BioMarin Pharmaceutical has received the European Commission (EC) marketing approval for Voxzogo (vosoritide) to treat achondroplasia in children aged two years and above.
The drug was indicated as a once-daily injection to treat achondroplasia, which is the most common cause of dwarfism, in children aged two years and above, until growth plates are closed, after they reach final adult height.
Achondroplasia is a form of skeletal dysplasia, caused by a change in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth.
The disease is characterised by slowing of endochondral ossification and results in disproportionate growth in the long bones, spine, face and base of the skull.
Vosoritide is a modified C-type natriuretic peptide (CNP) that regulates fibroblast growth factor receptor 3 (FGFR3) signalling and promotes endochondral bone formation.
The US-based biotechnology company claimed that its Voxzogo is the first drug approved to treat children with achondroplasia in Europe.
BioMarin chairman and CEO Jean-Jacques Bienaimé said: “We are grateful to the families and study investigators and their teams, who dedicated their time to the clinical program to make this treatment option a reality for this community in the EU.
“Vosoritide is the most widely studied therapeutic option for achondroplasia with an ongoing robust clinical program built on more than a decade of research and development.
“We are committed to advancing the care of skeletal dysplasia and are proud to now provide the first pharmacological treatment option for achondroplasia in the EU.”
The EC approval is based on data from the vosoritide clinical development programme, including results from the Phase 3 study evaluating the efficacy and safety of vosoritide.
Long-term safety and efficacy from the ongoing Phase 2 dose-finding study further supported the approval, which showed sustained growth rates in kids with achondroplasia.
The company said that no acceleration of bone age was observed in the study, suggesting that the drug has no effect in reducing the total duration of growth.
In the trials, around 38,000 injections have been administered to children around the world, and the drug was generally well-tolerated at all doses.
The majority of adverse events (AEs) include mild injection site reactions and hypotension, with no serious adverse events (SAEs) reported in the study.
Currently, the US FDA is reviewing the New Drug Application (NDA) for vosoritide, with a Prescription Drug User Fee Act (PDUFA) target action date of 20 November 2021.
Vosoritide clinical programme investigator Klaus Mohnike said: “Today represents an important milestone for the European achondroplasia community.
“For the first time, medical professionals in Europe can offer a meaningful targeted therapeutic treatment option for children and families affected by achondroplasia.
“Achondroplasia is a serious, progressive, and lifelong condition, which can cause multi-system complications that in some cases require surgical intervention.
“This regulatory approval is based on improved height gain, one important determinant of day-to-day function for people with achondroplasia, and is a first step to understand the potential benefits of vosoritide beyond height over the long term.”