MSB to dispute FDA finding in Type A meeting, page-343

Sorry, I'll try that post again. HC editor is crap.

@Zenox

"I think approval may have also been complicated by the fact Mesoblast were applying for full approval for pediatric patients 18 years of age and under. There are currently no treatments for patients 12 and under, but there is an approved treatment for those 12-18 years. This may have led the FDA to request a randomized, controlled clinical trial and reject the option for accelerated approval. I think Mesoblast will now seek accelerated approval for patients 12 and under."

I think this a key and undervalued issue during discussions here. It was the basis for the technical regulatory argument put together by the 'scum for hire' law firm backed by Novartis. So it may be the procedural issue as management have been indicating and requires now the next lower value approval pathway to an exclusive U12 indication.

The question remains could the FDA have just approved initially with an U12 indication or was it technically not possible or inappropriate? Only the regulatory consultants can answer that one.

Just a note of caution and general comment about some of the excellent regulatory research being done by many here. I have worked in the regulatory field for many years and dealt with FDA submissions and audits. It is extremely complex, to say the least. There are the official CFR regs and then there are FDA guidance documents, various discussion and industry feedback documents, the pre amble and finally precedent decisions. Even the big boys like Novartis etc will pay up big for specialist teams of regulatory lawyers to thread the needle (if there is a pathway). It is incredibly complex especially when you want to operate outside the normal evidence requirements. The research done here has been good but it is honestly scratching the surface of issue and what may or may not be possible. Even most of the terms used in the scope of these regulations have their own extremely technical and detailed definitions which may result in the MSB case not even being relevant for that particular pathway we are focusing on.

It really is a matter of trust in the management team to execute. Yes, our common sense also does tell us a 9-1 vote is very strong and the FDA have set a huge precedent by going against here. But this also leaves us with many unanswered questions as to what was truly behind that decision and subsequently what is the most reliable and shortest path to recover from it.