FDA Grants Breakthrough Therapy Designation to Eiger’s Avexitide for Congenital Hyperinsulinism

The U.S. Food and Drug Administration granted Eiger BioPharmaceuticals Breakthrough Therapy Designation for avexitide, its experimental therapy for the treatment of congenital hyperinsulism.

Photo: Colleen Craig, vice president of metabolic diseases at Eiger

Congenital hyperinsulinism (HI) is a rare, genetic, pediatric metabolic disorder characterized by severe fasting and protein-induced hypoglycemia due to dysregulated secretion of insulin by the pancreas. Repeat episodes and/or dangerously low blood sugars increase the risk of neurological and developmental complications, including persistent feeding problems, learning disabilities, recurrent seizures, brain damage, or even death. Existing medical options are often ineffective or are associated with substantial side effects that discourage compliance and lead to suboptimal treatment outcomes. Subtotal pancreatectomy is an option, but this approach can be associated with life-threatening complications, does not immediately fully resolve hypoglycemia in most patients, and ultimately leads to the development of lifelong insulin-dependent diabetes.

Avexitide is a 31-amino acid peptide that selectively targets and blocks GLP-1 receptors, reducing dysregulated insulin secretion by the pancreas, and thereby reducing fasting and postprandial hypoglycemia. Proof of concept has been demonstrated in 39 patients across three phase 2 studies in neonates, children, and adolescents with congenital hyperinsulinism. Avexitide has been granted Breakthrough Therapy designation by the FDA for the treatment of congenital hyperinsulism, Orphan Drug designation by the FDA for the treatment of hyperinsulinemic hypoglycemia (which includes congenital hyperinsulinism), Orphan Drug designation by the European Medicines Agency for the treatment of congenital hyperinsulinism and Rare Pediatric Disease designation by the FDA.

Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on clinically significant endpoints.

Eiger’s application was supported by data from three completed phase 2 studies in 39 neonates, children, and adolescents with congenital hyperinsulinism.

Avexitide is also in development for post-bariatric hypoglycemia (PBH). Four clinical studies have been completed in 54 patients with PBH. Eiger has received concurrence from FDA and EMA on a single phase 3 study for patients with PBH. Avexitide has been granted Breakthrough Therapy designation by the FDA, as well as Orphan Drug designation by the FDA for the treatment of hyperinsulinemic hypoglycemia and Orphan Drug designation by the EMA for the treatment of non-insulinoma pancreatogenous hypoglycemia syndrome (NIPHS). Both of these broad orphan designations include PBH.

“Avexitide represents a promising, targeted approach for the treatment of congenital hyperinsulinism, an urgent, unmet medical need with no approved therapy,” said Colleen Craig, vice president of metabolic diseases at Eiger.

Author: Rare Daily Staff