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A groundbreaking study has revealed that a daily tablet, infigratinib, is highly effective at increasing height and improving proportional limb growth in children with achondroplasia, the most common form of dwarfism. The findings from this phase II study offer a potential alternative to daily injections, a treatment currently required for growth in these children.
The study, led by the Murdoch Children’s Research Institute (MCRI) and published in the New England Journal of Medicine, involved 72 children with achondroplasia aged 3-11 years from Australia, the UK, the US, Spain, France, and Canada. It showed that infigratinib was not only safe but also led to significant improvements in height and bone growth, increasing growth by 2.5cm annually over a period of 18 months.
Professor Ravi Savarirayan of MCRI, one of the study’s lead researchers, emphasized that the medication not only promotes growth but also enhances the overall quality of life for children with this condition. “Infigratinib has shown promising results in increasing growth and improving limb proportions, which is vital for these children’s well-being and development,” said Savarirayan.
The trial, sponsored by BridgeBio Pharma Inc, reported that while participants experienced some minor side effects, none had serious adverse reactions. This makes infigratinib an exciting alternative to vosoritide, the only currently approved therapy for children with achondroplasia in Australia, which requires daily injections. Vosoritide, added to the Pharmaceutical Benefits Scheme (PBS) in Australia last year, has been shown to improve bone growth, but it may not be suitable for all patients, especially those unable to tolerate injections.
Professor Savarirayan and his team previously conducted extensive research into vosoritide, demonstrating its effectiveness in treating children as young as four months old, but the new study provides hope for those who could benefit from an oral medication. “Infigratinib could meet a significant need for an oral treatment for children with achondroplasia, especially in areas where injections are less practical or not well tolerated,” he explained.
Achondroplasia, a genetic condition affecting approximately one in 20,000 newborns in Australia, is a type of skeletal dysplasia. The condition can cause severe medical issues, including spinal cord compression, sleep apnea, bowed legs, recurrent ear infections, and narrowing of the spinal canal. Tragically, children with achondroplasia are at a significantly higher risk of early death, being 50 times more likely to die before the age of five than their peers.
In response to the promising results, MCRI has already initiated a phase III trial and is also planning further studies, including research into the effects of infigratinib in children as young as 0-3 years.
As the search for new treatments continues, this study marks a major step forward in offering more accessible and effective care for children living with achondroplasia, potentially improving both their physical development and their overall quality of life.
