Ann: FDA Notifies Clinical Data Sufficient for Refiling aGVHD BLA, page-462

@@Wilba32 We will find out about a month after they resubmit, as the FDA will only accept the resubmission if they believe the applicant has addressed all outstanding CRL issues.

The FDA told Mesoblast in the Type A meeting that they would need more potency assay data before approving Ryoncil, and that if Mesoblast couldn't produce data from a potency assay that existed in the phase 3 trial that met the FDA criteria, then Mesoblast would need to conduct another trial.

As such, for the FDA to accept the resubmission.. Mesoblast must provide data from a potency assay that existed in the phase 3 trial. Or else the FDA would reject the resubmission and request that another trial be performed.

Lets remind ourselves what the FDA have asked Mesoblast to demonstrate this time around. Firstly, because there was no issue raised on efficacy or safety .. the FDA is comfortable that the phase 3 trial demonstrated efficacy, and that the product is safe.

The final piece of the puzzle for the FDA is that they want to ensure that the inventory Mesoblast have on hand intended for use post approval, is substantially the same / just as potent or effective, as the product used in the phase 3 trial.

At this stage of the review process (single unresolved issue), Mesoblast must have a very good idea of what the FDA actually want to see i.e. the criteria they must meet. If the FDA have been shown this data, and have advised Mesoblast that it appears to be sufficient for re-filing, it must be what the FDA were looking for. In fact Silviu said in an interview, that he thinks that the data they have in hand more than meets the criteria set by the FDA (suggest you listen from around 15 minute mark, but above references the 15m50sec mark)


https://youtu.be/HBGR6ZAQPPA?si=W2VB_buIRmYxWK6z

S_cafe interview