EU orphan status

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rccola335
Posts: 54
Joined: Sat Sep 28, 2019 10:00 pm

Re: EU orphan status

Post by rccola335 » Wed Jul 01, 2020 7:29 pm

this has the potential to lead to conditional approval - this is what Janssen was likely expecting and why they advertised for pricing managers in Europe for imetelstat for AML and MDS

biopearl123
Posts: 409
Joined: Fri Jul 20, 2018 5:13 pm

Re: EU orphan status

Post by biopearl123 » Wed Jul 01, 2020 10:24 pm

rccola335, I confess to not knowing enough about the EU approval process, can you elaborate? It does seem that orphan status was granted later than in the US. Made me wonder if they wanted to "start the clock" as late as possible as to extend the potential benefits. bp

rccola335
Posts: 54
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Re: EU orphan status

Post by rccola335 » Wed Jul 01, 2020 11:17 pm

https://www.ema.europa.eu/en/human-regu ... n-overview

talks about conditional approval and compassionate use - if you remember in August of 2018 Janssen placed imetelstat on their compassionate use list and pulled it off when it was noticed - claimed it was a mistake - companies like janssen don't accidentally place drugs on their compassionate use list - the science side of Janssen was ready to move ahead but the business side didn't make it happen

biopearl123
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Re: EU orphan status

Post by biopearl123 » Wed Jul 01, 2020 11:45 pm

rccola335, thanks for the link. My sense is that Geron has little interest in fostering compassionate use (to their discredit). But the following from the link you kindly provided is intesting:

"Conditional marketing authorisations may be granted if the CHMP finds that all the following requirements are met:

the benefit-risk balance of the product is positive;
it is likely that the applicant will be able to provide comprehensive data;
unmet medical needs will be fulfilled;
the benefit to public health of the medicinal product's immediate availability on the market outweighs the risks due to need for further data."

If only our own FDA would allow the same. Sure looks like all the boxes are checked. Thanks, bp

biopearl123
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Re: EU orphan status

Post by biopearl123 » Thu Jul 02, 2020 12:24 am

Considering that orphan designation was granted for MF in Dec of 2015 without subsequent market authorization one can only hope the EU will grant MA for both indications (finally) based on the link provided by rucola335. Of course I thought we would see positive movement five years ago when the MF orphan status was granted.

"On 14 December 2015, orphan designation (EU/3/15/1593) was granted by the European Commission to Janssen-Cilag International N.V., Belgium, for imetelstat sodium for the treatment of myelofibrosis."

rccola335
Posts: 54
Joined: Sat Sep 28, 2019 10:00 pm

Re: EU orphan status

Post by rccola335 » Thu Jul 02, 2020 12:31 am

-they get European approval and AML is back on the table and a partnership worth a premium will be had - or course a realistic buyout becomes possible also - someone buying Geron out would rather do it before they lock in a European partner to make the deal cleaner

is it being unrealistic? I always say money talks and bullshit walks - this would explain the surge in the ten dollar calls for January 2021 and the huge short exit - we have some interesting days ahead

Austin417
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Joined: Wed Oct 31, 2018 9:57 pm

Re: EU orphan status

Post by Austin417 » Thu Jul 02, 2020 3:43 am

Geron can now be seen as a pioneer that has reached previously undiscovered territory, and they have planted the flag. I can’t imagine a single major BP company not already putting the finishing touches on a BO offer.

Whether or not Imetelstat works is no longer up for debate: it has ALREADY proven its worthiness as a frontline drug. The multi-billion dollar opportunity ready and waiting for a top-3 BP company centers upon one thing: just how far, and how wide, can we take this unique MOA?

Ryan
Posts: 82
Joined: Sat Jul 08, 2017 1:41 pm

Re: EU orphan status

Post by Ryan » Thu Jul 02, 2020 9:14 am

I was just about to note that Imet received Orphan Status, for MF, about 5 years ago when BP kindly, and diligently, actually pulled the exact date and provided the facts.

This is a great thread, I can’t disagree with a word here. The only thing I can say is - anticipating a breakthrough has been a fools errand for a mind-blowing amount of time now (points in mirror).

Will this breakthrough actually be coming??? Or do we wait for 2023? It’s one or the other - it’s not IF Imet gets approved, but when...

I would be disingenuous if I said this factual statement - it would be great for patients and future patients with these truly insidious diseases. I do believe that, but I am also thinking about my investment, have to be totally honest. it’s been a long time coming, the disappointments (self-inflicted) have been countless, so I personally won’t be anticipating Conditional Approval in the near future.

So enough with the rambling.... “hat tip” to the posters on this thread as great points have been made and great information provided.

biopearl123
Posts: 409
Joined: Fri Jul 20, 2018 5:13 pm

Re: EU orphan status

Post by biopearl123 » Thu Jul 02, 2020 11:56 pm

Hi Ryan, right. Its deja vu all over again. 5 years ago I suspect Janssen thought they had an MA sown up for MF, 1. Unmet medical need 2. Requisite small treatable patient population 3. Plans for confirmatory data AND wait for it-- good PII data--NOT. As we know this one check box was not ticked and the PII data did not meet primary end points and Janssen ditched us. Now we have criterion in both MF and MDS for MA (granting orphan status is a forgone conclusion but a prerequisite for MA consideration)--this is not about orphan status but a conditional EU market approval according to the criterion set out and discussed above. Geron's definitely taking another run at it. This explains the long timelines to FDA approval, they have to for US approval. But it looks like a different pathway to approval (pending confirmatory studies) is underway for the EU. I think the FDA may come out looking not so good here. Would they force patients to travel to Europe (maybe Canada, Bermuda?) for drug? Do you agree? bp

Ryan
Posts: 82
Joined: Sat Jul 08, 2017 1:41 pm

Re: EU orphan status

Post by Ryan » Sat Aug 01, 2020 6:21 pm

Well there it is, Orphan Designation.

Seems this was delivered early in the week. Noting that the company announced there scheduled Quarterly call early in the week, moving it way up as compared to the calls in the past couple of quarters.

This is a day when we on this board need to “hat tip”, recognize and praise our friend Fishermangents who long ago recognized the potential of Imetelstat from his home in Europe. Thanks and praise!

biopearl123
Posts: 409
Joined: Fri Jul 20, 2018 5:13 pm

Re: EU orphan status

Post by biopearl123 » Sat Aug 01, 2020 7:49 pm

Ryan, I wish it were that straightforward. Get OD is a good thing but unless we see some glimmer of MA, the significance will be similar to the OD that was designated long ago in the US. For Fish’s sake and his work and thoughtfulness I hope this leads to MA in the EU soon but it’s still not a foregone conclusion. It might in and of itself help a potential partner make some rational decisions however. Regards, bp

Ryan
Posts: 82
Joined: Sat Jul 08, 2017 1:41 pm

Re: EU orphan status

Post by Ryan » Sat Aug 01, 2020 10:52 pm

Agreed BP, not earthmoving (yet), yet more progress.

And Europe moving forward, yes after all these years, is cause for celebration.

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