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BioMarin’s achondroplasia drug gets nod for children of all ages

The FDA cleared BioMarin Pharmaceuticals’s Voxzogo injection for treating achondroplasia, a common form of short-limbed dwarfism in children.

HQ Team

October 21, 2023: The FDA cleared BioMarin Pharmaceuticals’s Voxzogo injection for treating achondroplasia, a common form of short-limbed dwarfism in children of all ages.

Achondroplasia — the most common form of skeletal dysplasia — leads to disproportionate short stature in humans. It is a genetic condition affecting a protein in the body called the fibroblast growth factor receptor.

In achondroplasia, this protein begins to function abnormally, slowing down the growth of bone in the cartilage of the growth plate.

It is characterised by the 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 fibroblast growth factor receptor 3  (FGFR3) gene, a negative regulator of bone growth. It occurs in about one in every 25,000 newborns.

Once-daily injection

Voxzogo, a once-daily injection, acts as a positive regulator and promotes endochondral bone growth. The drug was the first therapy to be approved by the FDA for treating the genetic disorder under its accelerated approval pathway.

The expanded use approval now includes children of all ages with open growth plates.

The FDA nod came after the “overall safety profile” of Voxzogo in children under 5 years of age was similar to that seen in older children during trials, according to the statement.

Since the introduction of Voxzogo in 2021, the company has seen “strong patient demand” for the medicine worldwide. 

BioMarin has recently been able to secure increased fill-finish commitments in 2024 and beyond to meet this additional demand. There are approximately 800 children under five with achondroplasia in the US.

European approval

Voxzogo is currently approved in Europe for children with achondroplasia who are two years of age and older with open growth plates. 

In September, the European Medicines Agency’s Committee for Medicinal Products for Human Use adopted a “positive opinion.”

The agency recommended marketing authorisation to expand the use of Voxzogo injection to treat children with achondroplasia aged four months and older whose epiphyses are not closed.

 A final approval, “typically consistent with the committee’s recommendation,” is expected from the European Commission later this year. 

Voxzogo also recently received orphan drug designation from the FDA for the treatment of hypochondroplasia, a genetic condition caused by a mutation in the FGFR3 gene and characterised by impaired bone growth.

BioMarin plans to initiate a pivotal development program in hypochondroplasia later this year.

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