MSB to dispute FDA finding in Type A meeting, page-106

It is definitely probable Mesoblast and the FDA entered into some type of correspondence following the ODAC meeting, but these would not have been formal or detailed discussions.

It appears the FDA went with their original stance and ignored the recommendation of the ODAC committee, expert testimony, and the clinical benefit to children. It seems the FDA simultaneously applied a low threshold for rejection and a high threshold for approval. Maybe the current political climate played a role since there has been some serious scrutiny surrounding the FDA in the last few months, and September has been a particularly bad month for approvals. Maybe the petition from Lassman Law play a part as well. Overall it seems the FDA believes Ryoncil just missed the regulatory requirements for approval.

The best way I can explain the FDA approval process is to use an example where a patient goes into a hospital to see a doctor. Normally they will see a lower ranked doctor first (let's say a trainee or a registrar), who are the first to evaluate the patient. The registrar will consult other specialists to obtain an accurate assessment of the patient (or in this case Mesoblast's BLA application) and they will make a diagnosis (approval or not). Sometimes the diagnosis will be in dispute and the case will be escalated to the consultant (highest ranking doctor) who will make a more informed assessment of the patients condition.

Mesoblast have a clear case to take to the FDA to dispute this decision and there are precedents that work in their favour. As I understand it, Mesoblast will need to submit an application containing key information supporting approval (this cannot include new information) and this will be viewed by those higher up in the chain of command who will have to apply precedents to their decision making. The key difference here is they need to apply precedents. Today's CRL leads me to believe that the FDA does not want to set a new precedent by approving Ryoncil, but following a Type A meeting they can apply other precedents to facilitate approval. As SI said today, this is just part of a process.

Should we be concerned? No not yet. Maybe if they deny the Type A meeting, then sure. But even then, we still have COVID ARDs, CLBP and Heart readouts to come - all blockbusters.