P181: A phase 2/2b study of infigratinib in children under 3 years old with achondroplasia: Design of PROPEL Infant and Toddler

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Authors: Julie Elizabeth Hoover-Fong, Ravi Savarirayan, Melita Irving, Helen McDevitt, Supriya Raj, Rui Santos, Mars S. Skae, Klane K. White, Yun Bai, Snow Ge, Micaela Rodine, Karla Mehl
+1 more Daniela Rogoff
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Abstract

Infigratinib is an oral FGFR1-3 inhibitor that is in development for achondroplasia (ACH). In the PROPEL 2 study, treatment with daily infigratinib at a dose of 0.25 mg/kg was well tolerated and resulted in sustained increases in annualized height velocity and improved upper-to-lower body segment ratio in children aged 3 to 11 years old with ACH. These findings provided clinical proof-of-concept to proceed with assessing the safety, pharmacokinetics, and efficacy of infigratinib in children under 3 years old with ACH.

Fields of Study & Subjects

Connective tissue disorders research Growth Hormone and Insulin-like Growth Factors Craniofacial Disorders and Treatments

Publication details

Journal / source
Genetics in Medicine Open
Volume
4
Pages
103675–103675
Publication date
1 January 2026
Language
EN