Enobia Pharma, an emerging biotech 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 (FDA) for ENB-0040, its enzyme replacement therapy (ERT) for hypophosphatasia, a rare, lifethreatening genetic bone disease. In August 2008, Enobia dosed the first patient in a phase-I clinical trial of ENB-0040.
"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 officer 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 programme."
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 United States. This 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.
Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease. Patients have low levels of the tissue non-specific form of alkaline phosphatase, an important regulator of bone mineralization, leading to rickets in infants and children and osteomalacia (soft bones resulting from poor mineralization) in adults.
ENB-0040 is a fusion protein that includes the catalytic domain of human tissue nonspecific alkaline phosphatase (TNSALP), an immunoglobulin Fc domain and a patented anionic peptide used to target the enzyme to bone.
Enobia Pharma Inc, is a private, Montreal based company focused on the development of therapeutics to treat serious bone disorders for which there is no currently approved drug therapy.