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Medicure Announces Pivotal Phase 3 Trial IND Filing with FDA for Treatment of Seizures Associated with Pyridox(am)ine 5'-phosphate oxidase (PNPO) Deficiency
Medicure Announces Pivotal Phase 3 Trial IND Filing with FDA for Treatment of Seizures As...
About this update from Medicure Inc.
[{"type":"text","content":"\n \n \n \n Medicure Announces Pivotal Phase 3 Trial IND Filing with FDA for Treatment of Seizures Associated with Pyridox(am)ine 5'-phosphate oxidase (PNPO) Deficiency\n \n \n /* Style Definitions */\nspan.prnews_span\n{\nfont-size:8pt;\nfont-family:\"Arial\";\ncolor:black;\n}\na.prnews_a\n{\ncolor:blue;\n}\nli.prnews_li\n{\nfont-size:8pt;\nfont-family:\"Arial\";\ncolor:black;\n}\np.prnews_p\n{\nfont-size:0.62em;\nfont-family:\"Arial\";\ncolor:black;\nmargin:0in;\n}\n \n \n \n \n \n \n Canada NewsWire\n \n \n \n \n \n WINNIPEG, MB\n \n ,\n \n Jan. 7, 2021\n \n /CNW/ - Medicure Inc. (\"\n \n Medicure\n \n \" or the \"\n \n Company\n \n \") (TSXV: MPH) (OTC: MCUJF), a cardiovascular pharmaceutical company, today announced that through its\n \n Barbados\n \n subsidiary, Medicure International Inc., it intends to file an Investigational New Drug (\"IND\") application with the U.S. Food and Drug Administration (\"FDA\") pertaining to its legacy product Pyridoxal 5′-phosphate (\"P5P\", also referred to as \"MC-1\") for the treatment of seizures associated with pyridox(am)ine 5'-phosphate oxidase (\"PNPO\") deficiency. The majority of patients with PNPO deficiency have mutations in the PNPO gene, which is required for the production of normal levels of P5P. In connection with the IND, the Company will proceed with a Phase 3 clinical trial to treat PNPO deficient patients with a daily dose of MC-1.\n \n \n The FDA and the European Medicines Agency (\"EMA\") have both granted a Rare Pediatric Disease Designation to MC-1 for the treatment of seizures associated with PNPO deficiency. Additionally, the FDA has granted Orphan Drug Status and a Rare Pediatric Disease Designation to MC-1 for the treatment of PNPO deficiency.\n \n \n Under the Creating Hope Act passed into federal law in 2012, the FDA grants a Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer 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.\n \n \n \"MC-1 has the potential to become the first FD...