Alexion reports that an orphan drug designation has been granted by Japan’s Ministry of Health to Soliris® (eculizumab) for the treatment of patients with myasthenia gravis. In patients with MG, uncontrolled complement activation due to antibodies directed at the neuromuscular junction can ultimately lead to profound and debilitating weakness of various muscle groups throughout the body. This significant muscle weakness can impair patients’ ability to walk, speak clearly, swallow and, in some cases, to breathe … more

Earlier, Soliris® received the orphan drug designation from the Japanese ministry for the treatment of neuromyelitis optica. In patients with that disease, chronic, uncontrolled complement activation results in severe damage to the central nervous system, predominantly impacting the optic nerve and spinal cord … more