Enobia Pharma Receives FDA Orphan Drug Designation for Hypophosphatasia Product Candidate ENB-0040
MONTREAL, Sept. 19 /PRNewswire/ — 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 U.S. Food and Drug Administration (FDA) for ENB-0040, its enzyme replacement therapy (ERT) for hypophosphatasia, a rare, life-threatening 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 PhD, 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 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 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 U.S. 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. Disease severity is inversely proportional to the age at symptom onset, but morbidity can be cumulative and worsen with age. Clinical severity ranges from the severe perinatal or infantile form, with profound skeletal hypomineralization and respiratory compromise often causing death, to a more slowly progressive and debilitating osteomalacia in adults.
In the infantile form, infants may appear normal at birth but develop serious symptoms in the first six months of life. These can include failure to thrive, respiratory failure, fractures, and seizures. Radiographic findings include generalized hypomineralization and rickets. Mortality in these patients may be as high as 50%. In the childhood form, patients have varying degrees of hypomineralization, frank rickets, short stature, bone pain, muscle weakness, delayed motor milestones, early loss of deciduous teeth, and may experience frequent, poorly-healing fractures. In the adult form, the underlying osteomalacia causes bone pain due to overt or poorly-healing stress fractures that in some cases stops ambulation.
ENB-0040 is a fusion protein that includes the catalytic domain of human tissue non-specific alkaline phosphatase (TNSALP), an immunoglobulin Fc domain and a patented anionic peptide used to target the enzyme to bone. Preclinical studies of ENB-0040 in the “knockout” mouse model of severe hypophosphatasia were recently published in the Journal of Bone and Mineral Research [June 2008:23:777-787] and showed that subcutaneous administration of ENB-0040 significantly improved survival and prevented the skeletal and dental manifestations of the disease.
About Enobia Pharma Inc.
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. Enzyme Replacement Therapy for the treatment of hypophosphatasia is the Company’s lead program. In 2007 Enobia completed a $40M Series B financing lead by OrbiMed Advisors and CTI Life Sciences.
Company Contact: Julie Anne Smith, (514) 596-2901, extension 214
CONTACT: CONTACT: Julie Anne Smith of Enobia Pharma, +1-514-596-2901,ext. 214