Infigratinib Shows Promise as Oral Treatment for Achondroplasia in Children
核心洞察
A Phase II study reveals that infigratinib, an oral medication, significantly increases height in children with achondroplasia (搜索), the most common form of dwarfism.
The study demonstrated that infigratinib boosts bone growth and enhances the quality of life for children aged 3-11 years with achondroplasia (搜索).
Infigratinib increased the rate of growth by 2.5 cm per year over 18 months, improving overall height and body segment ratio, with only minor side effects.
A daily oral tablet, infigratinib, is showing promise in treating achondroplasia (搜索) in children, potentially increasing height and improving proportional limb growth. The Phase II study, published in the New England Journal of Medicine, indicates that infigratinib could provide a safe and effective alternative to daily injections for children with this condition.
The multi-center trial, led by Murdoch Children's Research Institute (MCRI), involved 72 children aged 3-11 years with achondroplasia (搜索) from Australia, the UK, the US, Spain, France, and Canada. The study, sponsored by BridgeBio Pharma Inc (搜索), found that infigratinib increased the rate of growth by 2.5 cm per year over 18 months. This increase in growth led to improved overall height and a better upper-to-lower-body segment ratio among participants. While some participants experienced minor side effects, no serious adverse reactions were reported.
Impact on Growth and Quality of Life
MCRI Professor Ravi Savarirayan emphasized that infigratinib not only promotes bone growth but also enhances the quality of life for children with achondroplasia (搜索). "Our new study found that infigratinib is a safe and effective drug that increases growth in children with achondroplasia and could meet a need for an oral medication for those with this condition. This is especially important for those children who can't tolerate daily injections and in parts of the world where oral medications are more practicable than injections."
Current Treatment Landscape
Currently, the only approved therapy for children with achondroplasia (搜索) in Australia is vosoritide, administered via daily injection. Vosoritide was added to the Pharmaceutical Benefits Scheme (PBS) last year. MCRI is the largest vosoritide clinical trial site globally. Professor Savarirayan and his team have previously demonstrated that vosoritide improves bone growth development in patients as young as four months up to 18 years of age.
Achondroplasia: Prevalence and Complications
Achondroplasia (搜索), the most common form of dwarfism, affects approximately one in 20,000 babies in Australia. This genetic condition can lead to severe medical complications, including spinal cord compression, sleep apnea, bowed legs, narrowing of the spinal canal, and recurrent ear infections. Children with achondroplasia face a significantly higher mortality risk, being 50 times more likely to die before the age of five compared to their peers.
Future Research
Professor Savarirayan mentioned that a Phase III trial has commenced, and plans are underway for further studies involving children aged 0-3 years with achondroplasia (搜索).