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Dyax Corp. (NASDAQ:DYAX) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its drug candidate DX-2930, its fully human monoclonal antibody inhibitor of plasma kallikrein, for use in the treatment of hereditary angioedema (HAE).
Dyax is developing DX-2930 to be a long-acting, prophylactic agent that prevents HAE attacks. Development plans include a dosage formulation that will permit infrequent self-administration by small volume, subcutaneous injection. DX-2930 is currently being studied in a placebo-controlled, dose-escalation Phase 1 trial in normal individuals. Results from this study are expected in the first quarter of 2014.
“Through our experience in the development and commercialization of KALBITOR
® (ecallantide), we have gained a deep understanding of the HAE market and patient needs,” said Gustav Christensen, President and Chief Executive Officer of Dyax. “There is still a significant unmet medical need within the HAE community which we plan to address with DX-2930. Orphan drug designation is an important element of our development strategy for DX-2930 as we work to further improve the health and quality of life for individuals suffering from this painful and often debilitating condition.”
Orphan drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides FDA assistance in clinical trial design, an exemption from FDA user fees and eligibility for a seven-year period of market exclusivity in the U.S. after product approval.
Discovered using Dyax’s proprietary phage display technology platform, DX-2930 is a novel, fully human monoclonal antibody inhibitor of plasma kallikrein. Uncontrolled plasma kallikrein activity leads to excessive generation of bradykinin, a vasodilator thought to be responsible for the localized swelling, inflammation and pain characteristically associated with HAE. Preclinical studies suggest that DX-2930 will have a long half-life in humans, offering the potential for a long-acting and sustained therapeutic effect with less frequent dosing. Dyax is currently developing DX-2930 as a subcutaneous injection for the prevention of HAE attacks and the candidate is currently in a Phase 1 clinical trial.