FDA approves Soliris to treat neuromyelitis optica spectrum disorder

Alexion Pharmaceuticals Inc. announced that the FDA approved Soliris (eculizumab) for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. Approximately three quarters (73%) of all patients with NMOSD test positive for anti-AQP4 auto-antibodies. The FDA approved Soliris following an expedited six-month priority review.

NMOSD is a rare, severe autoimmune disease that attacks the central nervous system without warning. These attacks, also referred to as relapses, can cause progressive and irreversible damage to the brain, optic nerve and spinal cord, which may lead to long-term disability. Complement activation due to anti-AQP4 antibodies is one of the primary underlying causes of the destruction in these patients. In the PREVENT trial, Soliris, a first-in-class complement inhibitor, demonstrated safety and efficacy and met its primary endpoint of prolonging the time to first adjudicated relapse and reducing the risk of relapse.

“NMOSD is a serious disease with devastating consequences,” said Michael Levy, M.D., Ph.D., a consultant to the company and Associate Professor of Neurology at Massachusetts General Hospital in Boston. “Each attack can result in potentially irreversible consequences—causing blindness or losing the ability to walk—so preventing relapse is the primary goal of treatment. With the approval of Soliris, there is now for the first time an FDA-approved treatment available to NMOSD patients to help reduce the risk of relapse.”

NMOSD disproportionately strikes young women in the prime of their lives, with the average age of first onset at just 39 years. Race is also a significant risk factor for disability and mortality in NMOSD. In the U.S., African Americans are over-represented among patients diagnosed with NMOSD and more likely to suffer more frequent and more severe attacks. Previously known as Devic’s Disease, NMOSD is often confused with other neurological illnesses such as multiple sclerosis (MS), which can lead to delays in diagnosis and treatment with medicines that can worsen disease progression.