New policy for approval of drugs for rare diseases

[China Pharmaceutical network enterprise news] edaravone injection is a star product of Xiansheng pharmaceutical industry As a small molecule free radical scavenger, edaravone injection can clear ROS and block ALS, that is to say, oxidative stress pathway of patients with gradually frozen human disease Oxidative stress is a very important link in the pathogenesis of ALS On May 5 this year, the US FDA approved edaravone for the treatment of ALS (the new policy for the approval of drugs for rare diseases has come out, and the pioneer pharmaceutical industry is enjoying good results Source: Baidu picture) Dr Mou Hua, science officer of Jiangsu pioneer pharmaceutical industry, said that recently, he would like to have a communication with the leaders of the drug approval Office of the State Drug Administration To see if edaravone injection can pass the green channel and get the approval of new indications quickly One of the factors that made Dr Mou Hua confident was that on May 11, the State Food and Drug Administration issued a new regulation on encouraging the research and development of drugs and devices for rare diseases, saying that the Department of health and family planning should publish the catalogue of rare diseases and establish a registration system for patients with rare diseases Applicants for rare disease treatment drugs and medical devices can apply for relief of clinical trials and accelerate the review and approval of medical devices for rare disease treatment For rare disease treatment drugs and medical devices that have been approved to be listed abroad, they can be approved to be listed conditionally, and relevant research can be made up within the specified time after listing Edaravone injection is a star product of Xiansheng pharmaceutical industry, which was used to treat stroke It is understood that the annual sales of this drug is more than 1 billion On May 5 this year, the FDA approved edaravone as a treatment for ALS ALS is commonly known as gradually frozen human disease, because the pathogenic factors are not clear, and there is no effective way to segment by segment this disease Most patients watch their muscles shrink a little bit until they are frozen, unable to use them flexibly in their own bodies, and the brain is very clear Doctors say that most patients with this disease watch themselves die little by little Edaravone, as a small molecule free radical scavenger, can clear ROS and block the oxidative stress pathway in ALS patients Oxidative stress is a very important link in the pathogenesis of ALS Animal experiments and clinical phase II and phase III trials showed that edaravone can reduce the oxidative stress response of nerve cells, reduce the deposition of 3-NT and SOD1, and reduce the motor dysfunction of patients According to Dr Mou Hua, gradually frozen human disease is still a relatively difficult rare disease in the world, and the international situation is also not clear about the pathogenic factors, and can not be cured, so it can only be delayed In order to promote the control of this disease, FDA of the United States found and studied edaravone, and found that it had obvious curative effect, so it took the initiative to contact the Japanese drug regulatory department, hoping that the drug developed by Mitsubishi company of Japan could enter the American market as soon as possible Based on the initiative of FDA in the United States, Dr Mou Hua thinks that maybe it can be promoted as soon as possible to help patients According to Dr Mou Hua, at this stage, the focus of the production and research of edaravone for ALS can be divided into two aspects One is the rapid approval process of CFDA applying for rare disease drugs approved abroad The second is to study and verify the efficacy of compound edaravone (phase III clinical trial stage), which has stronger free radical scavenging effect, in the treatment of ALS So as to eventually bring faster and more effective treatment for ALS patients It is understood that at present, the recognized effective drug for this disease is riluzole, but this drug is more than 20 years ago, from the development and production of Sanofi Relative to the change of disease, maybe there should be new or complementary drugs in the market In terms of policies and regulations, if we don't reform, according to the old rules, after a drug has completed clinical trials abroad, it must be done again when it enters China According to one doctor, the rare diseases and common diseases in China are not the same as each other They are much more complicated and slow to do Because of the small sample size For example, edaravone, which is currently used by the pioneer pharmaceutical industry for cerebrovascular disease indications, needs to approve the secondary indications According to the old rules, it may take 3, 5 years or even longer to start from scratch With the new deal, it is possible to directly declare it, that is, to approve it first and then complete the corresponding experiments, which will greatly speed up the pace For patients, it's also good news In the later stage, the effect of medication for patients will decrease a lot Moreover, the cost of riluzole for one month is more than 4000 yuan, all of which are at their own expense Edaravone, on the other hand, was used in one package at a significantly lower price.