USFDAJun 30, 2026, 08:01 AM
Monopar Receives FDA Rare Pediatric Disease Designation for ALXN1840
AI Summary
Monopar Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease (RPD) designation to its late-stage candidate, ALXN1840 (tiomolibdate choline), 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 expedite the review of a future marketing application or be sold. The company's CEO highlighted the urgency of bringing forward new treatment options for Wilson disease, a serious genetic disorder.
Key Highlights
- FDA granted Rare Pediatric Disease (RPD) designation to ALXN1840 (tiomolibdate choline) for Wilson disease.
- RPD designation offers potential for a pediatric Priority Review Voucher (PRV) upon NDA approval.
- PRV can be used for priority review of a subsequent marketing application or sold/transferred.
- Priority review can reduce FDA's target review time by several months.
- ALXN1840 is a late-stage candidate for Wilson disease, a rare genetic disorder affecting copper excretion.
- ALXN1840 demonstrated rapid and sustained copper mobilization in a Phase 3 pivotal trial.
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