FDA 关于 PTCT 的药物申请的评论和投票。

FDA 关于 PTCT 的药物申请的评论。

大意就是公司把原来的资料重新整理，往一块凑了凑，没有新的东西。

 

我的感觉他们生不逢时， 其实他们的结果和SRPT的差不多， 就是有那么一点点意思， SRPT被批了， 他们可能不服，就再纠缠上诉。严格说都不改批准。

Conclusion

Ultimately, no positive results from any prospectively planned analyses that are persuasive have been provided with this application. The applicant has presented only the results from numerous post hoc and exploratory analyses that are intended to mitigate two negative clinical trials. In 2011, the applicant claimed that the effectiveness of ataluren had been established based on the post hoc analyses of the ADP population in Study 007. However, when this conclusion was prospectively evaluated in Study 020, the results were clearly negative. This finding directly highlights the frequently misleading nature of exploratory analyses of negative trials. It is arguable that some trends observed in the applicant’s data may warrant further prospective investigation, which the Agency has consistently encouraged the applicant to consider. Even so, for the reasons discussed above, it seems quite possible that any future study designed based on exploratory analyses of Study 020 will also turn out to be negative, just as Study 020, which was based on exploratory post hoc analyses from Study 007, was negative. The analogous results from the applicant’s development of ataluren for the treatment of nmCF offer a similar cautionary tale. Overall, the data intended by the applicant to establish the effectiveness of ataluren for the treatment of nmDMD are not persuasive.

。。。。。。

1. VOTE: Has substantial evidence of a treatment effect for apaziquone over placebo been demonstrated?

Voting Results YES: 0 NO: 14 ABSTAIN: 0

Committee Discussion: The committee noted that this drug may have activity in patients with NMIBC but based on the data that was presented, it was unanimously agreed that substantial evidence of efficacy had not been shown. One statistician on the panel stated that the sponsor did not meet their primary endpoints in both studies, 611 and 612, and that the subgroup analyses were ad-hoc and could lead to potentially biased estimates of the treatment effect in the subgroups of interest. In addition, it was commented that the pooled analysis of the two studies didn’t have a prospective protocol. The pooled analysis of 611 and 612 was done post-hoc and doesn’t provide the same level of statistical certainty or robustness as the two separate trials would have. Also, the committee noted there was a substantial amount of missing data and that this may have impacted the estimated effect. The committee recommended that the sponsor continue the development of this drug because of the unmet need in this population and the seemingly low toxicity profile of apaziquone compared to current therapy. One committee member noted that to encourage development in nonmuscle-invasive bladder cancer that the urologic community needs to further define appropriate endpoints for clinical trials. Please see the transcript for details of the committee discussion. 2. DISCUSSION: For those who voted “yes” to question 1 that an effect has been demonstrated, please discuss the clinical meaning of the results of studies 611 and 612. Committee Discussion: The unanimous vote of NO to question 1 precluded the need for this discussion. Please see the transcript for details of the committee discussion. The meeting was adjourned at approximately 11:57 a.m.

https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/PeripheralandCentralNervousSystemDrugsAdvisoryCommittee/UCM577349.pdf

 

所有跟帖： 

• 这是啥时候的评论？为啥今天跌了又涨上来呢？ -wolf21- ♂ (0 bytes) () 09/26/2017 postreply 11:01:12

• 今天早上刚出来的，回升的也莫名其妙， 估计还得跌，有可能 到10以下。（加上了原文链接） -zd3y- ♂ (0 bytes) () 09/26/2017 postreply 11:05:04

• 希望跌到5块左右，想想总可以把 -wolf21- ♂ (0 bytes) () 09/26/2017 postreply 11:06:29

• 这PTCT也有一夜暴跌73%的历史，快两年了，只cover一半loss。技术上不行嘛！ -Laoxisi- ♂ (0 bytes) () 09/26/2017 postreply 12:20:32