Hi Cathy, I think the questions you posed on YMB are good ones and perhaps some further discussion is warranted. The following is my interpretation only.
The points that Dr. Scarlett and Dr. Rizzo made deserve emphasis. I think they are good news indeed but how that news is disseminated and interpreted and valued remains to be seen.
1. Reversal of fibrosis in MF is correlated with longevity and improvements in symptoms. This has not be described previously anywhere in the medical literature (except perhaps in a preliminary way in Dr. T's NEJM article) and anywhere in the world.
2. Imetelstat has demonstrated transfusion independence durability not described previously in the medical literature anywhere in the world.
3. Anywhere in the world--I think that's the take home message.
Additional points:1. The ET study showed profound effects in virtually all patients (Boerlacher, NEJM) . 2. There is preliminary evidence for drug activity in PV, high risk MDS and AML (internal file data).
Hope that helps, I am watching for your registration request. Regards, bp
Message to Cathy
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Re: Message to Cathy
Hello Andrew and thank you for getting me on this board. I am not sure I have much to contribute but I do have a lot to learn about this fascinating drug.
Also thanks for addressing some of my points over at the YMB. Your answers are very helpful.
I would add one point from the recent CC that I think is very important and was not picked up much. That has to do with the CMC module as part of the NDA procedure. The validation of this module is often the stumbling block for many a BLA/NDA approval and can take years even under favorable conditions.
This point was brought up in the CC and the answer was that as Geron outsources its manufacturing the compatability assessment for commercialization of the clinical stage drug is constantly being appraised. In other words, the CMC module is constantly being developed during the clinical trials and will be ready to go when the NDA is submitted. That is really huge news.
Again thank you and hello kmall - I hope you have been well.
Cathy
Also thanks for addressing some of my points over at the YMB. Your answers are very helpful.
I would add one point from the recent CC that I think is very important and was not picked up much. That has to do with the CMC module as part of the NDA procedure. The validation of this module is often the stumbling block for many a BLA/NDA approval and can take years even under favorable conditions.
This point was brought up in the CC and the answer was that as Geron outsources its manufacturing the compatability assessment for commercialization of the clinical stage drug is constantly being appraised. In other words, the CMC module is constantly being developed during the clinical trials and will be ready to go when the NDA is submitted. That is really huge news.
Again thank you and hello kmall - I hope you have been well.
Cathy
Re: Message to Cathy
Hello Andrew and thank you for getting me on this board. I am not sure I have much to contribute but I do have a lot to learn about this fascinating drug.
Also thanks for addressing some of my points over at the YMB. Your answers are very helpful.
I would add one point from the recent CC that I think is very important and was not picked up much. That has to do with the CMC module as part of the NDA procedure. The validation of this module is often the stumbling block for many a BLA/NDA approval and can take years even under favorable conditions.
This point was brought up in the CC and the answer was that as Geron outsources its manufacturing the compatability assessment for commercialization of the clinical stage drug is constantly being appraised. In other words, the CMC module is constantly being developed during the clinical trials and will be ready to go when the NDA is submitted. That is really huge news.
Again thank you and hello kmall - I hope you have been well.
Cathy
Also thanks for addressing some of my points over at the YMB. Your answers are very helpful.
I would add one point from the recent CC that I think is very important and was not picked up much. That has to do with the CMC module as part of the NDA procedure. The validation of this module is often the stumbling block for many a BLA/NDA approval and can take years even under favorable conditions.
This point was brought up in the CC and the answer was that as Geron outsources its manufacturing the compatability assessment for commercialization of the clinical stage drug is constantly being appraised. In other words, the CMC module is constantly being developed during the clinical trials and will be ready to go when the NDA is submitted. That is really huge news.
Again thank you and hello kmall - I hope you have been well.
Cathy
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biopearl123
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Re: Message to Cathy
Hi Cathy, welcome and thanks for posting. I agree the FDA is and will be scrutinizing the manufacturing process and this is often a stumbling block to final approval. I think it is inescapable that some major portion of the supply line is linked to Asia, perhaps China. (Kmall is the expert here), I think Scarlett et al are trying to reassure those who think so that everything is intact and even if it were interrupted due to COVID that enough drug is available on the shelf now to continue the studies. Having this high quality team with plenty of experience with the previous Janssen supply line should be reassuring. I suspect much of the previously established supply chain set up pre and intra Janssen remains intact so they didn't not have to recreate the wheel. One hopes. bp