Danazol for ITP

Phase 3 trials for fostamatinib were completed in August 2016. The results have not been published, and reports are that the raw numbers were not encouraging, with just 18% of patients achieving a stable response. Nonetheless, they are proceeding with FDA approval, which makes me think there is more to the story. Perhaps there was a subgroup of patients for whom fostamatinib was effective, they didn't say. What they do say is that when fostamatinib is successful, it is very successful.

finance.yahoo.com/news/edited-transcript-rigl-earnings-conference-050402863.html
Edited Transcript of RIGL earnings conference call or presentation 2-May-17 9:00pm GMT
Thomson Reuters StreetEvents•May 5, 2017

FDA has conditionally accepted the proprietary name Tavalisse for our lead investigational drug candidate, fostamatinib disodium, Tavalisse.

Data from our FIT Phase III clinical program were consistent across our 2 pivotal studies, 047 and 048, as well as the long-term, open-label extension study 049. Patients who responded to Tavalisse had a timely, robust and a sustained response to treatment. These 3 themes, timely, robust and sustained, underscore the potential value of Tavalisse, which Tavalisse may offer patients. The rapid response gives an early feedback as to whether Tavalisse may be a viable treatment option for patients with ITP. The robust response becomes clear when you see that in the pivotal studies 047 and 08, patients' platelet counts rose and then held steady at about 100,000 platelets per microliter of blood.

Our analysis also demonstrates that Tavalisse may provide an enduring benefit based on the median data of 16 months treatment from studies 047 and 048 and increasing, which gives patients and their doctors confidence that the disease is controlled in the long term. As expected, the FIT Phase III clinical program confirmed that Tavalisse has a consistent and predictable safety profile, the profile aligned with the safety database of over 5,000 patient years, which we have accrued.

Note from the story that the studies (for all uses of fostamatinib) encompassed a total of 5200 patient years, and the application to the FDA, if printed out, would total approximately 1.6 million pages that would stack over 500 feet high. No wonder drugs are so costly!

Yea that 18% number sounds very interesting to me. That implies that the drug works with just one of the four types of ITP/PIT. So in a study it should be very obvious that only patients with steroid response X, IVIG response Y, and maybe allergy response Z is a strong predictor of success when treated with it.

That kind of applicability pre-assurance to patients could make it become the instant dominant treatment for ~20% of patients.

One of the slides from the Rigel earnings call tells a better story: while 18% achieved a stable response above 100,000, another 11% achieved an "intermediate response" which was essentially >50,000 platelets. 29% is still not impressive... unless they can identify ahead of time which patients might benefit!
Here is the link:

static2.seekingalpha.com/uploads/sa_presentations/854/8854/slides/16.jpg?1493765338

Some info for you rjsmyth.
I happen to run across PDSA user 'sachmo16' who was on the fostamatinib trial.
pdsa.org/discussion-group/search.html?searchuser=sachmo16&exactname=1&searchdate=all&order=inc&childforums=1

Looking over his reported steroid and IVIG response, it seems to match yours. Specifically, he has a poor steroid response and a weak (two week) IVIG response. Further, he was living off of regular IVIG infusions before the trial. Oh my.

Might want to check into fostamatinib with your doc.

Hal, the fostamatinib trials for ITP are no longer recruiting, and FDA approval of fostamatinib is not anticipated until sometime in 2018, if at all. I do not know of any way to receive treatment at this point... do you?

No signs of Fostamatinib gaining approval in UK as far as I can see. Have read 2 documents lately both of which state "if" it gets approval with no time scales mentioned at all.

I am not familiar with the FDA drug approval process at all.
Is the FDA approval process just a paper work review process for the trial completed? For approval, did Rigel pay the FDA to do this - like the way a patent approval process works? If it works like a patent application, Rigel wouldn't ask for approval if they don't plan on marketing it. Because approval cost $.

I can only speculate that Rigel will eventually seek European approval only if it becomes FDA approved.

If I'm reading correctly, fostamatinib is a maintenance drug, yes? Having a bunch of patients on it for a couple decades sounds very profitable.

Here is the link mrsb posted earlier. Apparently Rigel considered it a failure then because the 18% response was far lower than expected.
www.pmlive.com/pharma_news/rigel_shaken_after_fostamatinib_misses_phase_iii_trial_target_1173284

Seeking Alpha? A common interest. Do you think cornering 20% of the ITP market would 'move the needle' for Rigel profits? 326 million dollar company...

Stopped Danazol as it was giving me no real benefit i.e. without IVIG it would not sustain a decent platelet count and some side effects.

H. Pylori stool antigen test negative.

Last week count was 3 before IVIG with nasty blood blisters in my mouth and feeling pretty rotten. This week (mid term between IVIG infusions) 5 with no symptoms but suprisingly low!

Going to try and look after myself a bit better (abstain from alcohol, caffeine etc.).

Shame I never got to try Fostamatinab and the failure to pass the phase3 trials means it seems pretty much dead and buried in the UK.

Back to square one.

rjsmyth, in my PDSA forum post research, I came across this post:
pdsa.org/discussion-group/13-general-discussion-for-parents/26783-timothy-rituximab-and-nplate.html#27968

In this case Rituxan was ineffective for remission. But, after NPlate response was lost, Rituxan was used to regain NPlate response.

LOL, I think I suggested this idea in an earlier post. 'lucidawn' story is evidence of the possibility of it working for you.