Oral infigratinib shows more growth in achondroplasia phase 3 trial

A dose taken by mouth once a day is at the center of a phase 3 trial for children with achondroplasia. After 52 weeks, those who received oral infigratinib showed a significantly greater increase in growth from baseline than children given placebo, according to results published in the New England Journal of Medicine.

Achondroplasia is a skeletal condition marked by disproportionate short stature and can bring medical, functional and psychosocial challenges. Treatments typically require daily or weekly injections, while medication is most effective when started early—an administration burden that an oral option could potentially reduce.

The trial included 114 children ages 3–17: 75 received infigratinib and 39 received placebo. The comparison found a greater increase in growth for the infigratinib group over the 52-week period.

For families, the concrete potential is a simpler treatment routine. A once-daily medicine taken by mouth could make treatment easier to administer than daily or weekly injections, particularly when early treatment is considered. Salusky’s argument that oral dosing may suit infants is an editorial interpretation; the trial participants described were ages 3–17.

The result remains a clinical-trial finding, not evidence that the treatment is already in routine use. Salusky and Jueppner also emphasized international collaboration in research on rare genetic bone disorders.