The orphan drug designation will entitle Enobia to a seven-year period of marketing exclusivity for the drug upon FDA approval, as well as the opportunity to apply for funding from the US government to defray costs of clinical trial expenses, tax credits for clinical research expenses and potential waiver of the FDA’s application user fee.
Robert Heft, CEO of Enobia, said: “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. 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.”
The FDA grants orphan drug designation to encourage biotechnology and pharmaceutical companies to develop products that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 people in the US.