Children's myasthenia drugs were approved for the first time in China

On May 7, Food&Drug Administration (FDA) approved the drug Ruzurgi (amifampridine) of Jacobus Pharmaceutical Company for the treatment of Lambert-Eaton myasthenia syndrome (LEMS) in patients aged 6 to 17. It is understood that this is the first time that FDA has approved a new drug for LEMS pediatric patients. Just last November, the FDA approved a treatment regimen for adult patients with LEMS. Journalists noted that the FDA has accelerated the approval of dozens of orphan drugs since 2018. On the contrary, China's policy on orphan drugs has gradually been liberalized, the speed of approval has been accelerating, and favorable policies have emerged frequently. Shi Lichen, founder of Beijing Dingchen Management Consulting Co., Ltd., told reporters that because of the green channel for approval, the speed of approval of rare diseases drugs has been significantly accelerated, and the speed of introducing rare diseases drugs from abroad to domestic market has been further accelerated. As long as it meets the requirements of listing, it only takes about a year or even a few months to achieve listing.

The world's first drug for myasthenia in children has been approved

On May 7, the news came that Ruzurgi, an orphan drug, was approved by FDA and its indication was LEMS in children aged 6-17. Orphan medicine refers to medicines for rare diseases. Journalists learned from the FDA website that Dr. Billy Dunn, Director of Neurological Products of the FDA Drug Evaluation and Research Center, said the FDA would continue to work to promote the development and approval of treatment for rare diseases, especially for children. He believes that this decision will provide pediatric patients with LEMS with much-needed treatment options.

FDA official website shows that LEMS is a rare autoimmune disease, which can affect the neuromuscular connection of patients, and lead to weakness and other symptoms in affected patients. In people with LEMS, the body's own immune system attacks neuromuscular junctions (connections between nerves and muscles) and destroys the ability of nerve cells to send signals to muscle cells.

FDA website points out that LEMS may be related to other immune diseases of patients themselves, but is more common in patients with cancer, such as small cell lung cancer. The prevalence of LEMS in pediatric patients is not yet clear, and the overall prevalence rate is estimated to be three parts per million. The evidence of adequacy and good control of adult LEMS drugs also provides strong support for Ruzurgi's application in patients aged 6 to 17. It is known that the most common side effects of Ruzurgi are burning or tingling (abnormal sensation), abdominal pain, indigestion, dizziness and nausea. If patients develop allergic reactions such as rash, urticaria, itching, fever, swelling or dyspnea after taking Ruzurgi, their medical staff should be informed immediately.

According to FDA statistics, there are more than 7000 rare diseases known in the world, accounting for about 10% of the number of human diseases. There are also more than 15 million patients with rare diseases in China. In recent years, orphan drugs have been on the market at an ever-accelerating pace. FDA has approved a variety of related drugs to be on the market, with 59 new drugs coming on the market in 2018 alone, a record. The number of orphan drugs approved in China is relatively less than that in the United States. Ding Jinxi and Li Na, academics at the Center for Pharmaceutical Industry Development and Research of China Pharmaceutical University, have previously listed 64 orphan drugs in the United States and 16 orphan drugs in China from 2000 to 2010, respectively. At the same time, there are still some diseases that are still in the status quo of "there are drugs outside the country, but there are no drugs inside the country". According to the China Rare Disease Drug Accessibility Report 2019 issued jointly by the Center for Rare Diseases Development and IQVIA, 74 of the 121 rare diseases in the First Rare Disease Catalogue published in China were "curable with medicines", i.e. drugs for these 74 rare diseases were approved in the European Union and the United States as of December 2018. However, there are still 21 rare diseases drugs that have not been introduced into the domestic market.

However, this situation has gradually improved. On May 22, 2018, the first batch of rare diseases catalogue was jointly issued by many departments in China, which included 121 kinds of diseases, and took an important step to support the use of drugs for rare diseases. Previously, the executive meeting of the State Council pointed out that from March 1 this year, for the first batch of 21 rare disease drugs and 4 raw materials, VAT will be levied at 3% for import links with reference to anticancer drugs, and VAT will be levied at 3% for domestic links with a simple method. In addition, on April 17 this year, the State Medical Insurance Bureau released the Work Programme for the Adjustment of the National Medical Insurance Drug Catalogue in 2019, giving priority to the treatment of major diseases such as non-medical insurance varieties, cancer and rare diseases in the national essential medicines. For patients with rare diseases, high drug prices are still a barrier in front of them. It is the hope of many patients to include rare diseases in medical insurance. According to Shi Lichen, the inclusion of rare diseases into medical insurance needs to be carried out step by step, and will be gradually integrated into different regions and diseases according to the actual economic situation of different regions, namely, the ability to pay for medical insurance.