Achondroplasia -- the most common form of dwarfism -- is caused by pathogenic gain-of-function variants in the gene encoding fibroblast growth factor receptor 3.Treatment with the oral tyrosine kinase inhibitor infigratinib led to significant increases in growth among children with achondroplasia in the phase III PROPEL 3 study.The once-daily capsule targets FGFR1-3 and provides an alternative to injectable treatments.
Treatment with a once-daily FGFR1-3 tyrosine kinase inhibitor led to significant increases in growth among children with achondroplasia, the phase III PROPEL 3 study showed.
The least-squares mean change from baseline in the annualized height velocity at week 52 was 1.58 cm/year in the infigratinib group and -0.16 cm/year in the placebo group, for a between-group difference of 1.74 cm/year (95% CI 1.31-2.17), reported Ravi Savarirayan, MBBS, MD, of Royal Children's Hospital in Parkville, Australia, and colleagues.
In addition, the difference between infigratinib and placebo in the least-squares mean change from baseline to week 52 in the achondroplasia-specific height z score was 0.32 (96% CI 0.23-0.41). There was also a trend toward a decrease in the upper-to-lower body segment ratio, but this did not reach significance (least-squares mean change -0.02, 95% CI -0.06 to 0.01).
