TOKYO, Jun 03 (Pulse News Wire) – RIBOMIC Inc. (4591.T) announced today that its Phase III clinical trial for umedaptanib pegol, a treatment for achondroplasia in pediatric patients aged two to 14, received Institutional Review Board (IRB) approval at one of the participating medical institutions.
The trial, which was approved by PMDA in April 2026, aims to verify the efficacy, safety, and pharmacokinetics of umedaptanib pegol through weekly subcutaneous injections of 1 mg/kg over 78 weeks. The primary endpoint of the study is to evaluate changes in annualized height velocity (AHV) compared to pre-treatment observation periods. The trial plans to enroll 16 patients across multiple medical facilities.
RIBOMIC expects to initiate patient enrollment shortly following further IRB approvals at additional sites. Umedaptanib pegol, designated as RBM-007, is an aptamer that strongly inhibits fibroblast growth factor 2 (FGF2). It targets the underlying mechanism of achondroplasia by blocking excessive FGF signaling, which hinders normal bone development.
The drug has been recognized as an orphan medicinal product by the Ministry of Health, Labour and Welfare due to the rarity of the condition, affecting approximately one in 25,000 newborns.
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