@reginaldpHi Reg. Firstly, you have certainly upped your game...

@reginaldp

Hi Reg. Firstly, you have certainly upped your game and I would like to thank you for your recent posts.

Secondly, i would like to say that my understanding of the BLA process, is that the FDA normally allow 15 BUSINESS days for formal responses to 483 letters within the 6 month PDUFA timetable ….which implies that Mesoblast may get the answer to its BLA resubmission (all other things being equal), somewhat earlier than August 2nd 2023.
Thirdly, in terms of reimbursement, I think there is an excellent case for Ryoncil pricing similar to NKymriah. I am currently minded to model at $550-600k per treatment for paediatric dosing. Mesoblast of course has already indicated that it may want to use a price per vial strategy. If they subsequently seek to extend the label to only high risk (Grade C/D) adults sr aGVHD…the implication guided by price per vial, is a price tag of over a million for adults because of the average weight per adult patient relative to the mean paediatric patient. This would be justified bearing in mind Rux only achieves decent efficacy in Grade B patients in terms of terms of long term survival.
Fourthly, is you look at hospitalisation costs extending to as high as $1.8m for treating Grade D paediatric patients … or the patient outcomes (as few as 1 in 10 survive in this ultra orphan indication category) there may be a case for bypassing steroids altogether, and prescribing RYONCIL immediately based on clinical grading and biomarkers.
Lastly, people should remember that there are many more bone marrow transplant treatments using unrelated or unmatched donors these days... which have a higher risk of rejection. Sadly, demand for BM transplants is much higher than the number of operations performed …so it is possible that greater risk will be taken matching candidates longer term if there is a credible “antidote” to the problems of graft versus host rejection. OP


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