regulatory
confidence high
sentiment positive
materiality 0.55
Monopar receives FDA Rare Pediatric Disease designation for ALXN1840 in Wilson disease
Monopar Therapeutics
- FDA granted Rare Pediatric Disease (RPD) designation to ALXN1840 (tiomolibdate choline) for Wilson disease.
- RPD designation is for therapies treating serious/life-threatening diseases primarily affecting children under 18.
- Upon NDA approval, Monopar may receive a pediatric Priority Review Voucher (PRV).
- PRV can be used for priority review of a subsequent application or sold/transferred to another sponsor.
- CEO Chandler Robinson noted the designation underscores the serious impact of Wilson disease.