MC-1 for the Treatment of PNPO Deficiency

Pyridox (am) ine 5'-phosphate oxidase (PNPO) deficiency is an inborn error of vitamin B6 metabolism, which causes an epileptic encephalopathy. This deficiency is caused by mutations in the PNPO gene encoding this enzyme. Intractable neonatal epileptic encephalopathy within the neonatal period is the classical presentation of PNPO deficiency. Intractable neonatal epileptic encephalopathy is characterized by the onset, shortly after birth, of drug-resistant seizures associated with severe neurological dysfunction.

Medicure Eliglble for Priority Review of MC-1 for PNPO Defiency by FDA

MC-1 has received both Rare Pediatric Disease Designation and Orphan Drug Designation from the FDA for the treatment of seizures associated with PNPO deficiency. Additionally, the European Medicines Agency ("EMA") has granted an Orphan Drug Designation to MC-1 for the treatment of PNPO deficiency. Medicure also received fast track designation for use of MC-1 for the treatment of PNPO deficiency, which will facilitate the review of Medicure's New Drug Application ("NDA") by the FDA.

If MC-1 is approved, Medicure may be eligible for a Priority Review Voucher (PRV) a highly valuable asset that can accelerate the review of a future therapy or be sold to support Medicure's continued growth and rare-disease research efforts.

About MEND-PNPO Clinical Trial

Medicure is conducting a Phase 3, prospective, open-label, multi-center study to assess the safety and efficacy of MC-1 in patients with confirmed PNPO deficiency via genetic analysis (MEND-PNPO). For more information about this study, please visit clinicaltrials.gov

View our Expanded Access Policy for MC-1

Contact Information:

Laura Cole, PhD
Project Manager
Telephone: 204-487-7412
Email: lcole@medicure.com