FDA grants rare disease drug status to AmideBio’s pediatric hyperinsulinism treatment

BOULDER — The U.S. Food and Drug Administration granted a rare pediatric disease designation to diabetes drug developer AmideBio LLC.

The Boulder-based AmideBio received the designation for its AB-G023 drug candidate, a genetic treatment for hyperinsulinism in children. With the designation, the company would receive a voucher for priority review of a different drug if AB-G023 is approved for sale, which the company could use for another drug in its development pipeline or sell to a different firm.

Hyperinsulinism occurs when a patient’s pancreas overproduces insulin and causes low blood sugar, or the opposite effect of diabetes. The condition affects only one out of every 25,000 to 50,000 newborns and can cause seizures, brain damage and death if left untreated, as the brain primarily uses sugars to fire neurons.

While researchers have long used the compound glucagon as a treatment for hyperinsulinism, the compound has a short lifespan when kept in a solution before being administered to a patient. AmideBio’s drug is thought to be more stable while on the shelf, making treatment easier over a longer period of time.

“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 CEO Pawel Fludzinski in a statement.

AmideBio is preparing to file an investigational new drug application to drug regulators, which is required before beginning Phase I clinical trials on humans.

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