General Comments / Chat, page-6221

Great article. Have probably read it 5 times over the past couple of years. Something that's just jumped out at me today though:



Interesting to ponder. BTD = breakthrough designation. RAC might have discussed this recently with the FDA considering the positive pre-IND guidance. The PRV is a carrot you receive after a drug is approved. BTD could expedite Zan's approval and commercialisation.

"A breakthrough therapy designation is for a drug that treats a serious or life-threatening condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies."

"The Food Drug and Cosmetic Act (21 USC 356) states that a request for a breakthrough therapy designation may be made concurrently with, or at any time after, the submission of an application for [the IND]. To maximize the benefits of the program, FDA encourages sponsors to submit breakthrough therapy designation requests by the time of the end-of-phase-2 meeting, and also before initiation of the clinical trial(s) intended to serve as the primary basis for demonstration of efficacy"

https://www.fda.gov/regulatory-information/food-and-drug-administration-safety-and-innovation-act-fdasia/frequently-asked-questions-breakthrough-therapies#:~:text=A%20breakthrough%20therapy%20designation%20is,(s)%20over%20available%20therapies.