According to a story from BusinessWire, the drug developer Alexion Pharmaceuticals, Inc., recently announced positive results for its Phase 3 trial testing Soliris as a therapy for neuromyelitis optica spectrum disorders in patients that are APQ4 auto antibody positive, an important biomarker that is used to identify the disease. However, about 25 percent of patients with neuromyelitis optica spectrum disorders (NMOSD) do not have this biomarker.
About Neuromyelitis Optica Spectrum Disorders (NMOSD)
Neuromyelitis optica spectrum disorders (NMOSD) is a term meant to include both neuromyelitis optica patients and those that lack the APQ4 auto antibody but still present similarly otherwise. This disorder is also known as Devic’s disease. It is characterized by inflammation of the optic nerve and spinal cord along with destruction of the myelin sheath, an insulating, protective layer surrounding nerve cells. It is considered an autoimmune disease in which the immune system mistakenly begins attacking parts of the body. It is frequently associated with other diseases, such as viral infection and antiMOG associated encephalomyelitis, that latter of which can be a direct cause in some cases. Symptoms include blindness, urinary incontinence, spastic paralysis of the legs and arms, reduced sensation, and overall muscle weakness. Symptoms can be treated, but many patients are left with a degree of impairment. To learn more about neuromyelitis optica, click here.
About The Study
In the study, Soliris was effective in reducing the risk of NMOSD relapse. In comparison to placebo, Soliris was able to reduce the chance of a patient experiencing disease relapse by 94.2 percent. After a period of 48 weeks, 97.9 percent of patients who were treated with Soliris were relapse free compared to just 63.2 percent of the placebo group. These results suggest the Soliris could be a highly effective tool for preventing relapse, which is common for nearly 85 percent of patients.
Soliris has already been active on the market as a therapy for atypical hemolytic uremic syndrome and paroxysmal nocturnal hemoglobinuria. It is a monoclonal antibody which function as a complement inhibitor, and it is also the first therapy approved for these indications. Patients who use Soliris are at an elevated risk of meningococcal infection, and patients must agree to follow a strict risk evaluation and mitigation protocol before beginning treatment.