Medicure Inc. announced that the FDA provides complete approval to enroll patients in its pivotal Phase 3 clinical trial to evaluate the use of its investigational product MC-1 for treatment of a rare pediatric disease called pyridox(am)ine 5'-phosphateoxidase (PNPO) deficiency. The study involves approximately 10 patients at sites in the United States and Australia, and the Company is seeking marketing approval initially in those countries. The U.S. Food and Drug Administration ("FDA") has granted both Orphan Drug Designation and Rare Pediatric Disease Designation to MC-1 for the treatment of seizures associated with PNPO deficiency.

Additionally, the European Medicines Agency ("EMA") has granted Orphan Drug Designation toMC-1 for the treatment of PNPO deficiency. Under the Food and Drug Administration Safety and Innovation Act (FDASIA) passed into federal law in 2012, the FDA grants a Rare Pediatric Disease Designation for serious and life-threatening diseases in which the serious or life-threatening manifestations primarily affect individuals from birth to 18 years of age, with a prevalence of less than 200,000 people in the United States. If a new drug application ("NDA") for MC-1 for patients with PNPO deficiency is approved, the Company may be eligible to receive a priority review voucher ("PRV") from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application.