Why JB1975 should be under the impression that having a patent pending for aGVHD has not been disclosed, only he knows . He could of course read the Company website !
As for the size of a BLA application, the number of pages in a submission I can safely say is in no way price sensitive, nor would it give anyone an advantage …many applications can amount to over 500,000 pages
That’s all for now @JB1975, i hope my contributions have been factual rather than too mischievous. I have personally been a victim of delays in the regulatory process having lost substantial sums of money in the process on this share. I originally took your comments seriously but I believe you are naive if you think Mesoblast did not get formal guidance from the FDA on some of the more contentious points raised ..such as how many individual donors were required to be used in GVHD001 to provide the datasets. The main reason I am still invested (and starting to make some of the money back), is the MAGIC biomarkers COMBINED with all the clinical trial data is extraordinary, especially as we now know longer term survival rates and safety profile , allowing the FDA , should they choose, to relent and give authorisation for this first in class therapy . The truth is the FDA own 2011 potency guidance was judged by many to be not prescriptive enough and many of their decisions around single label trials were inconsistently applied. I believe the delays may well have cost hundreds of thousands of people their lives as it delayed the start of numerous clinical trials subsequently ..but maybe Silviu was pushing his luck using an orphan disease with a small dataset as the “test case”. The FDA must have looked on aghast after the New York Times reported in 2018 that Dr Piero Anversa, formerly of Harvard Medical School, had to retract 31 studies on MSC’s which contained fabricated or falsified data. None of these trials invloved Mesoblast, but it was a dark time for regenerative therapies.
Moving on , we are now awaiting validation of Ryoncil for the first of many inflammatory indications. The prize is not just sr aGVHD, HLHS,refractory Crohns or Ulcerative Colitis ….but potentially safe and efficacious therapies for three biggest causes of hospitalisations in the USA, namely ischemic heart disease, diabetes and CLBP ….not to mention ARDS responsible for 1 in 10 emergency beds in the USA often because of rapid onset bacterial and viral sepsis…or potentially COPD ! Remember the “right to try” legislation which obliges the FDA to allow physicians to use therapies for life threatening conditions or diseases, which have already completed Phase 1 trials in extenuating circumstances …Mesoblast has previously conducted an extensive historic repertoire of clinical trial data across numerous indications…so the off label use could be extraordinary…but the Company will probably need 1-2 years to partner and seriously scale up manufacturing production. The FDA needs to move fast…their prevarication and bureaucratic process has failed their citizens while other countries such as Japan , Germany and Korea are allowing these cells to be used to save patients lives. This reflects extremely poorly on the regulator …in my opinion of course . OP
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