Canadian pharmaceutical company Medicure has received full approval from the US Food and Drug Administration (FDA) to enrol subjects in a Phase III clinical trial of its investigational product, MC-1.

MC-1 is indicated to treat the rare paediatric disease known as pyridox(am)ine 5′-phosphate oxidase (PNPO) deficiency.

The study will assess the therapy's ability to treat this condition, enrolling around ten patients at centres in Australia and the US.

Medicure is initially seeking marketing approval for the product in these two countries.

The company has secured orphan drug designation and rare paediatric disease designation from the FDA for MC-1 to treat seizures associated with PNPO deficiency.

It has also received orphan drug designation from the European Medicines Agency (EMA) to treat PNPO deficiency.

Medicure CEO and board of directors chair Dr Albert Friesen said: “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.”

Under the FDA Safety and Innovation Act (FDASIA), the FDA can designate a rare paediatric disease status for serious or life-threatening illnesses that primarily impact individuals from birth to 18 years of age.

These diseases must have a prevalence of less than 200,000 people in the US.

The FDASIA was passed into federal law in 2012.

Based in Winnipeg, Medicure is involved in the development and commercialisation of therapies for the cardiovascular market in the US.

The company is currently focusing on the marketing and distribution of AGGRASTAT (tirofiban hydrochloride) injection and ZYPITAMAG (pitavastatin) tablets in the country through its subsidiary Medicure Pharma.

Its Marley Drug pharmacy in North Carolina provides an extended supply drug programme that serves customers in 50 US states, as well as Washington DC and Puerto Rico.