A phase 3 clinical trial published in NEJM Evidence demonstrates that once-daily injectable vosoritide significantly improves growth outcomes and quality of life for children with hypochondroplasia. Led by Dr. Andrew Dauber, the CANOPY HCH-3 trial of 81 children found that vosoritide increased annualized growth velocity by 2.33 cm per year and mean standing height by 2.35 cm over placebo after one year. Currently, there are no FDA-approved treatments for hypochondroplasia.
Vosoritide phase 3 trial results
- ▪The CANOPY HCH-3 trial was a randomized, double-blind, placebo-controlled study conducted at 23 sites across nine countries.
- ▪Participants in the CANOPY HCH-3 trial had a confirmed genetic diagnosis of hypochondroplasia and a height standardized deviation z score of -2 or less for age and sex.
- ▪The phase 3 CANOPY HCH-3 trial evaluated the efficacy and safety of once-daily subcutaneous vosoritide in 81 children aged 3 to 17 years with hypochondroplasia.
Annualized growth velocity increase
- ▪Children receiving once-daily subcutaneous vosoritide achieved a 2.33 cm per year greater increase in annualized growth velocity at 1 year compared with those receiving placebo.
- ▪The primary endpoint of the CANOPY HCH-3 trial was the change in annualized growth velocity from baseline to 1 year.
Height outcomes in children
- ▪The CANOPY HCH-3 trial found no difference in upper-to-lower body segment ratio between the vosoritide and placebo groups among children younger than 11 years.
- ▪Children receiving vosoritide in the CANOPY HCH-3 trial achieved a 2.35 cm greater increase in mean standing height at 1 year compared with those receiving placebo.
Adverse events profile
- ▪No serious adverse events, deaths, or incidents of slipped capital femoral epiphysis were reported in the vosoritide group during the CANOPY HCH-3 trial.
- ▪Adverse events occurred in 87.8% of the vosoritide group and 72.5% of the placebo group during the CANOPY HCH-3 trial, with no events leading to drug discontinuation.
Hypochondroplasia treatment approval
- ▪Dr. Andrew Dauber stated that longer-term research is needed to confirm if the growth effects of vosoritide are sustained and translate to improved function and decreased medical comorbidities.
- ▪The FDA previously approved vosoritide, marketed as Voxzogo by BioMarin Pharmaceutical, to improve growth in children of all ages with achondroplasia with open epiphyses.
- ▪There are currently no FDA-approved medications to treat hypochondroplasia, a skeletal condition that causes short stature in children.
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