Eiger Announces FDA Breakthrough Therapy Designation for Avexitide for Treatment of Congenital Hyperinsulinism
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 a clinically significant endpoint(s).
"Avexitide represents a promising, targeted approach for the treatment of congenital hyperinsulinism, an urgent, unmet medical need with no approved therapy," said
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 EMA for the treatment of congenital hyperinsulinism and Rare Pediatric Disease Designation by the FDA.
Avexitide is also in development for post-bariatric hypoglycemia (PBH). Four clinical studies have been completed in 54 patients with PBH.
Avexitide is not approved or commercialized for any indication.
About Congenital Hyperinsulinism (HI)
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.
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