A new study reveals that a promising daily tablet can significantly boost height and enhance proportionate limb development in children with achondroplasia, the most prevalent type of dwarfism. This breakthrough may eliminate the need for a daily injection previously required to promote growth.
This second-phase study, conducted by the Murdoch Children’s Research Institute (MCRI) and published in the New England Journal of Medicine, found that the drug infigratinib, which is still under investigation, is both safe and effective for treating children with achondroplasia between the ages of 3 and 11.
Professor Ravi Savarirayan from MCRI stated that infigratinib not only accelerates bone growth but also enhances these children’s quality of life.
The research, funded by BridgeBio Pharma Inc, included 72 children with achondroplasia from Australia, the UK, the US, Spain, France, and Canada. It indicated that the medication could increase growth rates by 2.5cm a year over the course of 18 months, leading to taller heights and a better upper-to-lower-body segment ratio. Most participants experienced mild side effects, with no serious adverse reactions reported.
In Australia, the only approved treatment for children with achondroplasia is vosoritide, which is administered through a daily injection and was added to the PBS last year. MCRI holds the title of the largest vosoritide clinical trial site globally. Professor Savarirayan and his team have previously demonstrated that the drug aids bone growth in patients as young as four months old up to those aged 18.
“Our latest research shows that infigratinib is a safe and effective medication that enhances growth in children with achondroplasia and could fulfill a significant need for an oral treatment for these patients,” said Professor Savarirayan. This is particularly vital for children who cannot tolerate daily injections and in regions where oral medications are more feasible than injections.”
Achondroplasia is the most common form of skeletal dysplasia, affecting approximately one in 20,000 newborns in Australia. This genetic disorder can lead to serious health issues like spinal cord compression, sleep apnea, bowed legs, narrowing of the spinal canal, and recurring ear infections. Children with achondroplasia have a 50 times greater risk of dying before age five compared to their peers.
Professor Savarirayan noted that a phase III trial has commenced, and plans for further studies on children aged 0-3 years with achondroplasia are in progress.
