The findings of this study were published in the New England Journal of Medicine on November 18, 2024.

Participants reported only minor side effects from infigratinib, with no serious adverse reactions noted during the study.

Currently, the only approved therapy for achondroplasia in Australia is vosoritide, a daily injection that was added to the Pharmaceutical Benefits Scheme in 2023.

MCRI is the largest clinical trial site for vosoritide globally, and previous research has shown its effectiveness in promoting bone growth in younger patients.

A recent study led by the Murdoch Children's Research Institute (MCRI) has demonstrated that the oral tablet infigratinib effectively increases height and improves limb growth in children with achondroplasia, the most common form of dwarfism.

This Phase II clinical trial involved 72 children aged 3 to 11 from multiple countries, including Australia, the UK, the US, Spain, France, and Canada, and was funded by BridgeBio Pharma Inc.

Results indicated that infigratinib treatment led to an average growth increase of 2.5 cm per year for up to 18 months, significantly enhancing overall height and improving the upper-to-lower-body segment ratio.

Achondroplasia affects approximately one in 20,000 babies in Australia and is associated with serious health complications, including spinal cord compression and increased mortality risks in early childhood.

Professor Ravi Savarirayan emphasized the importance of having an oral option like infigratinib for children who cannot tolerate injections, especially in regions where oral medications are more feasible.

Following the promising results, MCRI is initiating a Phase III trial and planning further studies involving younger children aged 0 to 3 years with achondroplasia.