Hi @bkj007, "If FDA has told them adult trial can't proceed...

Hi @bkj007,

"If FDA has told them adult trial can't proceed prior to BLA approval, it's a bit hypocritical as they are still happy for ongoing treatment of children in EAP."

Absolutely agree. And IMO it shouldn't feel strange at all from a Mesoblast perspective, having been burned twice. MSB wants the FDA to make definitive and enforceable statements of its approval of Remestemcel-l. That's actually the most likely scenario, as I see it.

Consider how poorly Mesoblast has been served in the past by simply expecting the FDA to do the right thing.

For example, prior to the FDA’s issuing of the CRL on 30 September 2020, the FDA decided to convene ODAC itself - it called for ODAC's expert advice. And it had affirmed every ODAC vote in favour of approval of a therapy since 2006 - there were 23 examples from 1 January 2006 to 31 May 2019, during which ODAC voted on theapprovability of therapies 61 times (excluding biosimilars,generics, and meetings involving the Paediatric Oncology Subcommittee). The FDA approved the therapy every time ODAC voted in favour of approval (30 out of 30). Understandably, the FDA did not follow the vote of ODAC in seven instances when ODAC voted against approval and the FDA ultimately approved the treatment (7 out of 30). And there was one instance of a tied ODAC vote and the FDA went on to approve the treatment.

So, consequently, the non-approval of the Mesoblast BLA as per the CRL in late 2020 i.e. in the circumstances of a positive ODAC vote (9-1) had no precedent in at least the 14 years prior to that event. Check MSB's Amended Defence in the Federal Court proceedings.

As MSB holders, we might think it wise then & wait a little longer, as Mesoblast appears to be doing, for the FDA to actually spit out its finding in hard, cold paper form that Mesoblast has done enough. See if its actions match its words.

It shouldn't take that long.

Cheers,
GLTAH