Act001, a new glioma drug from Nankai University, has been recognized as a rare disease drug by FDA. The domestic clinical trial is expected to start next month

Source: on November 20, 2017-11-24, drug era, after nearly nine years of research and development by Professor Chen Yue's team of Nankai University, the anti glioma stem cell drug with the number of act001 was recognized as a rare disease drug (i.e orphan drug) by the U.S Food and Drug Administration (FDA) (see the figure above) At present, the drug has begun clinical trials in Australia and the United States Professor Chen Yue of Nankai University told "Shenwai frontier" that domestic clinical trials are expected to start recruiting patients in Tianjin Cancer Hospital next month According to Professor Chen Yue, act001 obtained the orphan drug qualification certification of FDA in advance, which means that act001's clinical application and test in the United States will receive a package of preferential policies from FDA, including fast review channel, shortened clinical trial cycle, etc If it can be listed successfully in the future, it will also enjoy preferential policies such as 7-year protection period Professor Chen Yue said that in late September, we submitted an application to the US Food and Drug Administration (FDA) for orphan drug designation, which contains the results of phase 1 clinical trials in Australia It is expected to receive a reply from FDA within 90 days, and the result will be approved within less than 2 months, and the approval date is November 8 The indication is glioblastoma multiforme, which is the majority of glioblastoma The current status is "designed", that is, it has been identified However, it has not been approved for orphan indication In the future, it is expected to be obtained through clinical trials and corresponding reviews The identification of American FDA orphan drug means that the originality of act001 has been recognized by FDA Phase II clinical trials of glioblastoma multiforme in Australia and the United States are expected to start next year It is reported that the biggest difficulty in the treatment of glioma is that it is difficult for drugs to enter the brain The concentration of act001 in the brain is 1.8 times that in the blood The concentration of temozolomide, the best drug to treat gliomas before, was only 40% of that in the blood.