BioMarin Pharmaceutical Inc. (BMRN) said Friday that the U.S. Food and Drug Administration has approved the supplemental New Drug Application or sNDA for Voxzogo (vosoritide) to increase linear growth in pediatric patients with achondroplasia with open epiphyses or growth plates. This indication is approved under accelerated approval based on an improvement in annualized growth velocity.
Previously, Voxzogo was indicated for children who were 5 years of age and older.
This expanded indication now includes children of all ages with open growth plates.
Voxzogo is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates.
Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face, and base of the skull. This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth.
In September, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorization to expand the indication for Voxzogo for injection to treat children with achondroplasia aged 4 months and older whose epiphyses are not closed.
A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission later this year.
Voxzogo is also approved in Japan in children from birth who have achondroplasia with open growth plates. In addition, it is approved in Brazil in children who are 6 months and older with open growth plates as well as in Australia in children with achondroplasia who are 2 years of age and older with open growth plates.
Voxzogo also recently received orphan drug designation from the FDA for the treatment of hypochondroplasia, a genetic condition caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene and characterized by impaired bone growth.
BioMarin plans to initiate a pivotal development program in hypochondroplasia later this year.
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