Monopar Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to ALXN1840 (tiomolibdate choline, TMC). ALXN1840 is Monopar's late-stage candidate for the treatment of Wilson disease. The RPD designation is granted to therapies intended for serious or life-threatening diseases primarily affecting children aged 0-18. This designation provides Monopar with the potential to receive a pediatric Priority Review Voucher (PRV) upon NDA approval, which can expedite the review process or be sold. Wilson disease is a rare genetic disorder affecting approximately 1 in 30,000 people worldwide, caused by mutations in the ATP7B gene leading to toxic copper accumulation. ALXN1840 is a novel first-in-class albumin tripartite complex (ATC) activator that mobilizes and sequesters excess copper. Clinical data from a Phase 3 trial showed ALXN1840 met its primary endpoint, demonstrating rapid and sustained copper mobilization significantly greater than standard of care over 48 weeks. The drug showed durable clinical improvement and a favorable safety profile across 645 patient-years of follow-up in 266 patients. ALXN1840 is an oral tablet with once-a-day dosing. Monopar also has radiopharmaceutical programs for imaging and treating advanced cancers.