Ann: FDA Accepts Mesoblast's BLA Resubmission for Remestemcel-L, page-84

I know some will believe this is a simple administrative tick by the FDA.. but I believe the FDA had every right to send it back as an incomplete response if they considered that the bare minimum requirement would be an RCT. The fact they accepted it tell us that this is not the case, and that whatever response provided addressed the underlying reason why the FDA had the RCT recommendation i.e. efficacy, and now they will assess the adequacy of that response.

On the back of a successful phase 3 single arm trial, ODAC 9-1 vote, existing approved products having no benefit on survival (new information since BLA/ODAC), better understanding of SR-aGVHD and outstanding long term survival benefits of Ryoncil... not to mention the impeccable safety profile. If the CMC issues are dealt with adequately ... then the FDA now have to weigh up the benefit & risk of approving Ryoncil.

The biggest risk is manufacturing related IMO ... that is, MSB not being able to adequately assure the FDA that each lot will have the same 'active ingredient'. If that is addresssed, then there really is no other risk they can stack up against the benefits (albeit not gold standard, RCT)... but with no other treatment available for children, a failing approved product for adults... what does the FDA have to lose by approving Ryoncil?

Looking ahead...

- End of May investor call; any hint of an inspection at Lonza Singapore.. and this will get very interesting
- CHF meeting next quarter
- CLBP phase 3 trial to commence mid year

goodluck all, I'm off to enjoy the day