MC-1

MC-1 is an investigational drug that contains pyridoxal 5'-phosphate monohydrate (PLP), a naturally occurring metabolite of pyridoxine (vitamin B6). Medicure Pharma is currently evaluating the safety and efficacy of MC-1 to treat patients with pyridox (am) ine 5'-phosphate oxidase (PNPO) deficiency.

Pyridoxal 5'-Phosphate-Dependent Epilepsy

Pyridox (am) ine 5'-phosphate oxidase (PNPO) deficiency is an inborn error of vitamin B6 metabolism, which causes an epileptic encephalopathy responsive to pyridoxal-5-phosphate (PLP). This deficiency is caused by mutations in the PNPO gene encoding this enzyme. Intractable neonatal epileptic encephalopathy within the first hours of life 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 Pharma has received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA) for MC-1 for the treatment of PNPO deficiency.

Clinical Trial(s)

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

Contact Information:

Pat Follows
Project Manager
Telephone: 204-594-3410
Email: pfollows@medicure.com