Enobia Pharma, an emerging biotech company focused on developing novel therapeutics for serious bone disorders, has received US Food and Drug Administration (FDA) Fast Track status to ENB-0040 for the treatment of hypophosphatasia (HPP).
ENB-0040 is Enobia's enzyme replacement therapy (ERT) for HPP, a rare, potentially life-threatening genetic bone disease for which there is no currently approved therapy.
"We're pleased that the FDA has recognized that ENB-0040 may provide a much needed treatment for patients with this devastating, often fatal inherited bone disease," said Hal Landy, chief medical officer and vice-president of Medical Affairs at Enobia.
Enobia will now have access to ongoing discussions and communications with the FDA to facilitate the ENB-0040 development and review process.
In its letter to the company, the FDA stated that they granted Fast Track designation in part because HPP is an untreated, serious and potentially life-threatening disorder. The FDA went on to note that Enobia's proposal to evaluate the efficacy and safety of ENB0-0040 in restoring bone mineralization would target 'a serious aspect of the disorder'. FDA also stated that 'preliminary clinical data have suggested that ENB-0040 has potential as a specific treatment for hypophosphatasia'.
Results from the company's phase-I trial in HPP adults and its ongoing trial in severely affected HPP infants will be presented by Michael Whyte, of the Shriners Hospitals (St Louis, MO) at The Endocrine Society's 91st Annual Meeting ENDO 09 Conference on Thursday, June 11, 2009 at 9:30 am ET in Washington.
ENB-0040 is a subcutaneous enzyme replacement therapy of tissue non-specific alkaline phosphatase (TNSALP) fused to a patented bone targeting peptide.
Enobia Pharma 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.