A new daily tablet has shown promise in promoting growth and improving limb proportions in children suffering from 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 the daily injections currently used to treat the condition.

The Phase II study evaluated the investigational drug infigratinib in 72 children aged 3-11 years from six countries, including Australia, the UK and the US.

Over the 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 his upper and lower body segments.

MCRI Professor Ravi Savarirayan highlighted the significance of the findings: “Infigratinib not only enhances bone growth, but also improves the quality of life for children with achondroplasia. It provides a much-needed oral option for those Those who are unable to tolerate injections and in areas 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. Although highly effective, 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 adverse reactions.

Achondroplasia, a genetic condition affecting one in 20,000 babies in Australia, causes short stature and can cause serious complications such as spinal cord compression, sleep apnea and bowed legs. Children with this condition have a significantly higher risk of death before the age of five.

The MCRI team has now started Phase 3 trials and plans to explore the drug’s potential for younger children aged 0-3 years as well.

For families struggling with achondroplasia, this new oral therapy offers hope for a more convenient and effective solution to improving growth and overall well-being, the researchers said.