Hi Rebecca, it has always been the case that when a drug...

Hi Rebecca, it has always been the case that when a drug exhibits clear superiority, that the trial can be shut down and the placebo cross over to the drug arm. Example: In the case of the Apex trial in Multiple Myeloma. It was halted in 2004 when superiority of Bortezomib over the standard chemo was established. The placebo was offered the new drug. That was just the first example I found. Another early myeloma drug was approved out of p2
Orphan drug aside, ATH434 has a low bar of nothing to prove superiority over. The sticking point may be that not enough patients, total, have been treated to demonstrate safety to the FDA satisfaction. Not sure of the numbers required there. I get the feeling Alterity expect to be doing a P3 next year.
I did say in my post that this could be approved out of p2, if the 201 demonstrates superiority over the placebo. Halting, slowing or reversing progression would all be spectacular, considering there are no currently approved treatments for MSA. IMO
What I should have said is that the burden of proof/evidence for early approval is lower for rare/orphan life threatening diseases. I know what the website says. For instance they will consider smaller trials and in some cases even accept biomarkers as evidence. The situation now with MSA patients is they go along with the arrays of tests to confirm the diagnosis, but even when the diagnosis is complete, there is nothing else to do but monitor the progression of symptoms till all body functions are lost. ATH434 is urgently needed, if it works.