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Orphan Drug Designation Granted for DX-890, Cystic Fibrosis Indication
The FDA grants orphan drug designation for products that are intended to treat rare, life-threatening diseases or chronically debilitating conditions that affect no more than 200,000 patients in the U.S. at the time of application. Further criteria include the ability of the product to provide significant patient benefit over available treatment or to fill an unmet medical need where no other treatment exists. Orphan drug designation may result in seven years of market exclusivity in the United States upon FDA product approval, provided that the sponsor company continues to meet certain conditions established by the FDA. Upon marketing authorization and during the period of market exclusivity, the FDA does not accept or approve other applications to market the same medicinal product for the same therapeutic indication. Other incentives provided by orphan designation include protocol assistance and eligibility for research and development support. Protocol assistance includes regulatory assistance and reduced filing fees, as well as advice on the conduct of clinical trials.
Cystic fibrosis is a chronically debilitating and life threatening condition and although satisfactory methods of treatment of the condition have been authorized in the United States, the FDA has indicated that Dyax and Debiopharm have provided data suggesting that DX-890 may offer a significant benefit to those affected by the disease.
"Orphan drug status in both the U.S. and Europe is a significant step in the development and commercialization of DX-890," said Dr. R.-Y. Mauvernay, President and CEO of Debiopharm. "CF is a rare, life-threatening and chronically debilitating condition and we hope that our clinical development of DX-890 will result in an improved treatment for patients living with this disease."
"DX-890 is the second of Dyax's compounds in clinical development to receive orphan drug designation in both the U.S. and Europe, and we're very pleased that the U.S. regulatory authorities recognize the potential of this potent inhibitor of human neutrophil elastase to provide a treatment alternative to patients suffering with cystic fibrosis. We believe that there are significant markets for drugs to treat orphan indications, and are committed to advancing DX-890 in collaboration with our colleagues at Debiopharm," commented Henry E. Blair, Chairman, President and CEO of Dyax Corp.
Cystic fibrosis (CF) is a genetic disease affecting approximately 30,000 children and adults in the United States. According to the CF Foundation's National Patient Registry, the median age of survival for a person with CF is 33.4 years.
Mutations in the CF gene cause the body to produce an abnormally thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections. Inflammation in the airways of CF patients is characterized by persistent and excessive neutrophil infiltration, which release large quantities of destructive oxidases and proteases, including human neutrophil elastase (hNE). Inhibition of hNE allows control of the inflammatory process early in the course of the disease, and may limit the damaging effects of excessive inflammation, thus delaying progression of pulmonary deterioration and potentially decreasing mortality.
DX-890 is a highly specific and potent inhibitor of hNE and may act as a novel anti-inflammatory agent to target neutrophil-related inflammation.
A collaborative Phase IIa open label study, conducted by Debiopharm in 25 adult CF patients, has demonstrated the tolerability and pharmacological effect of DX-890 on the inhibition of hNE in the lungs of CF patients, when administered as a nebulised formulation. The results of the study show that with a 30mg/3ml dose of DX-890, complete inhibition of hNE was observed in 10 out of 19 patients (53%) and partial inhibition was observed in 5 out of 19 patients (26%). The total response rate was 79%.
Recently, the final patient was treated in a second collaborative Phase IIa open label study, conducted by Debiopharm in children with CF. Topline safety and pharmacology results from this study are expected to be announced during the first quarter of 2004.
Source: Dyax Corp and Debiopharm S.A.