FDA approves Soliris® for chronic neuromuscular disorder.

The first treatment for adults with generalized myasthenia gravis (gMG) to be approved in 60 years passed through the FDA on October 23. Alexion Pharmaceuticals, Inc. demonstrated in Phase 3 trials and ongoing open-label extension studies, that its candidate Soliris (eculizumab) provided treatment benefits for patients with significant unresolved disease symptoms.

Intended for anti-acetylcholine receptor (AchR) antibody-positive adults, Solaris, announced Alexion is approved for patients who have failed immunosuppressive treatment and continue to suffer from chronic and debilitating unresolved symptoms including problems breathing, talking, swallowing and walking. According to Alexion, these patients are at increased risk for complications, disease and hospitalization for related crisis events.

Commenting on the advance, Professor James F. Howard, M.D., Department of Neurology at the University of North Carolina and lead investigator in the new indication’s clinical development said, “I am pleased that the FDA recognized the comprehensive clinical data supporting the benefits of Soliris for patients with anti-AchR antibody-positive gMG. It is exciting that patients who have not responded adequately to existing therapies will now have a new treatment option that was shown in clinical studies to improve patients’ symptoms, their ability to carry out activities of daily living and their quality of life.”

Alexion explained, chronic uncontrolled activation of the complement system, part of the immune system, plays a central role in generating unresolved disease symptoms and the disease’s other life-threatening complications. “By selectively and effectively inhibiting the terminal complement cascade,’ said Alexion, “Soliris targets a critical underlying cause of the disease.”

John Orloff, Executive Vice President and Head of Research & Development at Alexion offered this conclusion on news of the approval: “We are proud that we could apply our deep understanding of complement biology to develop Soliris for the treatment of patients with this debilitating neuromuscular disorder.”