FDA Approves Congenital Hyperinsulinism Treatment

April 23, 2020
Kenny Walter

Kenny Walter is an editor with HCPLive. Prior to joining MJH Life Sciences in 2019, he worked as a digital reporter covering nanotechnology, life sciences, material science and more with R&D Magazine. He graduated with a degree in journalism from Temple University in 2008 and began his career as a local reporter for a chain of weekly newspapers based on the Jersey shore. When not working, he enjoys going to the beach and enjoying the shore in the summer and watching North Carolina Tar Heel basketball in the winter.

ABG-023 overcomes the limitations glucagon because it remains stable in solutions.

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.


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