Ann: FDA Supports Accelerated Approval Pathway for Heart Failure, page-118

MSB has placed the FDA in a very difficult situation. The Phase 3 trial failed to meet the MACE primary endpoint which means the far more clinically meaningful overall survival secondary endpoint can’t be analysed statistically. Without statistically supported data the FDA really, really doesn’t like to give accelerated approval outside of ultra-orphan indications. I can’t think of any example where the FDA has given accelerated approval in a situation like MSB. If only MSB had followed the FDA’s advice and included OS in the primary endpoint.

My best guess is the FDA is quite intrigued with the OS results, but they will ask MSB to run another trial before considering accelerated approval. With luck this will only be a medium sized Phase 2b, but I am not sure MSB has the investor runway to do this. I certainly hope I am wrong.