FDA Approves Alexion’s Solaris to Treat Rare CNS Disease

The U.S. Food and Drug Administration gave Alexion Pharmaceuticals expanded approval for 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.  According to the National Institutes of Health, women are more often affected by NMOSD than men and African Americans are at greater risk of the disease than Caucasians. Estimates vary, but NMOSD is thought to impact approximately 4,000 to 8,000 patients in the United States.

“NMOSD is a serious disease with devastating consequences,” said Michael Levy, 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 can be 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 FDA approval is for the use of Soliris in adult patients who are anti-AQP4 antibody positive. NMOSD is an autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord.

The approval of Soliris injection for intravenous use for the treatment of NMOSD was based on a clinical study of 143 patients with NMOSD who had antibodies against AQP4 who were randomized to receive either Soliris treatment or placebo. Compared to treatment with placebo, the study showed that treatment with Soliris reduced the number of NMOSD relapses by 94 percent over the 48-week course of the trial. Soliris also reduced the need for hospitalizations and the need for treatment of acute attacks with corticosteroids and plasma exchange.

Soliris was first approved by the FDA in 2007 to reduce destruction of red blood cells in adults with a rare blood disease called paroxysmal nocturnal hemoglobinuria, for the treatment of adults and children with a rare disease that causes abnormal blood clots to form in small blood vessels in the kidneys (atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy), and for the treatment of adults with Myasthenia Gravis who are anti-acetylcholine receptor antibody positive.

The drug carries a boxed warning to alert health care professionals and patients that life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris, and that such infections may become rapidly life-threatening or fatal if not recognized and treated early. In the NMOSD clinical trial, no cases of meningococcal infection were observed.

Soliris as a treatment for NMOSD was granted Priority Review and Orphan Drug designation by the FDA. It is currently under review in Europe and Japan.

Author: Rare Daily Staff