Orphan being discussed AML/MDS/JMML

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rccola335
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Orphan being discussed AML/MDS/JMML

Post by rccola335 » Fri Sep 04, 2020 2:03 am

the agenda just posted - that month said 30 day discussion and today it said 60 day discussion
https://www.ema.europa.eu/en/documents/ ... ing_en.pdf

biopearl123
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Re: Orphan being discussed AML/MDS/JMML

Post by biopearl123 » Fri Sep 04, 2020 5:57 am

rccola335, Thanks for posting this. What is curious to me is why and how the Pediatric committee is doing the reviewing when to my knowledge all of the hematologic studies have been done on patients over the age of 18. The only pediatric study I can remember was related to brain tumors and we know how that went. Even so it appears that discussions are ongoing at the EMA per your post. Looking at earlier committee recommendations, it looks like 120 days is the magic number. The absolute numbers of cases in the EU of juvenile AML, MDS and JMML must be relatively small. (But it would at least be a start!). Where are the adult discussions? It is a real problem not understanding the approval process and why the emphasis seems to be on pediatric patients. Do you know if perhaps the Pediatric Committee is in charge of all Orphan Drugs? Is it a protocol issue? I can't make sense out of it. Would appreciate your thoughts. bp

biopearl123
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Re: Orphan being discussed AML/MDS/JMML

Post by biopearl123 » Fri Sep 04, 2020 5:57 pm

Incidence of MDS in pediatric age group: 1-4 per million. Population of Europe: 741 million. High end estimate of pediatric cases 4 x 741= 2,964. Low end estimate of cases 741. Orphan indeed. bp

cheng_ho
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Re: Orphan being discussed AML/MDS/JMML

Post by cheng_ho » Fri Sep 04, 2020 6:39 pm

What they're discussing is waiving the pediatric testing requirement, as you'll remember was done for the Phase 2.

This is a very good thing, it will speed up the trial... and as biopearl points out, incidence in children is super rare.

kmall
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Re: Orphan being discussed AML/MDS/JMML

Post by kmall » Fri Sep 04, 2020 10:21 pm

Andrew, as you stated earlier, it's a start. Perhaps pediatric is a prerequisite for MA? If it's safe and effective for children then maybe logic or data dictates efficiency in older patients? Would love to get some insight on the EU approval process in general and if this has any bearing for broader approval from someone who actually has knowledge in this arena. Thanks for bringing this to our attention rccola335.

rccola335
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Re: Orphan being discussed AML/MDS/JMML

Post by rccola335 » Sat Sep 05, 2020 12:03 am

the large ATM is only explained by expansion - 3 statistician's - the recent patents - going for Orphan for pediatric AML enforces that plan for for for AML for adult - the amount of capital potentially being raised would be needed for going for the whole enchilada - the full heme spectrum plus the possibility of solid tumors - expanding the field increases the value for a potential sale

rccola335
Posts: 58
Joined: Sat Sep 28, 2019 10:00 pm

Re: Orphan being discussed AML/MDS/JMML

Post by rccola335 » Sat Sep 05, 2020 12:04 am

the large ATM is only explained by expansion - 3 statistician's - the recent patents - going for Orphan for pediatric AML enforces that plan for for for AML for adult - the amount of capital potentially being raised would be needed for going for the whole enchilada - the full heme spectrum plus the possibility of solid tumors - expanding the field increases the value for a potential sale

CKTC
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Re: Orphan being discussed AML/MDS/JMML

Post by CKTC » Sat Sep 05, 2020 6:53 pm

@biopearl123

Without having to research this ( which I have no interest in doing), I think what you are seeing is the PDCO notice of a discussion on Geron’s application for a pediatric investigation plan (PIP) wavier for imetelstat in MDS/AML. Janssen received a similar waiver for imetelstat in MF (EMEA-001910-PIP-15) back in 2016. This particular application is EMEA-001910-PIP03-20. The reason the notice reads Imetelstat – Orphan, is not because the application is part of the orphan designation process for MDS/AML, but because imetelstat is already an EMEA orphan designated drug (MF).

The Pediatric Committee (PDCO) reviews the applicant’s PIP. The PIP specifies a drug’s development plan within the pediatric population. If the PDCO determines “X” number of children will be using the medicine, it will recommend clinical studies be conducted in this population. If few children are expected to use the drug, the PDCO will recommend a waiver be granted. I expect the PDCO will grant a waiver for MDS/AML.

biopearl123
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Re: Orphan being discussed AML/MDS/JMML

Post by biopearl123 » Sat Sep 05, 2020 11:27 pm

CKTC and Bill, thank you for clarifying. The PDCO process seems to be a little more public than the actual approval process. For example, I don't think we knew that orphan designation was under discussion until the PR, I imagine the same would be true for MA, if it is even under any consideration at this point. Getting the waiver seems pro forma at this point. What we don't have is any window into the actual market approval process itself. Again, thanks, bp

mistergern
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Re: Orphan being discussed AML/MDS/JMML

Post by mistergern » Sun Sep 06, 2020 1:54 pm

Scarlett has become a believer in having an ATM at the ready. Unfortunately Geron's history would have been considerably less painful if Greenwood & Okarma had an ATM ready for the run to $73! Hopefully Scarlett is expecting a significant spike in the near future.

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