Rusfertide (for HCT Control)- FDA status

[EPguy]

I came across this info today.

Mar 28 2024: "Protagonist Therapeutics said it plans to seek FDA approval for rusfertide for treating polycythemia vera in late 2025"

cancer.gov/news-events/canc...

and an explanation why it has taken so long, an FDA hold a few years ago:

Oct 2021 "The FDA has removed the full clinical hold that had previously been placed on trials evaluating rusfertide (PTG-300)"

They were monitoring mice on it and "found benign and malignant subcutaneous skin tumors" Seems ok in humans since trial resumed, but no doubt they are watching carefully. Sometimes they use very high doses in these sort of non-clinical studies, but it's not specified.

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One application for Rusf may be HCT resistance on IFN, a problem we're seeing regularly on the forum.

[Lena70]

Very exciting news.

[hunter5582]

Good news!

[saltmarsh]

Thanks for the update. Definitely good news

[Lyndjs]

Thank you for sharing and update.

Lx

[ritaandscooter1]

Good news! My Mayo MPN recommended this for iron deficient, phlebotomy dependent people like myself. Will also help keep HCT stable. He said the only draw back is the injection site gets extremely irritated and must have injection every week. He said there is a better drug in the works (Disc) that he is hoping to put me in the clinical trial in the fall. Basically does the same thing but much less side effects. He highly recommends this one heading for clinical trials.

[EPguy in reply to ritaandscooter1]

Good to know on the irritation. Sounds like the pt quickly runs out of fresh injection sites.

On the new one I think you refer to MWTX-003:

ir.discmedicine.com/news-re...

It's in trial for MF right now, interesting that it can help reduce both MF anemia and PV HCT. Part of the complex interactions going on.

clinicaltrials.gov/study/NC...

[ritaandscooter1 in reply to EPguy]

I think there is a new Disc drug trial coming out in the fall which may be different than the above links you sent?? Not entirely sure how different they are. Iron retention is involved with both. Will find out when I see the doc next time.

[EPguy in reply to ritaandscooter1]

Agree, the new trial would be for PV, while the current one is for MF.

[ritaandscooter1 in reply to EPguy]

Thanks for this info! I'm so happy they are coming out with drugs to improve ones QOL especially due to iron deficiency.

[EPguy in reply to ritaandscooter1]

I've come to appreciate how lucky we've been to have the treatments we have. having a condition with none. Every step makes someone's life better.