Cell Therapy News/Articles, page-16791

Well.... In the FDA's own words as Si and Krause said. The FDA said they need more potency data ( not efficacy ) OR if the data cannot be obtained then conduct another trial.

We don't know exactly what the nature of the data is that the FDA are asking for, so I assume they have mentioned specific stuff, and MSB have realized they don't have that data, and need to gather it.

It might even be as simple as a site release test needing to be conducted to validate that the shelf life does not effect the potency. Whatever it is, MSB seems to have some but not all of the data, and that makes me think it might be something to do with commercial lot testing.

When I went through the history of the product over the last 2 years - there has been a change to something. It is not in manufacturing - as Si stated the shelf product is the exact same product, exact same process.

So, I have to assume it may be either a change to the release testing reagents, or the release testing cutoff values, or an additional assay being added to commercial product ( that may not have been fully applied now ) , since one of the companies press releases mentioned that changes were in place for future product, and that indicates to me whatever that change is, is not necessarily for present product.

So, I don't think real world data or intervention trial is required. MSB have commited to an adult trial, and at the end of the day, the adult trial this time will target the correct group and meet end points for approval for all patients IMO.