ImetelChat

We know orphan drug status was conferred in Dec. But there is a difference between orphan drug stats and orphan drug approval. Thanks to Annie we know the committee for orphan drugs (COMP) met as recently as last week but we don't know what happened. The agenda is available for review and I think the link has been posted. Imetelstat was reviewed but we don't know what came of it. Also there were not accompanying notes so I assume the material was redacted. Since the ODD was already conferred six months ago I think along with Annie that perhaps this meeting was to confer a change from "orphan drug status" to something more permanent, perhaps a recommendation for approval that I assume then goes to the EMA for final approval, this is the agenda we should watch. Can anyone get a sense for the time line at this point? How often does the EMA meet and if a recommendation did come from the COMP for approval, which I think might be reasonable to assume than we may be very close to EU approval. If we dig, we will find. Regards, bp

I am not sure what the difference is. OD approval seems to similar as 'normal' approval? Or are you aware of special tests and procedures that are needed to follow in order to get OD approval?

In December 2015 imetelstat got ODD based on convincing arguments and unanymous decision (I read that on the old YMB, but can't verify the source). Here the text of that decision:

Public summary of opinion on orphan designation
Imetelstat sodium for the treatment of myelofibrosis

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive
opinion on 12 November 2015 recommending the granting of this designation.
__________________________
Opinions on orphan medicinal product designations are based on the following three criteria:
• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary
before a product can be granted a marketing authorisation.

The link to the Public Summary: http://www.ema.europa.eu/docs/en_GB/doc ... 200889.pdf

On the EMA website itself we can read the following about ODD for imetelstat:

'The sponsor has provided sufficient information to show that imetelstat sodium might be of significant benefit for patients with myelofibrosis. The medicine works in a different way to existing treatments and early studies showed a reversal of bone marrow fibrosis, which is not achieved by authorised treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.'

http://www.ema.europa.eu/ema/index.jsp? ... 058001d12b

From this I read that in order to keep the orphan status, the initial results (i.e. reversal of fibrosis), on which ODD was based, should be confirmed at the time of market autorization (i.e. market approval). That means that the process of keeping OD status is depending of the process of market authorization. So there is not a separate process. Imetelstat will get OD approval at the moment it gets market authorization. Until then imetelstat will receive the usual benefits of prolonged patent protection and benefits on administrative costs.

Fish, right. Then why the second visit to the committee six months later, if not to confirm drug response as required by the committee? Assuming it was confirmed then can we imply market authorization is imminent? (I am trying not to jump the gun here but I keep coming back to the same conclusion that at this point approval just awaits action by the EMA.) bp

I can imagine that the EMA tries to do intermediate evaluations of non-approved ODD drugs, just to see if everything is still okay with the drug. That means that they may have received some kind of intermediate data from JnJ.

The CHMP website shows a timeline of 277 days from submission to approval for most EMA meds. The ODD may have a faster timeline but I am not sure. We know the application had to precede November by some time when ODD was considered (or I don't know if the clock starts when ODD is granted), anyway we are at the very least 180 days into the process. The CHMP does not meet in August of course because it is Europe and everyone is on vacation. The July CHMP agenda will include a report from the July COMP. I submit that a September meeting of the CHMP could include action to be taken on Imetelstat. Agree, disagree? That will probably run out the 277 day time clock. If I am way off here please so say. bp