Monopar Therapeutics Receives FDA Rare Pediatric Disease Designation for ALXN1840 for the Treatment of Wilson Disease
The FDA granted Rare Pediatric Disease designation to ALXN1840 for the treatment of Wilson disease, providing Monopar with the potential to receive a Priority Review Voucher upon future NDA approval. ALXN1840 is a late-stage candidate that previously met its primary endpoint in a Phase 3 pivotal trial, demonstrating significant copper mobilization compared to standard of care over 48 weeks.
Key figures
- Drug Name
- ALXN1840 (tiomolibdate choline, TMC)
- Phase Of Trial
- Phase 3
- Patient count
- 266
- Patient years
- 645
- Incidence rate
- 1 in 30,000
- Trial duration weeks
- 48
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