Ann: FDA Accepts Mesoblast's BLA Resubmission for Remestemcel-L, page-60

Good news. This has been a rolling submission and the OTP has had plenty of time to opine on the additional potency assay should it really have not been fit for purpose. A Class 2 is anything from 2 months UP to 6 months, but as so many key parts have already passed review, such as manufacturing PLI, safety, efficacy and advisory panel, I would be surprised if this is not all wrapped up by early November because the resubmission is supposed to focus primarily on the outstanding items from the previous submission plus about a month to agree labelling and post approval commitments.
The FDA , in my opinion, would have thrown out the application if their previous CMC concerns had not been addressed to a considerable extent..hence the wording in the press release, which, whilst pretty standard, is very reassuring. With new fouryearmortality data almost double that of treatment alternatives at 49% of a largely severe grade patient population, this all looks pretty academic to me. Passing the pre license manufacturing inspection makes it very difficult to argue with batch variability , purity and consistency. I would give this a 95%+ chance of approval now it has been accepted . Remember, the EAP and virtually every validated biomarker (or combinations with Minnesota high grade) back up the clinical evidence. If the FDA had any real doubts they could have just pushed Mesoblast to reapply under accelerated approval pathway…which the Company had previously offered to do !
For many exhausted holders, the temptation to exit for a few months might help them deal with the PTSD that we have all been through. Now Silviu has a PDUFA date, he can start to do proper investor meetings overseas and move on to preparing for the end stage heart submission for accelerated approval. Over the next few months the hot money will arrive and hopefully some decent new institutional holders. We are dealing with a binary outcome, but we are extremely close to the finish line now.


Please do not rely on the facts or opinions discussed in the above post when making an investment decision.