A landmark Phase 3 trial has demonstrated that infigratinib, a selective fibroblast growth factor receptor inhibitor, represents a significant advancement in treating achondroplasia, the most common genetic form of skeletal dysplasia. The international trial, published in the New England Journal of Medicine, enrolled 132 children aged 2–11 years and tracked outcomes over 24 months of treatment across North America and Europe.
The oral therapy increased annualised growth velocity by 50–60% compared to natural disease progression, with treated children achieving 8.5–9.2 cm annual growth versus the expected 5–6 cm without intervention. Beyond growth gains, participants demonstrated functional improvements in mobility and daily activities, with a manageable safety profile supporting the drug’s potential clinical utility. Regulatory decisions are anticipated by mid-2025, pending completion of long-term safety data collection through age 18.
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