Rux is currently 2nd line after HU and Bes. This was a direct issue for me when my new medicare part D denied my Rux Rx even as I've been on it approaching two years. They said I must have tried HU and Bes to be approved. I have, HU had more sides and regulars know how Bes turned out. I was approved shortly.
So I've been looking for a clear answer how or why Rux is not 1st line. It's in the trial some UK members are in: "MITHRIDATE"
"A positive result from this study could lead to the development of ruxolitinib as a new first-line therapy for patients with PV."
They seek to answer "whether JAK2 inhibitor therapy has the potential to alter the natural history of PV " We know from some rcent studies that it did, this a larger study and will compare all 3 drugs side by side for the first time to "allow a deeper analysis of the effect of HC, IFN, and ruxolitinib on disease biology over time."
Primary completion is late 2028. So that likely is the earliest it will get the new 1st line approval. This happens to be when the Jakafi patent runs out. So Incyte has no reason to do this study and in fact they are not, "Principal Investigator: Claire Harrison, Acting on behalf of the Sponsor (UK)"
UK govt is the sponsor, while Novartis and our own MPN Voice are "collaborators." I assume that means Incyte is offering the Rux pills and will use the results to guide future drug research.
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EPguy
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Unfortunately, when ruxolitinib was developed, hydroxyurea was already the standard first-line treatment for PV, and the trials were designed to position it as a second-line option. I expect this had largely to do with how cheap hydroxyurea is. PhramEssential did not make this mistake in designing their clinical trials. They designed to trial with the intention of Besremi being a first-line treatment option. Once approved by regulatory agencies, this makes Besremi easier to access. It will now take time and effort for Incyte to change the indication for ruxolitinib to be a first line treatment option for PV as it is for MF. It would be in the best interests of the MPN community for this to happen. Providing evidence that RUX can alter the natural history of PV would support this contention since that would make it a superior option to HU. Hopefully the research will have the desired outcome.
It's primary date is 2027, and has no HU limitation. But it's only a ph2. My guess they will await the ph3 MITHRIDATE results to apply, as suggested by the NCT explicit title for the trial:
MITHRIDATE: Ruxolitinib Versus Hydroxycarbamide or Interferon as First Line Therapy in High Risk Polycythemia Vera (MITHRIDATE)
Quick look at clinicaltrials.gov shows over 400 results for Rux. I checked a random well known med, xeljanz, and it's only ~300. So it's surprising with that much investment they didn't long ago do a broader ph 3 trial for PV.
Thank you EPguy for posting, always relevant and thought provoking. Much appreciated! Very interesting and encouraging trial, especially for me now that I have officially failed on Besremi (increased allele burden and development of chronic hives). That makes me a 2-time loser so far (HU and BES) since my diagnosis 3 1/2 years ago. After several months without BES, just aspirin and a phlebotomy, I started on Rux this week and am hoping for the best. My MPN specialist and Allergist are hopeful that Rux will help with the chronic hives so I don't have to keep taking massive amounts of anti-histamines and Montelukast. I'm glad to see this trial is in the works. Keeping fingers crossed that my Rux experience is a good one with no events or progression and improved VAF. The good news is that I'm actually feeling pretty good, so I can't be too complaining. May these trials continue to advance us toward a CURE and may the force be with us all!
Thank you for posting, EPguy! I had to go through Hu and Interferon first to be allowed on Rux ("as the last measure"- the way my hematologist put it). I didn't tolerate interferon and had horrible side effects on HU. I am keeping my fingers crossed for Rux.
If it works better for many patients, and avoids going through the painful experiences, before it can be tried, it would be good.
Cost seems secondary since my ins co was ok with a Bes Rx (been there). So it's a strictly bureaucratic process. 1st line would be nice.
Understand the weight, it fixed my weight loss and then some. I've got it stabilized by eating the same stuff just less of it. One might say I get higher mpg now not needing to stuff myself.
I am on the MITHRIDATE trail and was advised when I started on it that patients were `randomly` selected but I have my doubts. I was not successful in being selected to trial Ruxolitnib which was disappointing but to be fair I am not doing too bad on HU. I still have to complete a daily diary for 3 years as part of the trial which I do question to myself as I think HU is already well researched and has been used for so long they must know everything about it. However, the trial does have the benefit of of more regular check ups so I am going along with it and have already completed 1 year which has gone surprisingly fast. I suppose any help with research is a step along the way to finding a cure for this. I am sure costs are a consideration and it is interesting to see that the Rux patent runs out is 3 years as I am sure that will make it more affordable and help it to become a 1st line treatment. Fingers crossed they will one day find a cure. Good luck with your journey 👍
You're right on HU being well studied. The Bes trials were specifically against HU. What has been missing is IFN vs Rux. Also largely missing is Rux vs anything for pts who are tolerant to HU. They need this for the 1st line status. So you are one of the latter. Are they doing Jak2 mutation tests? This is a must for a modern long term trial.
Correct: HU has been very effective for the majority for decades and is inexpensive. Jakafi (Rux) comes off patent in December 2028 and is VERY expensive thus forcing all controllers of drug budgets to be cautious before that date. lets hope the generics guys are ready with a cheap alternative on day 1.
Rux is a "small molecule" drug that is manufactured using basic chemistry. These are generally easy to make in contrast for example to biologics (IFN and hundreds of other even more complex). So there should be plenty of knock offs. But they do still need authority approval to prove they are indeed equivalent. But this is way easier for small molecules.
Incyte may have their once a day pill by then, (FDA rejected the application) but that convenience won't support a large price diff.
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