RARE Daily

Alexion’s Soliris Wins European Approval to Treat Rare CNS Disease

August 29, 2019

Rare Daily Staff

The European Commission has expanded approval of Soliris to include its use as a treatment for neuromyelitis optica spectrum disorder, a rare autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord.

Neuromyelitis optica spectrum disorder (NMOSD) leads to vision loss and paralysis in about 50 percent of patients from recurring attacks. NMOSD disproportionately strikes young women in the prime of their lives, with the average age of first onset at just 39 years.

NMOSD is an autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord. It is associated with antibodies that bind to a protein called aquaporin-4 (AQP4). Binding of the anti-AQP4 antibody appears to activate other components of the immune system, causing inflammation and damage to the central nervous system. About three quarters of patients with NMOSD test positive for the AQP4 biomarker. The European approval is for the use of Soliris in adult patients who are anti-AQP4 antibody positive.

“In a disease marked by unpredictable relapses that each have the potential for irreversible consequences, such as blindness or the inability to walk, the primary treatment goal is prevention of such attacks. Nearly all patients treated with Soliris were relapse free in the phase 3 study, demonstrating the potential of Soliris to change the treatment paradigm for this devastating disease,” said John Orloff, executive vice president and head of research and development at Alexion. “This approval marks an important milestone for the NMOSD community, which now has an approved treatment option that can help protect patients from the next potentially devastating relapse.”

The European approval is based on results from late-stage trial that is still underway. At 48 weeks, 98 percent of patients treated with Soliris were relapse free compared to 63 percent of patients receiving placebo. Of the patients treated solely with Soliris, without receiving other immunosuppressive therapies, 100 percent were relapse free at 48 weeks compared to 61 percent in the placebo group. Sustained effects were observed through 144 weeks of treatment.

European approval of Soliris to treat NMOSD follows its recent approval in the United States for the same condition. As a treatment for NMOSD, it was granted Orphan Drug designation in the United States, European Union, and Japan, and is currently under review by regulators in Japan.

Soliris (eculizumab) was first approved by the U.S. Food and Drug Administration in 2007 to reduce destruction of red blood cells in adults and children with a rare blood disease called paroxysmal nocturnal hemoglobinuria, a disease that causes abnormal blood clots to form in small blood vessels in the kidneys.  

Photo: John Orloff, executive vice president and head of research and development at Alexion

Stay Connected

Sign up for updates straight to your inbox.

FacebookTwitterInstagramYoutube