Monopar's ALXN1840 Receives FDA Rare Pediatric Disease Designation

Monopar Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to ALXN1840, its late-stage candidate for the treatment of Wilson disease. This designation provides the company with the potential to receive a pediatric Priority Review Voucher (PRV) upon NDA approval, which can be used to obtain priority review for a subsequent marketing application or be sold. ALXN1840, a novel first-in-class treatment, previously met its primary endpoint in a Phase 3 pivotal trial, demonstrating rapid and sustained copper mobilization.