A new daily tablet increases growth in children with dwarfism, study finds

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 adverse reactions. Achondroplasia, a genetic condition affecting one in 20,000 babies in Australia, leads to shorter stature and can cause severe complications such as spinal cord compression, sleep apnoea, and bowed legs.

Children with this condition face a significantly higher risk of mortality before age five. The MCRI team has now begun a phase III trial and plans to explore the drug’s potential for even younger children aged 0-3. For families dealing with achondroplasia, this new oral therapy offers hope for a more convenient and effective solution to improve growth and overall well-being, the researchers said.

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