FDA Approves Congenital Hyperinsulinism Treatment

The US Food and Drug Administration (FDA) has granted an orphan drug designation for ABG-023, a solution stable, soluble glucagon analog designed to treat congenital hyperinsulinism (CHI).

The new treatment, developed by AmideBio, overcomes the limitations of glucagon, a proven effective treatment CHI that is rendered impractical for long-term administration because it is instable in solution.

CHI is a rare genetic disease affecting newborns and children, resulting in persistent hypoglycemia, which can lead to serious neurological complications such as seizures and brain damage. The disease causes a defect in the pancreas that can result in the patient having severe hypoglycemia because of the over production of insulin.

CHI impacts approximately 1 in every 50,000 new births annually.

The existing treatment options often necessitate the surgical intervention of partial or full pancreatectomies, which can result in the patient developing type 1 diabetes.

"This orphan drug designation is an important step for AmideBio's development of ABG-023 and for CHI patients and their parents, Pawel Fludzinski, CEO and President of AmideBio, said in a statement. “Glucagon has been shown to be a potentially game-changing treatment for these patients if it can be more readily administered, including for use in pumps. Our initial studies of ABG-023 have shown it to have great promise in overcoming the shortfalls of glucagon without sacrificing efficacy.”

AmideBio currently has a pipeline of novel biotherapeutics targeting metabolic disease that is generated by leveraging core competencies in intelligent design of drug candidates together with BioPure technology to deliver high purity and difficult-to-manufacture peptides of any length with purity.