Medicure Secures USFDA Nod To Treat Rare Paediatric Disease

Medicure Inc announced that the FDA has granted full approval to enroll patients in its pivotal phase 3 clinical trial to evaluate the use of its investigational product MC-1 for the treatment of pyridox(am)ine 5'-phosphate oxidase (PNPO) deficiency. The research will involve about ten patients at facilities in the United States and Australia, and the business will seek marketing permission in those countries first.

The US Food and Drug Administration (FDA) has granted both Orphan Drug Designation and Rare Paediatric 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 to MC-1 for the treatment of PNPO deficiency, as per pharmabiz. 

The FDA grants a Rare Paediatric Disease Designation under the Food and Drug Administration Safety and Innovation Act (FDASIA), which became federal law in 2012, for serious and life-threatening diseases whose 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 the FDA approves a new drug application (NDA) for MC-1 for patients with PNPO deficiency, the business may be entitled for a priority review voucher (PRV), which can be redeemed for priority review of any future marketing application.

"MC-1 has the potential to become the first FDA-approved therapy for patients with PNPO deficiency. We sincerely thank all of the clinicians, patients and their families for participating in this study." said Dr. Albert D. Friesen, CEO of Medicure and Chair of its board of directors.