EMA action yesterday?

[cheng_ho]

There was a thread about this on the YMB... it looks like Marissa killed the YMBs. Maybe we'll see some of the reasonable posters here.

Anyway, there was supposed to be a committee action yesterday or today.

[Fishermangents]

I guess we need to wait for the minutes. Let's keep an eye on it.

YMB killed: given the recent level of messages that's the best thing to do. I just don't understand the new format. I don't recognize the names and there seems to haven been a selection of posts from the old board. Well, who cares....

[hiprock88]

Committee for Orphan Medicinal Products (COMP)
Draft agenda for the meeting on 11-13 July 2016

Pre-meeting list of participants and restrictions in relation to declarations of interests applicable to the items of the agenda for the COMP plenary session to be held 11-13 July 2016. See July 2016 COMP minutes (to be published post September 2016 COMP meeting).

Treatment of myelofibrosis
Action: For adoption
Documents tabled:
Draft Summary report
Notes:There has been 1 designation for this condition: EMA/OD/154/15 Imetelstat sodium

[Fishermangents]

What could 'adoption' mean here? Adoption for the agenda, or something else?

[biopearl]

If one adopts an orphan, he or she becomes a member of your family. bp

[cheng_ho]

Anne posted this on the new YMB (Marissa is clearly shorting YHOO, the board is unusable):

From Draft COMP Agenda 11-13 July 2016

Imetelstat sodium - EMA/OD/154/15

Janssen-Cilag International N.V.; Treatment of primary myelofibrosis
COMP coordinator: Frauke Naumann-Winter
Following review of the application by the Committee, it was agreed to rename the
indication to treatment of myelofibrosis.
The Committee agreed that the condition, treatment of myelofibrosis, is a distinct medical
entity and meets the criteria for orphan designation.
The intention to treat the condition with the medicinal product containing imetelstat sodium
was considered justified based on early clinical data in affected patients demonstrating the
potential to reverse fibrosis.
The condition is chronically debilitating due to anaemia, splenomegaly, extramedullary
haematopoiesis, constitutional symptoms such as fatigue, night sweats and fever, cachexia
and leukemic progression. The condition is also life-threatening with median survival of
approximately 1.3 years for patients with high-risk disease.

The condition was estimated to be affecting approximately 0.6 in 10,000 persons in the
European Union, at the time the application was made.
In addition, although satisfactory methods of treatment of the condition have been
authorised in the European Union, the sponsor has provided sufficient justification for the
assumption that the medicinal product containing imetelstat sodium may be of significant
benefit to those affected by the condition. The sponsor has provided early clinical data that
demonstrate reversal of fibrosis, which is not achieved by any authorized treatments. This is
attributed to the novel mechanism of action which inhibits the clonal expansion of malignant
cells. The Committee considered that this constitutes a clinically relevant advantage.
A positive opinion for imetelstat sodium, for treatment of myelofibrosis, was adopted by
consensus.

[Fishermangents]

I don't see this text on the 11-13 July agenda. Where did he/she got it from? However, there has been a similar text in the report of the 14 December 2016 decision on the ODD for imetelstat:

"Public summary of opinion on orphan designation; Imetelstat sodium for the treatment of myelofibrosis" (published by EMA on 29 January 2016)

link: http://www.ema.europa.eu/docs/en_GB/doc ... 200889.pdf

[biopearl]

Hi Fish, Annie has posted some very interesting and I think very pertinent material. I hope she will participate in this board, YMB is now the new tower of Babble. My question is that we know that the orphan drug designation was granted some time ago by the EU committee, does this same committee after appropriate review have the power to grant orphan drug "approval" (which I am taking to mean marketability as opposed to "orphan drug designation")? Or does a larger EMA committee have to be involved for final approval. One interpretation of the posted material is that they approved the drug with out any clear qualifiers so I think we need some parsing of the language to know exactly what happened. What are your thoughts?

[cheng_ho]

If anyone finds the best links on the EMA site for following imet's status, please post... that might make a good sticky for this board. That and a thread keeping track of all trial enrollment numbers.

OT, does anyone know anything about TAM-818, or the status of Sierra?

[Fishermangents]

Thread on trial enrollment numbers is launched. I will check how best to keep track of EMA developments. We can start a thread for that one too.

No idea about TAM-818 or Seirra.