Listen to Story A new daily tablet has shown promise in boosting growth and improving limb proportions in children with achondroplasia, the most common form of dwarfism, according to a study. The research, published in the New England Journal of Medicine and led by the Murdoch Children’s Research Institute (MCRI), offers a potential alternative to daily injections currently used to treat the condition. The phase II study evaluated the investigational drug infigratinib in 72 children aged 3-11 from six countries, including Australia, the UK, and the US.
Over an 18-month treatment period, the children’s growth rate increased by an average of 2.5 cm per year. The drug also improved the proportionality of their upper and lower body segments .
Professor Ravi Savarirayan from MCRI highlighted the significance of the findings: "Infigratinib not only enhances bone growth but also improves the quality of life for children with achondroplasia. It offers a much-needed oral alternative for those unable to tolerate injections and in regions where oral medication is more feasible." Currently, the only approved treatment in Australia is vosoritide, a daily injection added to the Pharmaceutical Benefits Scheme last year.
While highly effective, the injections can be challenging for some children and families . Infigratinib, developed in partnership with BridgeBio Pharma Inc, was found to be safe during the trial, with participants experiencing only mild side effects and no serious advers.