A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

Summary

This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of participation in the QED-sponsored study PROPEL (QBGJ398-001).

Timeline

Start

2023-11-10

Primary completion

2025-12-18

Completion

2025-12-18

Outcome

Met primary endpoint

paper treatment with once-daily oral infigratinib for 52 weeks resulted in a significantly greater increase from baseline in the annualized height velocity than placebo. PMID 42370681 ↗

release “Change from baseline in AHV was superior to placebo at Week 52 with a mean treatment difference against placebo of +2.10 cm/year” bridgebio.com ↗

Publications

• Savarirayan R, Hoover-Fong J, Irving M, Arundel P, de Bergua JM, Campeau PM, Candler T, Cocanougher BT, Cormier-Daire V, Edouard T, Fredwall SO, Harmatz P, Hoernschemeyer D, Irgens HU, Jamuar S, Kannu P, Legare JM, Leiva-Gea A, McDevitt H, Onesimo R, Phillips J 3rd, Del Pino M, Robinson ME, Rossi M, Skae M, Ward LM, White KK, Schmidt J, Lystig T, Salvatici A, Bai Y, Butler PW, van Veenhuyzen D, Rogoff D. Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia. N Engl J Med. 2026 Jun 28. doi: 10.1056/NEJMoa2604565. Online ahead of print.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Infigratinib	Small molecule	0.25 mg/kg	Oral
Subject	Infigratinib	Small molecule	2 mg	Oral
Subject	Infigratinib	Small molecule	3.5 mg	Oral
Subject	Infigratinib	Small molecule	5 mg	Oral
Subject	Infigratinib	Small molecule	7 mg	Oral
Subject	Infigratinib	Small molecule	10 mg	Oral

Indications