The FDA has approved Soliris (eculizumab) for two new uses: generalized myasthenia gravis (gMG) in adults and children ages 6 and older who have a specific antibody, and neuromyelitis optica spectrum disorder (NMOSD) in adults with a different antibody. Soliris is a complement inhibitor, meaning it blocks part of the immune system that can cause damage. It was already approved for two other rare blood and kidney disorders.
This approval is for patients with gMG who test positive for anti-AChR antibodies, and for NMOSD patients who test positive for anti-AQP4 antibodies. These are serious autoimmune diseases that can cause muscle weakness, vision loss, and other problems. The drug is given through an IV, with different dosing schedules for each condition.
For patients, this means there is now an approved treatment for these specific forms of these diseases. However, Soliris has a boxed warning about a serious risk of meningococcal infection. Patients must be vaccinated before starting treatment. This approval does not mean the drug is right for everyone. It is important to talk to your doctor about whether Soliris is a good option for you, based on your medical history and test results.