A Phase 3 randomised controlled trial has quantified the growth-promoting efficacy of infigratinib in paediatric achondroplasia, demonstrating that oral treatment produces measurable, clinically significant improvements in linear growth. Children receiving infigratinib achieved annualised growth velocity of 8.5–9.2 cm per year, compared with the expected 5–6 cm per year observed in natural disease progression—representing a 50–60% relative improvement over two years of active therapy.
This magnitude of growth acceleration in a genetic disorder characterised by severely restricted skeletal growth represents a substantial clinical advance. The multi-centre trial, which enrolled 132 genetically confirmed achondroplasia cases across international sites, employed weight-adjusted dosing protocols and demonstrated adverse event profiles consistent with selective FGFR inhibition. These quantitative outcomes underscore the disease-modifying potential of infigratinib and support ongoing regulatory review processes expected to conclude by 2025.
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