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For more than 60 years, steroids and acetylcholinesterase inhibitors have been widely used as therapeutic options for patients with myasthenia gravis (MG), mostly because alternative means of immunotherapy have always been associated with serious adverse events (AEs).
Last night, Alexion Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Soliris (eculizumab) as a new treatment for adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody-positive. The drug exhibited clinical benefit for these patients who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms.
gMG is a debilitating, chronic and progressive autoimmune neuromuscular disease that can occur at any age. In patients with the condition, antibodies block, alter, or destroy the acetylcholine receptors at the neuromuscular junction. Symptoms most commonly revolve around weakness in an array of muscles, causing difficulties in seeing, walking, talking, swallowing, and breathing.
Last night, Alexion Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Soliris (eculizumab) as a new treatment for adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody-positive. The drug exhibited clinical benefit for these patients who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms.
gMG is a debilitating, chronic and progressive autoimmune neuromuscular disease that can occur at any age. In patients with the condition, antibodies block, alter, or destroy the acetylcholine receptors at the neuromuscular junction. Symptoms most commonly revolve around weakness in an array of muscles, causing difficulties in seeing, walking, talking, swallowing, and breathing.
Soliris is a complement inhibitor. The complement cascade, when activated in an uncontrolled manner, plays a pivotal role in gMG, as well as serious ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PHN) and typical hemolytic uremic syndrome (aHUS)—the other indications that Soliris is approved for.