FDA Grants Rare Pediatric Disease Designation to AmideBio’s Treatment for Congenital Hyperinsulinism

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to AmideBio’s glucagon analog (AB-G023) for the treatment of congenital hyperinsulinism.

Congenital hyperinsulinism (CHI) is a rare disease that affects newborns and children and results in persistent hypoglycemia that can lead to serious neurological complications including seizures and brain damage. It is caused by a defect in the pancreas that results in the over production of insulin. Approximately one in every 50,000 new births are diagnosed with CHI each year. Existing pharmaceutical treatments are less than satisfactory, often necessitating the surgical intervention of partial or full pancreatectomies, the latter resulting in the patient developing Type 1 diabetes.

AmideBio’s experimental therapy AB-G023 is a solution stable, soluble glucagon analog designed to overcome the limitations of glucagon, which while an effective treatment for CHI, is rendered impractical for long term administration given its instability in solution.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes AB-G023 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. There are no approved therapies for the condition.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

“This rare pediatric disease designation for AB-G023 shows continued recognition by the FDA of the importance of identifying an improved treatment for CHI patients and their parents,” said Pawel Fludzinski, CEO and president of AmideBio. “AmideBio’s AB-G023 has the potential to be more readily administered, including for use in pumps.”  

The development of AB-G023 was funded through Small Business Innovation Research (SBIR) grants from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Division of the National Institutes of Health (NIH).  It was granted Orphan Drug designation in April 2020.

Photo: Pawel Fludzinski, CEO and president of AmideBio

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.