24 June 2019

High hopes for drug to combat dwarfism

Paediatrics Research

An Australian-led drug trial has shown promise in improving growth rates for children with achondroplasia, the most common form of dwarfism, making it possible for them to grow at close to the same rate as average height children.

The research, published in the NEJM, focused on the results of phase II clinical trials for the drug vosoritide, which was tested on 35 children with the condition, aged between five to 14 years, who received varying doses of the drug.

Those that received the highest dose of the drug grew at a similar rate to average height children of the same age, a result which was sustained over three and a half years. On average, participants in the trial grew 50% faster than their baseline rate with no adverse effects to body proportion.

Vosoritide interferes with proteins that block bone growth, allowing an average annual growth rate boost in height of about 6cm, which is close to growth rates for children of average stature.

The phase two trial ran over four years across research centres in Australia, France, Britain and the United States, and was aimed at evaluating the safety of the drug and determining optimal dosage.

“This study is the very first report of a possible disruptive, precision therapy for children with achondroplasia, which we hope will improve their health outcomes and functioning, as well as increase their height and access to their environment,” lead author and Murdoch Children’s Research Institute clinical geneticist Professor Ravi Savarirayan said.