Voxzogo is the first treatment for achondroplasia, which is the most common form of disproportionate short stature, said the company

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FDA Centre for Devices and Radiological Health. (Credit: The U.S. Food and Drug Administration/Wikipedia.)

BioMarin Pharmaceutical has received the US Food and Drug Administration (FDA) accelerated approval for Voxzogo (vosoritide) to treat achondroplasia in children.

Voxzogo is indicated for increasing linear growth in paediatric patients with achondroplasia, aged five years and above, with open growth plates (epiphyses).

The US agency granted the indication under accelerated approval, based on an improvement in annualised growth velocity (AGV).

In addition to the approval, the FDA has also issued a Rare Pediatric Disease Priority Review Voucher (PRV), which facilitates priority review for a drug.

BioMarin is planning to use the ongoing open-label extension studies for continued approval in the same indication, which is subject to clinical benefit in confirmatory studies.

BioMarin chairman and chief executive officer Jean-Jacques Bienaimé said: “Voxzogo is a medical first that is rooted in BioMarin’s focus on molecular genetics and targets the underlying cause of the condition.

“More than a decade of scientific research underpins the medical advance that Voxzogo represents. We thank the FDA for recognizing its value as the first therapeutic treatment option for children with achondroplasia.

“We extend our gratitude to the community, clinical investigators and the children and their families, who participated and continue to participate in our comprehensive clinical research program as we continue to investigate the full potential of vosoritide.”

According to the company, endochondral bone growth is negatively regulated in with achondroplasia, due to mutation in fibroblast growth factor receptor 3 (FGFR3) gene.

Voxzogo is a C-type natriuretic peptide (CNP) analog, which serves as a positive regulator of the signalling pathway downstream of FGFR3 to promote endochondral bone growth.

It is the first treatment for achondroplasia, which is the most common form of disproportionate short stature, said the company.

The FDA approval was supported by results from a Phase 3 study evaluating the efficacy and safety of Voxzogo in 121 paediatric patients with achondroplasia, aged 5 to 14.9 years.

In the Phase 3 study, Voxzogo reduced the transient decreases in blood pressure, and showed a safety and efficacy of the dug in patients with achondroplasia.

The most common adverse reactions include injection site reactions, vomiting, joint pain, decreased blood pressure, gastroenteritis, diarrhoea, dizziness, ear pain, influenza, fatigue, seasonal allergy, and dry skin.

Voxzogo clinical trials investigator Lynda Polgreen said: “Achondroplasia is a lifelong genetic condition resulting from the disordered skeletal architecture caused by impaired endochondral bone growth throughout childhood.

“This approval is an important milestone representing the first time that physicians will be able to offer a therapy targeted at the root cause of the condition for families of children with achondroplasia aged five and older.”

In August this year, Voxzogo has receive the European Commission (EC) approval, with marketing authorisation reviews underway in Japan, Brazil, and Australia.

BioMarin aims to commercialise Voxzogo in the US by mid- to late-December this year.