FDA v. Geron

[LWS]

Let's get Imetelstat over the finish line.

It is now a binary decision, either the FDA approves (most likely) or the FDA denies. Denial would be a surprise considering 12 YES ODAC votes. Also, the integrity of the FDA would be questioned. The FDA (as far as I can tell) has never rejected an ODAC positive recommendation. I see no reason for the FDA to break with the past, especially since all of the blood cancer experts (that I am familiar with) support Imetelstat.

We have a little more than 1 month (until June 16th) to find out. Hopefully, we will not have to wait that long. There may be some label restrictions, but we already know that the most talked about side effects can be managed, controlled and eliminated in a short time. No blood-cancer medicine comes close to Imetelstat in trial successes, patients' needs and combination potential going forward.
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From Hoosier Investor » Fri May 10, 2024

I expect our label will be restricted to LR-MDS patients who are R/R to ESAs. This was the original target & goal of the trial, and the trial was designed accordingly. As has been previously communicated, the trial met its primary & secondary objectives in what appears to be an acceptable manner. On top of that, we have a 12-2 ODAC vote in favor of approval. Therefore, it seems highly likely we'll gain FDA approval in this LR-MDS indication.

[pursang]

BP, is there a block or mute function on this site ?
Thanks !

[biopearl123]

You have the option to read the posts you find interesting/relevant and not to read the ones you don’t.

[huntingonthebluffs]

Again, thank you Hoosier Investor for your thoughts on if Imetelstat could be used by patients R/R to Luspatercept.

This then would become an unmet need by LR MDS patients who have become R/R while being treated with Luspatercept, even though a possible life extending benefit for many with Imetelstat is sitting on the shelf but prevented by the FDA. Since no other options exist for LR MDS patients R/R to Luspatercept as no other CTs have included this as primary or secondary endpoints including Geron I assume they just continue receiving blood transfusions and getting sicker. BTW this also prevents them from using Imetelstat via the compassionate use program, correct? Short of another trial we also need to understand the biomarkers that would indicate who would receive the most benefit but surely that wouldn’t sway the FDA either.

I guess the question boils down to what % of the patient populations have started treatment on Luspatercept including RS- patients as all those patients are apparently forfeiting their option to use Imetelstat for the foreseeable future as a new CT would likely take years. If I was in my 70’s and regularly receiving several blood transfusions over an 8 week period, it seems inhuman and I would be infuriated to be prevented from having an option to use Imetelstat with a 40% chance of becoming TI or at least a significant reduction in RBCTs and the improvement in hemoglobin, fatigue, etc. I hope there are other paths / options open to these patients but not likely.

Maybe I am being ungrateful here, assuming that we get any approval but seems inappropriate to restrict something potentially life changing to a group of people that are in the last years of their lives anyway and have no other options and could likely tell within a few treatments if Imetelstat would help.

[jayfish101]

Isn't off label prescribing an option?

[huntingonthebluffs]

Good question jayfish101. Maybe Hoosier Investor could address how restrictive the FDA label would be in this case regarding off label usage and how doctors would proceed. Without Medicare/ supplemental insurance, it would be very expensive for any patient and I doubt without biomarkers increasing the odds of good results, probably not likely outcome for patients on the financial margin.