BridgeBio Pharma’s infigratinib produced significantly greater improvements than placebo in a Phase 3 trial in children with achondroplasia, according to data published in the New England Journal of Medicine. The study met its primary endpoint, showing a larger increase in annualized height velocity at week 52. In the randomized trial, 114 children were assigned in a 2:1 ratio to infigratinib or placebo. The least-squares mean change favored infigratinib by 1.74 cm per year for height velocity (P<0.001) and also improved height z score (P<0.001), while the upper-to-lower body segment ratio showed no significant difference. Adverse events were common in both groups, and investigators reported no serious adverse events or adverse events leading to discontinuation as related to either treatment arm.
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