GlobeNewswire·Jun 30, 2026, 8:00 AM ET

Monopar Therapeutics Receives FDA Rare Pediatric Disease Designation for ALXN1840 for the Treatment of Wilson Disease

Monopar Therapeutics Inc. announced that the FDA granted Rare Pediatric Disease (RPD) designation to ALXN1840 (tiomolibdate choline, TMC) for treating Wilson disease.
The RPD designation offers the potential for a pediatric Priority Review Voucher (PRV) upon NDA approval, which can reduce FDA review time by several months or be sold.
Wilson disease is a rare genetic disorder affecting 1 in 30,000 people caused by ATP7B gene mutations, leading to toxic copper accumulation.
ALXN1840 is a first-in-class albumin tripartite complex activator that mobilizes excess copper, limits oxidative damage, and blocks blood-brain barrier transport.
Phase 3 trial results showed ALXN1840 met its primary endpoint with rapid, sustained copper mobilization superior to standard of care over 48 weeks.
The drug demonstrated durable clinical improvement across 645 patient-years of follow-up involving 266 patients.
ALXN1840 is an oral tablet administered via a once-a-day dosing regimen.
Monopar Therapeutics also maintains radiopharmaceutical programs including MNPR-101-Zr, MNPR-101-Lu, & MNPR-101-Ac for advanced cancers.