Debiopharm S.A. and Dyax Corp. Granted Orphan Drug Designation for DX-890 by U.S. FDA
Cystic Fibrosis Indication
CAMBRIDGE, Mass. & LAUSANNE, Switzerland--(BUSINESS WIRE)--Dec. 15, 2003--Dyax Corp. (Nasdaq: DYAX - News) and Debiopharm S.A. (Debiopharm) announced today that the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) has granted orphan drug designation to the engineered protein inhibitor of human neutrophil elastase (EPI-hNE4), or DX-890, for the treatment of cystic fibrosis (CF). Today's news follows the announcement by the two companies in July that orphan drug designation for DX-890 in CF had also been granted by the Commission for Proprietary Medical Products (CPMP) of the European Community. DX-890 was discovered at Dyax Corp. and Debiopharm is responsible for clinical evaluation of the compound as a treatment for CF. Debiopharm has completed two Phase IIa clinical trials of DX-890 in CF patients in Europe and the companies are planning a larger Phase IIb study in Europe and the United States, to be initiated by Debiopharm in late 2004.
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. |
