Biotech Values

[jq1234]

ONXX: Carfilzomib

I read the critical briefing doc differently. I think almost all of the questions raised by FDA are in the context of single arm trial which couldn't accurately assess the risk/benefit analysis because all of the risks from single arm trial assigned to Carfilzomib.

I also think point #4 exemplifies the mission creep into the accelerated approval process. If the FDA wants randomized trials for accelerated approval, then we should probably just remove the accelerated approval pathway.

ODAC already said even for accelerated approval, they would like to see randomized trial. I don't agree to ask for randomized trial, it means it ends accelerated approval. Carfilzomib 266-patient trial certainly could be run as randomized trial, either with 1:1 or 2:1 vs investigator's choice, with ORR as primary endpoint. It allows better assessment of both efficacy and safety signal. I think sponsors who intend to file accelerated approval should keep this in mind. Otherwise, people can argue all day about whether Carfilzomib is worthy of accelerated approval, and won't agree.

But I don't think your comments are all that convincing considering the pathway that Velcade followed to approval. It seems to me that you would have been equally disappointed with Velcade's initial NDA application, because it was essentially identical to this carfilzomib application. I guess where you see a poorly run single arm phase 2 trial, I see a trial that is following in the footsteps of a very successful example of accelerated approval.

There is no point to compare to Velcade accelerated approval. The treatment regimen and options have changed, so has the regulatory process. Same trial same result doesn't mean approval 10 years later. I am somewhere in between of you two, I think Carfilzomib data is borderline for accelerated approval. I have no problem if it goes either way.