The report card of new drugs issued by FDA in 2019 shows that the development trend of orphan drugs is promising

[industry trends of pharmaceutical network] recently, FDA's CDER Office released the "report card" of new drug review for the whole year 2019 in accordance with previous practices According to statistics, in 2019, FDA approved 48 new drugs in total, involving more than 30 well-known multinational pharmaceutical enterprises such as Novartis, Lilly, Roche, and rookies such as Baiji Shenzhou Among them, it is worth noting that from the perspective of 48 approved new drugs, the development trend of orphan drugs is promising Of the 48 approved new drugs, 17 were orphan drugs with rare indications, accounting for nearly 40% of the total   It is reported that "orphan drugs" are used to prevent, treat and diagnose rare diseases Due to the small number of patients with rare diseases, low market demand and high cost of research and development, few pharmaceutical companies pay attention to the research and development of their treatment drugs Therefore, these drugs are vividly called "orphan drugs" Only 1% of the 7000 kinds of orphan diseases that have been confirmed have found effective Therapeutic drugs In recent years, more and more pharmaceutical companies began to be fascinated by this drug market, and accumulated a large number of sales in a small number of patients, avoiding the generic drugs and me too drugs competition that large-scale market drugs usually face   At present, the best-selling orphan drug is rituximab (rituximab) In 1997, as an orphan drug, it was approved to treat non Hodgkin's lymphoma With the expiration of the patent, its sales will decline, and its ranking will fall to the fifth by 2020 In the future, the new base company's "Remix" is expected to become the world's best-selling orphan drug, which is used in the hospital Total sales with indications are expected to reach $10 billion In fact, the global market for drugs for rare diseases is still hot, more and more orphan drugs have made progress in R & D, many countries have mature markets, and the number of approved orphan drugs has also increased significantly In order to protect the rights and interests of patients with rare diseases, China has also begun to introduce measures such as tax reduction, accelerated approval, and inclusion of orphan drugs in medical insurance to improve the R & D motivation of domestic pharmaceutical enterprises Nowadays, many domestic pharmaceutical companies have begun to lay out in the field of orphan drugs For example, kondini pharmaceutical started the research and development of orphan drugs in 2006 It is a pioneer in China's orphan drug market and plays a leading role in the drug market of idiopathic pulmonary fibrosis (IPF) Its future development direction is to further develop, manufacture and distribute drugs for the treatment of organ fibrosis and other rare diseases In addition, Jiangsu Hengrui pharmaceutical company's main orphan drug is atan (apatinib) It is a specific tyrosine kinase inhibitor targeting VEGFR2 independently developed by Jiangsu Hengrui In 2014, it was approved to treat gastric cancer in China, and it is also a safe and effective oral small molecule anti angiogenesis targeted drug in advanced gastric cancer in the world In fact, national support has always been an effective measure to protect the rights and interests of patients with rare diseases There are still many things we can do to encourage the research and development of orphan drugs and improve the accessibility of national specialty drugs I believe that in the future, with the gradual improvement of China's orphan drug policy, more enterprises will join in the research and development layout of orphan drugs, benefiting more patients with rare diseases.