Soliris (eculizumab) is currently approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). It is not approved in any country to treat MG.
Alexion Pharmaceuticals Executive Vice President and R&D Global Head Martin Mackay said patients with MG develop debilitating muscle weakness, impairing their ability to walk, speak clearly, swallow and, in some cases, to breathe normally, which could lead to a life-threatening myasthenic crisis.
"By specifically inhibiting the terminal complement pathway, which is believed to play a pivotal role in the pathophysiology of MG, we believe that eculizumab has the potential to help patients living with this devastating rare disorder," Mackay added.
The company is recruiting for a multinational, placebo-controlled registration trial of eculizumab in patients with refractory generalized MG.
MG is a rare, debilitating neurologic disorder and is caused by uncontrolled complement activation due to antibodies directed at the neuromuscular junction, ultimately leading to profound and debilitating weakness of various muscle groups throughout the body.