Monopar Therapeutics received FDA Rare Pediatric Disease designation for ALXN1840, potentially enabling a pediatric Priority Review Voucher upon NDA approval.

Key Highlights:

  • FDA granted Rare Pediatric Disease designation to ALXN1840 (tiomolibdate choline) for Wilson disease.
  • Designation could make the company eligible for a pediatric Priority Review Voucher if ALXN1840's NDA is approved.
  • Priority review can shorten FDA review time by several months, accelerating potential market access.
  • ALXN1840 is a once-daily oral therapy that in Phase 3 showed sustained copper mobilization vs standard of care.

Original SEC Filing:

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