Failed cancer drug finds success in phase III trial as achondroplasia treatment
Professor Ravi Savarirayan says more experimental cancer drugs are becoming treatments for rare diseases.
A cancer medicine is likely to arrive on the market next year — not for oncology, but for the most common form of dwarfism.
An Australian-led phase III study, published in The New England Journal of Medicine last month, paves the way for infigratinib, a fibroblast growth factor receptor 1-3 tyrosine kinase inhibitor, to become the first oral treatment to spur growth in children with achondroplasia.
Of 74 study participants aged 3-17 with achondroplasia, growth velocity increased by 1.58cm/year with 52 weeks on infigratinib. Meanwhile, velocity in a control group of 39 patients slowed by 0.16cm/year.
Height Z-score also improved with treatment: +0.32 SD higher than placebo.
