Successful trial shows new drug promotes bone growth in children with achondroplasia

A phase III multicentre clinical trial conducted in seven countries (Australia, Germany, Japan, Spain, Turkey, USA and UK) has successfully shown that a new drug boosts bone growth in children born with achondroplasia.

The study has been recently published in The Lancet and was led by Murdoch Children’s Research Institute along with investigators, Dr Louise Tofts, Paediatric Rehabilitation Physician at Kids Rehab, Children’s Hospital at Westmead and the Clinical Research Centre team at Kids Research.

Achondroplasia is a type of rare genetic bone disorder and is the most common cause of dwarfism. It affects 250,000 people worldwide, or about one in every 25,000 children. The condition is caused by a mutation in the growth factor receptor 3 (FGFR3) gene, which impairs bone growth resulting in children growing around 4 cm per year, instead of the usual 6 to 7 cm.

The experimental drug, Vosoritide, manufactured by BioMarin Pharmaceutical tested in this trial blocks the activity of FGFR3, potentially returning growth rates to normal. A total of 121 children aged 5 to 17 were enrolled in this trial - with 60 children receiving daily injections of Vosoritide grew an average of 1.57 cm per year more than the children who received placebo, which brought them almost in line with their typically developing peers.

The results from this study show that Vosoritide is effective for restoring typical growth velocity over 12 months in older children with achondroplasia, reducing the physical disability that comes with having severe short stature.

We are continuing to study its effects on younger children, where there is potential for modification of craniofacial growth and therefore the more severe complications of the condition, said Dr Tofts.

Click here to read the full study