VOXZOGO® (vosoritide for injection) is the only medicine indicated to increase linear growth in children with achondroplasia (4 months of age and older) whose epiphyses are not closed1. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.
MONTREAL, Feb. 11, 2026 /CNW/ – BioMarin has today announced Health Canada has granted a Notice of Compliance with Conditions (NOC/c) for PrVOXZOGO® (vosoritide for injection), the only approved medicine for eligible patients with achondroplasia in Canada.1
Achondroplasia is the most common form of dwarfism, affecting about 1 in 25,000 live births. The condition is caused by a gain-of-function pathogenic variant in the gene that encodes fibroblast growth factor receptor 3 (FGFR3), which leads to impaired endochondral ossification.2
VOXZOGO®, a C-type natriuretic peptide analogue, is the only pharmacological treatment available in Canada that has been demonstrated to improve body proportionality and increase growth velocity versus placebo in patients with achondroplasia.1,3
The treatment, first approved by the U.S. Food and Drug Administration (FDA) in 2021 and later by the European Medicines Agency (EMA)2, acts at the level of the intracellular signaling pathway downstream of the fibroblast growth factor receptor 3 (FGFR3) gene mutation, promoting endochondral ossification.1
“The approval of VOXZOGO in Canada marks a transformative moment for children and families living with achondroplasia. For the first time, we have a therapy that directly targets the underlying biology of the condition. This represents not only scientific progress, but real hope for families that have waited decades for new options,” said Dr. Peter Kannu, Paediatrician and Clinical Geneticist, University of Alberta.
Marie-Josee Toutounji, Medical Director, Canada, at BioMarin said “Health Canada’s authorization of VOXZOGO is a significant milestone for the achondroplasia community in Canada, and another step forward in BioMarin’s commitment to increase access to this therapy for patients and their families.”
Safety Summary
The results of the 111-301 study showed that VOXZOGO® was well tolerated. The most common adverse event related to its use was injection site reaction. The majority of events in the Phase 2 or 3 clinical studies were mild or moderate in severity.1,3
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.ca.
- VOXZOGO® Product Monograph. BioMarin Pharmaceutical (Canada) Inc.
- Savarirayan R, et al. International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with achondroplasia. Nat Rev Endocrinol. 2025;21:314–324.
- Savarirayan R, et al. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. Med. 2025;6(5):100566.
SOURCE BioMarin Pharmaceutical (Canada) Inc.
