US-based Dyax has received orphan drug designation from the US Food and Drug Administration (FDA) for its drug candidate DX-2930, a novel, fully human monoclonal antibody inhibitor of plasma kallikrein, for use in the treatment of hereditary angioedema (HAE).
DX-2930 is being developed as a long-acting, prophylactic agent that prevents HAE attacks and the company’s drug development plans include a dosage formulation that will permit infrequent self-administration by small volume, subcutaneous injection.
The drug candidate is currently being evaluated in a placebo-controlled, dose-escalation Phase I study in normal individuals and the results are expected to be released in the first quarter of 2014.
Dyax president and chief executive officer Gustav Christensen saidL "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."
DX-2930 was discovered using the company’s proprietary phage display technology platform.
According to the company, uncontrolled plasma kallikrein activity leads to excessive generation of bradykinin, a vasodilator thought to be responsible for the localised swelling, inflammation and pain characteristically associated with HAE.
The preclinical studies suggest that DX-2930 will have a long half-life in humans, providing the potential for a long-acting and sustained therapeutic effect with less frequent dosing.