You raise some fair and genuine issues, which Mesoblast were at great pains to deal with in the ODAC hearing ?
Now consider the following :
1) How do the FDA standardise or countenance the use of convalescent plasma and antibody therapies ? Or are we not taking about a level playing field here ? Is every heart transplant the same , is every blood donor equal ? The standard answer they like to give is that our cells are cultured before use ? Perhaps they can explain how they have just approved a refined salmon oil (icosapent ethyl ) for reducing cardiovascular disease...is every salmon the same ? Why do half the approved vitamin products on the market pass through the body without ever being absorbed ?
2) How can they approve CART therapies..or autologous therapies when donors can have inferior potency to healthy allogenic donors...even without similar halotyping ? ...and then push back allogenic therapies with greater efficacy.
3)There is virtually no therapy on earth which is equally effective in treating all patients. It is all down to the different genomics of the PATIENT. Even when pluripotent cells are used there can be different responses to the same therapy and similar levels of efficacy to the allogenic therapy.
4) You have to start somewhere. The FDA insists on a “iterative process” with pre clinical ,Phase 1,2, & 3 trials etc., We cannot keep changing our processes so close to approval otherwise they will simply insist on restarting the whole process...Catch 22.
5) The huge joke is that most of our clinical trials have been using just one or two cadaver donors. If you look at the LVAD trial, virtually all our treated cohort of almost 100 patients came from just one donor...and yet the genomics and particular ailments of the patients , not the potency of the cell’s themselves were determining factors in efficacy. Batch variability and consistency are vitally important but Quality Assurance in areas such as cryopreservation are equally as important is assessing potency ... and completely overlooked in the discussion .
6) The FDA are out of their depth. Their lecture on population number doubling and senescence at the ODAC meeting was comical . They were lecturing the likes of Kaplan, Simmons, Gronthos, Itescu and Galipeau who helped write the breaking research on the subject 15 ? years ago. Mesoblast is a commercial company that has spent hundreds of millions perfecting techniques that the FDA would not have a clue about. FDA did not even seem to be abreast about the latest research in to cell potency or quality potency attributes. I could advise the FDA on better procedures and i am not even working in the industry .
7) Ryoncil addresses an unmet need. Medicine is a continuous improvement process. The FDA are allowing therapies that have black box warnings all over the place.....and yet they want Mesoblast to show what precise mixture of 15 different immune pathways allow their perfectly safe and efficacious therapy works .
8) However many times Mesoblast explained about the evolution in the potency of the Ryoncil therapy...the FDA refused to acknowledge how the process had approved between the various clinical trials....which is why they are now insisting on a further trial...they even disputed the retrospective matching of patients undertaken by the Magic Consortium made up of world experts and questioned why a null hypothesis in the mid 40s was used, when they did not make similar objections to other approved treatments fo the same condition .
Lastly, you ask what appears a perfectly reasonable question .
“What if the 30% who died did so because the dose was not as effective as it was supposed to be ? “
Perhaps the FDA should ask the same of Jakafi when administered to a 12 year old with far less efficacy !
Perhaps you should ask the parents of children with srGVHD if they agree with the panel of experts, that Ryoncil is an acceptable risk to take when considering the mortality rate on the current standard of care. We are talking about very tiny batch variability which has produced very similar efficacy in three clinical trials and the EAP. When a therapy has stood the test of time over 10 years like Remestemcel (Ryoncil) its time to apply common sense ...sadly this is plainly lacking in the FDA right now.
Please do not rely on the facts or opinions expressed in the above post when making an investment decision. OP
MSB Price at posting:
$3.34 Sentiment: Buy Disclosure: Held
(20min delay)