---
title: Vosoritide Helped Children With Hypochondroplasia Grow Faster in a Phase 3 Trial
description: Published Phase 3 results show vosoritide helped children with hypochondroplasia grow faster, and BioMarin has asked the FDA to expand VOXZOGO's approval.
author: Dr Marina Nani (Editor-in-Chief)
date: 2026-09-10T11:26:05.497Z
updated: 2026-09-10T11:26:05.511Z
canonical: https://richhealthmagazine.com/article/vosoritide-hypochondroplasia-phase-3-trial-fda
image: https://cdn.nanimediahouse.com/pexels-a-happy-child-runs-through-a-grassy-field-under-a-blue-sky-r-8959621.jpg
categories: Health
content_type: News
region: United States
publication: Rich Health Magazine
schema_type: Article
---

Children with hypochondroplasia who took the drug vosoritide grew faster over a year than children given a placebo, according to results from a Phase 3 trial now published in the journal NEJM Evidence. Vosoritide, sold as VOXZOGO, is already approved in the United States for children with achondroplasia, the most common form of dwarfism. Its maker, BioMarin, has asked the Food and Drug Administration to expand that approval to include hypochondroplasia, a related but milder condition that affects bone growth in the arms and legs and comes from changes in the FGFR3 gene. The FDA has not yet ruled on the request, so this is published trial data and a pending application, not an available treatment for the condition.

## Children on the Drug Grew Nearly an Inch More in a Year

The study, [CANOPY-HCH-3](https://clinicaltrials.gov/study/NCT06455059), compared children taking vosoritide against children taking a placebo and tracked how fast they grew. After 52 weeks it met its main goal: children on the drug grew faster, adding about 2.33 centimeters per year in growth velocity over those on placebo, a gain of nearly an inch that was statistically significant. Their standing height rose by about 2.35 centimeters more than the placebo group over the same period. Children on vosoritide also showed early, not yet significant improvements in quality of life, and BioMarin says follow-up is continuing.

## The Safety Matched What the Drug Has Shown in Achondroplasia

The trial reported a safety profile consistent with what vosoritide has shown in children with achondroplasia. Most side effects were mild, and researchers reported no treatment-related serious adverse events. Greg Friberg, BioMarin's chief research and development officer, said the results provide a comprehensive picture of the impact of the drug across multiple measures of growth in children with hypochondroplasia. The company presented the findings as a late-breaking oral presentation at the European Society for Paediatric Endocrinology 2026 Annual Meeting.

For a family, the takeaway is narrow for now. Vosoritide is approved for achondroplasia, the hypochondroplasia results are newly published and encouraging on growth, and whether it can be prescribed for hypochondroplasia rests with the FDA. Hypochondroplasia has no cure, and any conversation about the drug for a child with the condition is one for the child's specialist as the application moves through review.

## FAQ

**Q: Can vosoritide increase height?**
In children with hypochondroplasia, the CANOPY-HCH-3 trial found the drug increased growth over a year compared with a placebo. The figures are an average across the trial, not a promise for any one child, and its effect on final adult height will take longer follow-up to know.

**Q: How is vosoritide given?**
For children approved to take it, VOXZOGO is given as an injection under the skin once a day, administered by a caregiver after a doctor sets the dose. It is not a pill.

**Q: What side effects did the trial find?**
The trial reported that most side effects were mild, with no treatment-related serious adverse events. In children who already take VOXZOGO for achondroplasia, the most common side effects have been injection-site reactions such as redness, swelling, itching or bruising.

**Q: When will the FDA decide?**
BioMarin has submitted its supplemental application and has not announced a decision date. Regulatory reviews of this kind typically run several months to about a year.