Ann: Mesoblast Resubmits BLA with FDA for Ryoncil Approval, page-187

Here is Phil Krause involved in BLA timelines at a very senior level within the FDA. Note timelines- safety- efficacy - further reviews required etc etc- one would think that Phil K is across the resubmission in every final minute detail. My plan B is to ring Cameron and ask what he thought he heard Si say.

@Phaedrus Happy Birthday- you will like this one
https://judiciary.house.gov/sites/evo-subsites/republicans-judiciary.house.gov/files/evo-media-document/2024-06-24%20Politics%20Private%20Interests%20and%20the%20Biden%20Administrations%20Deviation%20from%20Agency%20Regulations%20in%20the%20COVID-19%20Pandemic.pdf

When Pfizer filed a BLA, and the Biden FDA decided to grant priority to its review.

Although the BLA “was longer than [they] thought,” Dr. Krause explained that the normal
prioritized BLA review would have set an “action due date” (ADD) for approval at about
January 18, 2022.104 After this initial review, Drs. Gruber, Krause and Marks initially agreed to
speed up the process with a target ADD of mid-October 2021,105 which would have eliminated
three months from the typical priority BLA approval.106 Dr. Marks subsequently changed course
and asked that the ADD be moved up another month, to September 15, 2021, telling Drs. Gruber
and Krause that mid-October would be “taking too long.”107

Drs. Gruber and Krause both testified to the Subcommittee that they felt pressure to rush
the review for the licensing of the Pfizer vaccine despite the need for further review related to the
efficacy and safety of the vaccine.112 Dr. Gruber explained that the risk of myocarditis in young
men was “evident” under the EUA, and that risk required close evaluation under the higher BLA
review standards.
113
Nonetheless, the Biden Administration decided to push the approval process for an earlier
completion date. Dr. Marks went back to Drs. Gruber and Krause and explained that they would
“need” to complete the review faster than the September 15 target date.114 In a separate email on
July 15, 2021, Dr. Marks told Acting Commissioner Woodcock that Drs. Gruber and Krause
were “intransigent at this time on the Sept[ember] 15 date.”1


Now check this out - side note Phil's counsel was his brother who works at the US patent office!
6 U.S. HOUSE OF REPRESENTATIVES,
7 WASHINGTON, D.C.
8
9
10 INTERVIEW OF: PHILIP KRAUSE, M.D.
11
12
13 Thursday, September 7, 2023
14
15 Washington, D.C.

23 EXAMINATION
24 BY :
25 Q Dr. Krause, are you currently employed?
Appendix 142


10
1 A Yes. By myself.
2 Q Okay. And what is the name of your business?
3 A It's called Logics.Bio, LLC, but it's a consulting business.
4 Q Okay. You said Logics.Bio?
5 A Yes.
6 Q LLC?
7 A Yes.
8 Q Do you also work with Mesoblast, Incorporated?
9 A Yes. I'm on the board of directors of Mesoblast.
10 Q Okay. And how long have you been working in your consulting business?
11 A Since I left the FDA.
12 Q And how long have you been on the board of directors for Mesoblast?
13 A I don't remember exactly when I started, but a year, give or take.
14 Q Okay. And what is your title with Mesoblast?
15 A I'm just a member of the board of directors.
16 Q Okay. And what are your responsibilities on the board?
17 A Well, as a member of the board of directors of any company, I help provide
18 direction to the company in strategic matters, as well as obviously oversight of what the
19 CEO is doing.
20 Q And in your consulting business, Logics.Bio, LLC, what kind of work do you
21 do?
22 A I do consulting for companies that are interested in developing biological
23 products. I also do consulting for the World Health Organization. Yeah, those two things.
24 Q And did you work anywhere between your consulting business and
25 Mesoblast and the FDA?
Appendix 143


11
1 A No.
2 Q When did you first join the FDA?
3 A I joined the FDA in 1991. I did not bring a copy of my CV, and so I -- I've been
4 admonished not to guess if I can't remember something, but I'm pretty sure 1991 is right.
5 Q Completely okay, give or take.
6 A Yes.
7 Q And what made you want to work at the FDA?
8 A The FDA is an important public health agency. The FDA also offered me a
9 position as a research scientist where I could run a research laboratory, and gave me the
10 resources necessary to do that, and of course, it was appealing.
11 I trained at the National Institutes of Health and so was already in the area. So it
12 was appealing not to have to move. And so these were all factors that made it a good
13 place for me to start my career.
14 Q And I know you'd mentioned -- you said not having your CV in front of you.
15 To your best recollection, can you walk us through the positions you've held with the
16 FDA?
17 A I can. Would you mind if I gave you a little bit of background what I did
18 before I got to the FDA?
19 Q Absolutely.
20 A Because that will put things in context perhaps.
21 Q Of course.
22 A So I grew up in Urbana, Illinois. I received a -- well, I ultimately went to
23 medical school at Yale. I became board certified in internal medicine and in infectious
24 diseases.
25 I then did training in virology at the National Institutes of Health, and then ended
Appendix 144


12
1 up going to the FDA from there.
2 While at the FDA -- I'll give you the big picture first, and if you need more detail,
3 I'm happy to provide it -- I worked in a number of different capacities, ranging from
4 running a laboratory to being the deputy director of the Division of Viral Products --
5 actually, being the acting director of that same division for a year, being in the Office of
6 Vaccines Research and Review, the associate director for vaccine safety and medical
7 policy.
8 Then ultimately in around 2013, the deputy director of the Office of Vaccines,
9 which is the position I held until I left the FDA.
10 During the COVID pandemic, I was the -- the highest-ranking infectious diseases
11 physician in the Center for Biologics. During the COVID pandemic -- well, and while at the
12 FDA, I published over 100 peer-reviewed articles on topics that ranged from vaccinology,
13 virology, epidemiology, vaccine safety, and even biostatistics.
14 While at the FDA during the COVID pandemic, I also was assigned as a liaison from
15 the Office of Vaccines to the WHO, and in that capacity, very soon after the pandemic
16 began, the World Health Organization made me the chair of their expert working
17 committee on COVID vaccines, which entailed running frequent meetings on the topic of
18 COVID vaccine development, helping to coordinate international and WHO scientific
19 responses in addition to the work that I was doing for W -- or for FDA.


13 Q And is there a reason -- and there might not be, but is there any significance
14 to you being the acting director, not just the director? Like, did you not hold that position
15 the entire time that that working group existed?
16 A Oh, I'm sorry. I think you must've written something down wrong. So I was
17 the chair of the --
18 Q Oh, the chair.
19 A -- committee for WHO. I was the acting director of the Division of Viral
20 Products at FDA for a year or so. And I would guess this was around 2009 or 2010, but I
21 don't remember exactly what year that was.
22 Q I did get that mixed up. Thank you. Sorry. You've held a lot of positions.
23 And primarily, it sounds like you did work on virology, immunology, and
24 vaccinations. Is there any other science subject matter that I missed.
25 A Well, clinical trials.
15
1 Q Clinical trials.
2 A So I'm viewed as -- I'm viewed as -- I'm fairly well-known as an expert around
3 the world in all those topics.


19 Now, of course for BLA, one also would've needed the manufacturing data -- the
20 chemistry, manufacturing, and controls data, and that data wasn't at a level during the
21 original emergency-use authorization that one would've wanted during a BLA either.
22 And so, the initial emergency use authorization then, just to summarize, actually
23 had the level of efficacy data that one was either equivalent to or close to, but I would say
24 equivalent to that, which one would normally want for a BLA, because we had a very high
25 standard in the efficacy study.
Appendix 162


30
1 It did not have quite as much safety data as one would want for a BLA, but it had
2 enough safety data that one clearly determined that the benefits, you know, substantially
3 outweighs the risk, it turned out.

How much extra data did we provide- until they said appears sufficient- refile
For a BLA, FDA always reviews the individual data and is in general not so trusting
10 of published papers because they can't see the data.
11 And there are many cases where FDA has seen published papers that are
12 published even in very prestigious journals and have rejected some of the conclusions
13 from those papers when they've had a chance to actually see the data.

The paper is over 600 pages- Phil Krause interview touches on potency, licensing, marketing approval, EUA's vs BLA's- standards
required- etc etc.

What a person to have on board - personally liaising with the FDA for kids SR a GvHD- quite remarkable.

The best bit from one of the worlds elite health officials
"öbviously oversight of what the CEO is doing"



Bring it on - Narch Bupwards


Reg