WASHINGTON (dpa-AFX) - Alexion Pharmaceuticals (ALXN) announced that the European Commission has granted orphan drug designation or ODD to Soliris (eculizumab) for the treatment of patients with Myasthenia Gravis or MG, a rare, debilitating neurologic disorder caused by uncontrolled complement activation.
In patients with MG, uncontrolled complement activation due to antibodies directed at the neuromuscular junction can ultimately lead to profound and debilitating weakness of various muscle groups throughout the body.
Soliris is a first-in-class terminal complement inhibitor and is currently approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation.
Soliris is not approved in any country to treat MG. Alexion is enrolling patients in a multinational, placebo-controlled registration trial of eculizumab in patients with refractory generalized MG.
Myasthenia Gravis is a rare, debilitating neurologic disorder caused by auto-antibodies that recognize a specific target in the nerve-muscle junction, which results in life-long uncontrolled terminal complement activation causing tissue damage and interference with signalling between nerve and muscle fibers. Patients with MG initially experience weakness in their ocular (eye) muscles, and the disease typically progresses to the more severe and generalized form to include weakness of head, trunk, limb and respiratory muscles. Symptoms can include drooping eyelid, weakness in the arms and legs, slurred speech, difficulty chewing or swallowing, and difficulty breathing, which could lead to a life-threatening myasthenic crisis.
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