Home/Pipeline/MC-1 (Pyridoxal 5'-Phosphate)

MC-1 (Pyridoxal 5'-Phosphate)

Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) Deficiency

Phase 3Active, EnrollingNCT04706013

Key Facts

Indication
Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) Deficiency
Phase
Phase 3
Status
Active, Enrolling
Company

About Medicure

Medicure is a U.S.-focused, publicly traded pharmaceutical company with a dual-pillar strategy: commercializing established cardiovascular products and developing novel therapies for rare diseases. Its key achievement is building an integrated commercial model, combining a traditional sales force with the nationwide, direct-to-patient Marley Drug pharmacy platform. The company's near-term value driver is the Phase 3 MEND-PNPO trial for MC-1 in PNPO deficiency, a program with Rare Pediatric Disease, Orphan Drug, and Fast Track designations that could yield a valuable Priority Review Voucher upon approval.

View full company profile