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hrp0098fc15.4 | Late Breaking | ESPE2024

Oral infigratinib for children with achondroplasia: Month 18 results from the PROPEL 2 study demonstrate safety and durability of treatment effect on linear growth with improved body proportionality

Savarirayan Ravi , Maria De Bergua Josep , Arundel Paul , Pierre Salles Jean , Saraff Vrinda , Delgado Borja , Leiva-Gea Antonio , McDevitt Helen , Nicolino Marc , Rossi Massimiliano , Salcedo Maria , Cormier-Daire Valerie , Skae Mars , Kannu Peter , Phillips III John , Saal Howard , Harmatz Paul , Candler Toby , Muslimova Elena , Weng Richard , Bai Yun , Raj Supriya , Hoover-Fong Julie , Irving Melita , Rogoff Daniela

Objectives: Achondroplasia (ACH) is characterized by disproportionate short stature and is associated with medical complications and functional constraints. Infigratinib is an oral FGFR3 inhibitor that targets overactive FGFR3 signaling at its origin and is under development as a treatment option for children with ACH. We report Month 18 results from PROPEL 2 (NCT04265651), a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluat...

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Oral infigratinib for children with achondroplasia: Month 18 results from the PROPEL 2 study demonstrate safety and durability of treatment effect on linear growth with improved body proportionality

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