Soliris Receives New Indication to Treat Rare Autoimmune Disease
June 27, 2019 – The U.S. FDA has approved a new indication for Soliris® (eculizumab), manufactured by Alexion Pharmaceuticals. The product is now approved to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. Soliris is the first medication to receive FDA approval specifically to treat NMOSD.
NMOSD is a rare, severe autoimmune disorder that affects 4,000 to 8,000 individuals in the United States. It causes the immune system to attack healthy cells, most often in the eyes and spinal cord, which can lead to optic neuritis and transverse myelitis. Optic neuritis causes eye pain and vision loss, while transverse myelitis leads to numbness, weakness, or paralysis of the arms and legs, in addition to loss of bladder and bowel control. Approximately 50% of patients with NMOSD have permanent visual impairment and paralysis caused by NMOSD attacks. Attacks occur in clusters, with periods of remission in between that can last anywhere from days to years.
In clinical studies, 96% of patients treated with Soliris remained relapse free at 144 weeks of treatment, versus 45% of patients who received a placebo. One hundred percent of patients treated solely with Soliris and no other immunosuppressive therapies were relapse free at 144 weeks.
Recommended dosing under the new indication is 900mg administered once weekly via intravenous (IV) infusion for the first four weeks, followed by 1200mg for the fifth dose one week later, and then 1200mg every two weeks thereafter. A black box warning cautions that life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. The drug is available only through a Risk Evaluation and Mitigation Strategy (REMS) program.
Originally FDA-approved in 2007, Soliris is also indicated to reduce hemolysis in patients who have paroxysmal nocturnal hemoglobinuria (PNH), to inhibit complement-mediated thrombotic microangiopathy in patients who have atypical hemolytic uremic syndrome, and to treat generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) antibody positive.