Health Problem: Myasthenia gravis (MG) is a rare autoimmune disorder that impacts the neuromuscular system, leading to muscle weakness, particularly affecting the nasopharyngeal, limb, and respiratory muscles. MG is a progressive disease caused by autoantibodies that interfere with the transmission of acetylcholine from nerve endings to muscle cells. It is estimated that 36,000 to 60,000 people in the United States have MG. The current estimates of the mortality rate range from 0.06 to 0.89 deaths per million person-years.

Technology Description: Eculizumab is a recombinant humanized monoclonal antibody that is used for the treatment of generalized MG in adults who are anti-acetylcholine receptor antibody positive. Eculizumab is administered intravenously at a concentration of 5 milligrams/milliliter over a period of 35 minutes.

Controversy: Standard treatments for generalized MG include cholinesterase inhibitors and immunosuppressive agents; however, 10% to 15% of patients have disease that is refractory to these measures. Current treatments for refractory MG are not specific for MG and have limited effectiveness. Eculizumab targets the mediators of the destruction of the neuromuscular junction that leads to the muscular weakness associated with MG. However, it has yet to be determined whether eculizumab is an effective and safe treatment for refractory generalized MG.

Key Questions:

  • Is eculizumab effective in managing refractory generalized MG?

  • How does eculizumab compare with alternative treatments for refractory generalized MG?

  • Is eculizumab safe?

  • Have definitive patient selection criteria been identified for eculizumab for treatment of generalized MG?
