According to a story from GlobeNewswire, the biotechnology company argenx announced recently that its investigational therapy ARGX-113 was granted orphan drug status by the European Commission. This designation is based on a review from the European Medicines Agency (EMA). ARGX-113 is in development for treating myasthenia gravis.
Myasthenia gravis (MG) is a long term neuromuscular disease that has the potential to lead to weakness of the skeletal muscles. The muscles of the face and eyes are the most commonly affected, and many patients have trouble swallowing. Other symptoms include problems talking and walking, drooping eyelids, double vision, enlarged thymus, and inability to hold the head up. Myasthenia gravis is an autoimmune condition in which antibodies prevent the action of the nerves that responsible muscle contractions in the affected areas. Over the past century, treatment for myasthenia gravis has improved dramatically; in the past, many of those affected did not survive. Treatments today can manage symptoms to the point that the patient can perform most day to day activities, though they may still experience muscle weakness. To learn more about this disease, click here.
ARGX-113 has previously also received orphan drug designation in the U.S. The treatment has the potential to give MG patients a treatment option that is both more effective than those currently available and has less side effects. The treatment functions by targeting the immune system antibodies that are responsible for causing MG. When the activity of these antibodies is reduced, the symptoms of the disease improve.
