Alexion and independent investigators are evaluating the use of eculizumab for the treatment of severe,
life-threatening blood cell disorders, including Cold Agglutinin Disease (CAD) and ANCA Vasculitis. Alexion
has also initiated a Phase I study of TT30, a unique inhibitor of the alternative complement pathway with
a mechanism of action different from Soliris. The focus of the trial is to characterize TT30’s mechanism
of action and to develop initial safety data. When data from the Phase I study are available, Alexion will
evaluate the overall therapeutic potential of TT30 for various disease targets.
Cold Agglutinin Disease (CAD)
CAD is a rare blood disorder characterized by activation of the complement cascade and agglutination,
or sticking together, of red blood cells. CAD causes destruction of red blood cells and symptoms such
as fatigue, shortness of breath, weakness, darkened urine (hemoglobinuria), jaundice, and discomfort or
pain in affected limbs and tissues. In severe cases, complications related to progressive anemia or
blood transfusions may result in death. An investigator-initiated Phase 2 study of eculizumab in the
treatment of patients with CAD is currently under way.
Antineutrophil cytoplasm antibody (ANCA)-associated vasculitis, also known as AAV, causes
destructive inflammation of small-caliber arterial vessels, resulting in considerable morbidity
and mortality, with end-stage renal failure developing in more than 20% of patients at five years.1
Eculizumab is being evaluated in a Phase 2 study as a potential treatment for AAV.
- Hamour A, Salama AD, Pusey CD. Management of ANCA-associated vasculitis: Current trends and future
prospects. Therapeutics and Clinical Risk Management. 2010;6:253–264