05/11/2026
Ottawa, Ontario — Thursday November 5, 2026
Current targeted treatments for children with achondroplasia, a genetic condition that limits bone growth, require regular injections. Researchers wanted to know whether a once-daily oral medicine could offer children and families another option.
A major international Phase 3 trial tested infigratinib, an oral medicine designed to reduce the overactive signals that limit bone growth in achondroplasia.
“For families, having an oral therapy to help them grow could make a meaningful difference. This trial shows that a once-daily oral medicine can increase growth over one year, and longer-term research will help us understand what it may offer for the health and daily lives of children with achondroplasia.”– Dr. Marie-Eve Robinson
Marie-Eve Robinson with support from Joanna Lazier and Karine Khatchadourian led the CHEO study site, one of the trial’s largest recruiting sites. The trial included 114 children aged three to 17 across 27 sites in 10 countries.
After one year, children taking infigratinib grew an average of 1.74 centimetres more per year than those receiving a placebo. Most side effects were mild or moderate, and investigators did not link any serious side effects to the treatment.
The findings bring families one step closer to an oral treatment option for achondroplasia. Longer-term studies will examine whether the increase in growth continues and assess safety, mobility, quality of life, and other outcomes that matter to children and families.