A daily tablet could replace painful injections for the treatment of dwarfism.

A new drug has shown potential to improve growth and limb proportions in children with achondroplasia, the most common form of dwarfism. 

The treatment, infigratinib, offers a less invasive alternative to current daily injections, according to an international study led by the Murdoch Children’s Research Institute (MCRI).

A recent phase II trial involved 72 children aged 3 to 11 from six countries, including Australia. 

Researchers reported an annual growth increase of 2.5 centimetres on average, sustained over 18 months. Improvements in the proportionality of the upper and lower body were also observed.

Participants experienced mild side effects, with no serious adverse reactions. The findings suggest the oral treatment could improve not just height but also quality of life.

MCRI’s Professor Ravi Savarirayan says a tablet is particularly useful for children struggling with injections or living in areas where injections are less feasible. 

The only currently approved therapy in Australia, vosoritide, requires daily injections. It was added to the Pharmaceutical Benefits Scheme in 2022.

Achondroplasia occurs in one in 20,000 Australian births and is associated with disproportionate short stature and medical issues, such as spinal cord compression, sleep apnoea, and ear infections. Affected children face a significantly higher risk of early death, being 50 times more likely to die before age five than their peers.

Infigratinib works by targeting FGFR1–3 pathways, which influence bone growth. It has shown promise not only for growth improvements but also for addressing functional challenges tied to the condition. 

Building on these results, researchers are launching a phase III trial to further test infigratinib and exploring its use in children under three years old.

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