Dyax Receives DX-88 Orphan Drug Designation in Europe; Angioedema Indication
BUSINESS WIRE - January 08, 2003 08:02
CAMBRIDGE, Mass., Jan 8, 2003 (BUSINESS WIRE) -- Dyax Corp. (Nasdaq: DYAX) announced today that the European Commission for Proprietary Medicinal Products (CPMP) has granted orphan medicinal product designation for Dyax's recombinant inhibitor of human plasma kallikrein, DX-88, for treatment of acquired and hereditary angioedema. Dyax is currently conducting two phase II clinical studies of DX-88 in patients with hereditary angioedema (HAE).
The CPMP's confirmation of DX-88's orphan designation followed on a positive recommendation on November 15, 2002 by the Committee for Orphan Medicinal Products (COMP) of the European Agency for the Evaluation of Medicinal Products (EMEA). European orphan drug designation is granted for products that are intended to treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 persons. 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.
Incentives provided by orphan designation in Europe include protocol assistance and market exclusivity, as well as 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. Upon marketing authorization, and during the period of market exclusivity, applications to market a similar medicinal product for the same therapeutic indication may not be accepted or approved by the CPMP, except in very limited circumstances, for a period of up to 10 years.
Dyax has submitted a similar application for orphan designation of DX-88 with the United States Food and Drug Administration (FDA).
Hereditary Angioedema
The prevalence of hereditary angioedema is believed to be between 1/10,000 and 1/50,000 people worldwide. The condition is caused by reduced levels or function of C1 esterase inhibitor (C1-INH), a naturally occurring molecule that inhibits kallikrein and other serine proteases in the blood. HAE is characterized by acute episodic attacks of edema (swelling) in body parts, most notably the hands, feet, face, and abdomen. In the case of an attack that affects the airway passages, HAE can be life-threatening. Abdominal attacks are often associated with bouts of severe pain, nausea |
