BioMarin Pharmaceutical has seen its supplemental New Drug Application (sNDA) for Voxzogo (vosoritide) given the green light for use with children under five living with achondroplasia.

Voxzogo is intended to encourage linear growth in paediatric patients with achondroplasia with open epiphyses. Previously the drug had only been intended for children over five years old but the FDA approval has expanded the drug’s market.

The approval comes after BioMarin conducted a randomised, double-blind, placebo-controlled Phase II clinical trial evaluating the safety and efficacy of Voxzogo in children younger than five years of age.

Dubbed Study 111-206, researchers saw an improvement in linear growth in children with achondroplasia, with children ages two years and older exhibiting a mean height Z-score improvement of 1.1 to 1.4 as well as a mean height gain of 6.3 to 7.8 centimetres, when compared to untreated children with achondroplasia of the same age and sex.

The Phase II study also comes alongside another Phase III study in pediatric patients ages five years and older (Study 111-301), both intended to prove the efficacy and tolerability of the drug.

At present Voxzogo is currently marketed in the European Union in children with achondroplasia who are 2 years of age and older with open growth plates, and BioMarin has said that it has been able to secure increased fill-finish commitments in 2024 in order to keep up with demand.

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Hank Fuchs, president of research and development at BioMarin, said: “We are grateful for the collaboration of the achondroplasia community, physicians, and the children and their families who have played a crucial role in advancing this clinical program.

“We also look forward to further understanding the potential role of Voxzogo in other genetic short stature conditions, including hypochondroplasia.”

GlobalData’s Pharmaceutical Intelligence Centre found the 2022 global sales Voxzogo sat at around $169m, only one year after initial approval. This figure is expected to rise to $1.3bn by 2029.

GlobalData is the parent company of Pharmaceutical Technology.