Orphan status for Enobia's ENB-0040

Canada's Enobia Pharma, an emerging biotechnology company focused on developing novel therapeutics for serious bone disorders, announced that it has received Orphan Drug designation from the US Food and Drug Administration for ENB-0040, its enzyme replacement therapy for hypophosphatasia, a rare, life-threatening genetic bone disease. In August this year, Enobia dosed the first patient in a Phase I clinical trial of the drug.

"ENB-0040 represents a potential drug therapy for the patients with hypophosphatasia, an under-recognized disease that can be fatal in infants and cause serious disability in older patients that has no currently-approved FDA treatment," said Robert Heft, chief executive of Enobia. "The recent dosing of the first adult hypophosphatasia patient in our Phase I clinical trial and receipt of orphan drug designation are important milestones for this program," he added.