Congenital hyperinsulinism (CHI) is an ultra-rare pediatric genetic disorder characterized by excessive production of insulin by the pancreas. If untreated the elevated insulin levels in these patients can induce extreme hypoglycemia (low blood sugar) events, increasing the risk of neurological and developmental complications, including persistent feeding problems, learning disabilities, recurrent seizures, brain damage or even death.
There are no approved therapies for CHI and current standard of care treatments are suboptimal. In some cases, pancreatic surgery is a treatment option, but this approach is invasive and may require repeat surgeries.
RZ358 is designed to increase glucose in the bloodstream of CHI patients by reducing binding and signaling of insulin at its receptor. RZ358 is currently in Phase 2b development (the RIZE study, RZ358-606). For more information on the clinical trial please visit ClinicalTrials.gov.
RZ358 has received orphan drug designation in the United States and European Union.