Oral Infigratinib Seems Safe for Children With Achondroplasia

MONDAY, Nov. 18, 2024 (HealthDay News) -- For children with achondroplasia, oral infigratinib does not result in major safety signals and yields increased annualized height velocity and z score at a dose of 125 mg, according to a study published online Nov. 18 in the New England Journal of Medicine.

Ravi Savarirayan, M.B., B.S., M.D., from Murdoch Children's Research Institute in Melbourne, Australia, and colleagues examined the safety and efficacy of oral infigratinib in children with achondroplasia between the ages of 3 and 11 years in a phase 2 dose-finding study. Seventy-two children were enrolled in five sequential cohorts to receive infigratinib daily at doses of 0.016, 0.032, 0.064, 0.128, and 0.25 mg per kg body weight for six months (cohorts 1, 2, 3, 4, and 5, respectively), followed by 12 months of extended treatment.

The researchers found that all children had at least one adverse event during treatment, most of which were mild or moderate in severity; there was no resultant treatment discontinuation. An increased annualized height velocity was observed in cohort 5 (0.25 mg/kg); this persisted throughout the duration of the study, with a mean change from baseline of 2.50 cm per year at 18 months. Relative to an untreated achondroplasia reference population, the mean change from baseline in height z score was 0.54 at 18 months; in the upper-to-lower body segment ratio, the mean change from baseline was −0.12.

"Additional treatment duration will be required to determine whether the improvement in the upper-to-lower body segment ratio observed in these children at 18 months will continue and be associated with improvements in functionality," the authors write.

The study was funded by BridgeBio Pharma, which is developing infigratinib.

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