The designation was requested based on the potential for MC-1 to address an unmet medical need for PNPO deficiency, a serious and life-threatening condition
PNPO deficiency is a rare neurometabolic disorder, often leading to neonatal onset seizures that are resistant to other antiseizure medications, resulting in severe neurological dysfunction and ultimately death if untreated. (Credit: Ali Hajiluyi on Unsplash)
Medicure, a company focused on the development and commercialization of pharmaceuticals and healthcare products for patients and prescribers in the United States market, announced today that through its subsidiary, Medicure International Inc., the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for MC-1, an investigational product for the prevention or treatment of seizures associated with pyridox(am)ine 5′-phosphate oxidase (PNPO) deficiency. Fast Track designation is intended to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need.
The designation was requested based on the potential for MC-1 to address an unmet medical need for PNPO deficiency, a serious and life-threatening condition. Medicure is in the launch phase of its Phase 3 clinical trial to treat approximately 10 PNPO deficient patients at sites in the U.S. and Australia with daily doses of MC-1.
PNPO deficiency is a rare neurometabolic disorder, often leading to neonatal onset seizures that are resistant to other antiseizure medications, resulting in severe neurological dysfunction and ultimately death if untreated.
The FDA has granted both a Rare Pediatric Disease Designation and an Orphan Drug Designation to MC-1 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.
If a new drug application (“NDA”) for MC-1 for patients with PNPO deficiency is approved, Medicure is eligible to receive a priority review voucher from the FDA, which can be redeemed or sold, and provides significant value.
Source: Company Press Release