hrp0098p1-309 | Late Breaking 2 | ESPE2024

Safety and efficacy of efpegerglucagon in patients with congenital hyperinsulinism: interim results from a phase 2 study

Dastamani Antonia , Banerjee Indraneel , Mohnike Klaus , Puri Mala , Gwak EunJi , Kang Seohyun , Byeon JinHee , Hee Lee Moon , De Leon Diva

Introduction: Congenital hyperinsulinism (CHI) is a rare disease affecting 1 in 25,000 to 50,000 newborns that requires emergent treatment of recurrent hypoglycemia to prevent neurological sequelae. There is a high unmet need for novel treatments due to the limited effectiveness and adverse effects associated with the current standard of care medications, namely diazoxide and somatostatin analogues.Methods: Efpegerglucag...