A rare, debilitating neurologic disorder, MG 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.
Alexion executive vice-president, global head of R&D 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 said.
Currently, Soliris is approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), while it is not approved in any country for the treatment of MG.
The company also started recruiting for a multinational, placebo-controlled registration trial of eculizumab in patients with refractory generalized MG.