare skeletal growth disorders.
BioMarin Advances Rare Disease Innovation
The recent study highlights how BioMarin continues to focus on innovative treatments for genetic conditions with limited therapeutic options. According to the company, children receiving Voxzogo experienced improved annualized growth velocity compared to previous baseline measurements.
Voxzogo Shows Promise in Hypochondroplasia Treatment
With these findings, BioMarin aims to expand the clinical value of Voxzogo beyond achondroplasia into hypochondroplasia treatment. Researchers noted that the therapy was generally well tolerated during the Phase 3 trial, adding to the positive safety profile already associated with the medicine.
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