Medicure Inc., a company focused on the development and commercialization of pharmaceuticals and healthcare products for patients and prescribers in US market, 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 paediatric disease called pyridox(am)ine 5'-phosphate oxidase (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 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.
Under the Food and Drug Administration Safety and Innovation Act (FDASIA) passed into federal law in 2012, the FDA grants a Rare Paediatric 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.
"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.
Medicure is a pharmaceutical company focused on the development and commercialization of therapies for the US cardiovascular market. The present focus of the company is the marketing and distribution of Aggrastat (tirofiban hydrochloride) injection and Zypitamag (pitavastatin) tablets in the United States, where they are sold through the company's US subsidiary, Medicure Pharma Inc.
