We stopped discussing your "interesting" claim that a P3 is...

We stopped discussing your "interesting" claim that a P3 is needed?
No one said KK didn't say it, and the definition of the word combined with the FDA's proven example of a cGvHD drug after a P2 trial makes it clear for everyone capable of reading and comprehending that his statement is a factually correct.


Why am I against it?
Without seeing the data and how compelling (or not) it is, I'd rather have a robust data set than a questionable MoA and post-hoc analysis that delays a possible approval pathway. That is not just a slap in the face for the company and its investors, but also all the patients that were hoping to have access to a possibly life saving therapy.
If they want to have a chance for a P2 approval, they must first file for accelerated approval, something they have discussed with the FDA early on back in 2017. Unless we see them filing for RMAT once data in HR-aGvHD adults becomes available, I don't see KK even trying to go for approval straight after P2.

Now coming back to being impeded and something that made me question if MSB approval impacts CYP - right until Orphan Drug Designation was granted. I made various (unpopular) comments regarding it possibly having an impact when I checked the Organ Drug Register:

MSB called dibs on aGvHD using ex vivo cultured adult human mesenchymal stem cells back in 14/12/2005
https://web.archive.org/web/20220120125459/https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=214805

My concern was, especially given the court case I have quoted many times on here, does that cover "all" of aGvHD? Does it also cover adults?

Guess, what, since approved, it now says:

https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=214805

Interesting reading the added "Approved Labeled Indication," no?
This is where before opening your trap on HC or chime in with the ones the bottom feeder calls "someone smarter than me" should check the Code of Federal Regulations after reading your beloved announcement. I'll try and keep it simple and limit it to the first paragraph:
"[...] announced that it has received seven years of orphan-drug exclusive approval from U.S. Food and Drug Administration (FDA) for Ryoncil® (remestemcel-L) for treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older."
https://investorsmedia.mesoblast.com/static-files/767c14c2-0568-4dd5-ae8d-fbcdc42ec88b"orphan drug exclusive approval"
Instead of using TipRankGPT, perhaps visiting the source might make more sense:
Code of Federal Regulations
https://www.ecfr.gov/current/title-21/chapter-I/subchapter-D/part-316

"Orphan-drug exclusive approval or exclusive approval means that, effective on the date of FDA approval as stated in the approval letter of a marketing application for a sponsor of a designated orphan drug, no approval will be given to a subsequent sponsor of the same drug for the same use or indication for 7 years, except as otherwise provided by law or in this part. A designated drug will receive orphan-drug exclusive approval only if the same drug has not already been approved for the same use or indication."
https://www.ecfr.gov/current/title-21/chapter-I/subchapter-D/part-316

Same drug, same use, same indication.

Ignoring the fact that MSB has not invented MSCs and does not own MSCs, what is the use and indication protected for 7 years?

But but but?
"§ 316.2 Purpose.

The purpose of this part is to establish standards and procedures for determining eligibility for the benefits provided for in section 2 of the Orphan Drug Act, including written recommendations for investigations of orphan drugs, a 7-year period of exclusive marketing, and treatment use of investigational orphan drugs. This part is also intended to satisfy Congress' requirements that FDA promulgate procedures for the implementation of sections 525(a) and 526(a) of the act."

What is the exclusive marketing and (exclusive) treatment use protected here you think?
Again, here is what MSB announced, "seven years of orphan-drug exclusive approval from U.S. Food and Drug Administration (FDA) for Ryoncil® (remestemcel-L) for treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older."

What is CYP going after (something Prochymal tried and failed with protocol 265):
"adults with newly diagnosed high-risk aGvHD."
https://cynata.com/announcements/6960865Stick to your Ireland research to discuss, and ignore the things that actually matter. Same old same old with the bottom feeders. Selective reading, selective comprehension, selective discussion.

Go Ireland!

I'm sure there was a lot more witty and interesting findings in your post, all in an attempt to shift the focus away from things that matter, yet clearly you have no idea about and that's why Ireland is your go to, or the skip P3 comment. Anyways, you lost me after "Originally posted by pfeifer1982," as that was the only true statement of value in your post.