I believe that the FDA and other regulators did ask for changes in the SAP which caused delays in calling data lock. When changes are submitted and nothing is requested in a prescribed number of days, it's a presumptive approval. Nothing in writing says it's approved, but company's can proceed based on what is proposed.
Nothing is ever 100% guaranteed when it comes to the FDA, but I believe we have one of the strongest cases I've ever seen. I'm a big believer that historical data ought to be what experimental therapeutics work against, I know the regulators don't agree, but in this case, I think the German's convinced the others it was wrong not to dose all patients, and that's what essentially the last third of the patients received. If that's right, how can they deny an approval that's based on comparing the SOC with the experimental therapeutic that's been added to it.
I have a friend who's a nephrologist, but he has a potentially deadly condition in which he's certain that a drug approved for other treatment that's in trials for his condition would be much better than the standard of care. He got in the trial, but unfortunately got the control treatment which he could determine with blood trials. He's dropped out of the trial and will be spending a small fortune to purchase the drug, which he can do off label. I feel for him as I don't think trials should be run in this manner, but he's doing what he has to, and fortunately he can afford it. In many ways it's similar to those in England who're raising funds to get treatment with DCVax-L. When your life is on the line, you do what you can to survive. People who are willing to take experimental treatment should get that treatment, the history of their diseases is generally well known and if the experimental drug does just a little better than the SOC and that's caused by a placebo effect, what is the harm in putting the additional drug in the Doctor's tool kit. As always, JMHO.
Gary
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