Every June 15th is Myasthenia Gravis Care Day. A few days ago, the world’s first FcRn antagonist efgartigimod landed in the Boao Lecheng International Medical Tourism Pilot Zone, becoming a new breakthrough in myasthenia gravis drugs in my country in the past three decades.
As a key park for the construction of Hainan Free Trade Port, the Lecheng Pioneer Zone has basically achieved the simultaneous introduction of international innovative medical devices and the availability of drugs, and has entered a high-quality development. new stage. In the field of rare diseases, Lecheng has been committed to improving the efficiency of rare disease drug introduction, and providing integrated services from drug introduction, approval to treatment for rare disease patients.
Myasthenia gravis is a rare, chronic autoimmune disease caused by dysfunction of neuromuscular junction transmission. It has been included in my country’s “First Batch of Rare Disease List”. It is estimated that there are at least 200,000 myasthenia gravis patients in my country. If the symptoms are not well controlled, myasthenia gravis will seriously affect the daily life and work of the patient, reduce the mobility and quality of life, and bring a heavy burden to the family and society. More than 85% of adult myasthenia gravis patients will develop generalized myasthenia gravis within 18 months of onset, which can affect patients’ eye movements, swallowing, speech, activity and respiratory function to varying degrees, and even life-threatening in severe cases.
Currently the most common treatment options for myasthenia gravis include cholinesterase inhibitors, glucocorticoids, immunosuppressants, intravenous immunoglobulin, plasma exchange, anti-CD20 monoclonal antibody and thymectomy. Professor Zhao Chongbo, Chief Physician of the Department of Neurology, Huashan Hospital Affiliated to Fudan University, pointed out: “The disease management of patients with myasthenia gravis is difficult, and some patients clinically have symptoms control due to problems such as drug efficacy, tolerance and contraindication, and poor drug accessibility. It is not good and fluctuates repeatedly, and it will also suffer from side effects due to the long-term use of immunosuppressive drugs. In addition, due to the large individual differences of patients with myasthenia gravis, the progression of the disease is different, and there are a certain proportion of refractory patients and the potential development of myasthenia gravis. Crisis patients. efgartigimod provides clinicians and patients with an innovative treatment plan based on high-level evidence-based medical evidence with fast onset, durable efficacy and excellent safety for the treatment of myasthenia gravis.”
efgartigimod is a novel therapeutic drug for antibody-mediated autoimmune diseases and highly targeted immunoglobulin (IgG). In December last year, the world’s first FcRn antagonist efgartigimod was approved for marketing in the United States. It has become the world’s first and currently only approved FcRn antagonist, and the first approved therapy specifically for the treatment of generalized myasthenia gravis through endogenous reduction of pathogenic antibodies.
Professor Huang Shixiong, director of the Department of Neurology, Hainan Provincial People’s Hospital, said: “As a rare disease, the treatment of myasthenia gravis involves multiple disciplines. To accept new drug treatment, we have established a special medical team including neurology, emergency department, ICU, etc., and established a special remote multidisciplinary consultation mechanism with well-known experts in the field of internal neurology to maximize the protection of myasthenia gravis patients. The quality of medical treatment.”
Up to now, the number of imported licensed medicinal devices in the Lecheng Pioneer Zone has exceeded 220 cases. Zai Lab said that efgartigimod is also conducting global clinical studies in a variety of serious autoimmune diseases. On the day of the event, the Bethune·Healthy Dream-Myasthenia Gravis public welfare caring activity was also launched.
Xinmin Evening News reporter Zuo Yan