FDA Approves First Treatment for Rare Autoimmune Disease
The FDA approved Alexion’s Soliris (eculizumab) injection for treating neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4) antibody positive, making it the first treatment for the rare autoimmune disease.
NMOSD impacts the central nervous system, affecting mainly the optic nerves and spinal cord. Symptoms include loss of vision or blurriness in one or both eyes, weakness of limbs, paralysis in the limbs and loss of bladder or bowel control.
Soliris is approved for three other rare diseases, including paroxysmal nocturnal hemoglobinuria, a rare blood disease that destroys red blood cells, impairs bone marrow function and causes thrombosis.