Is Rusfertide FDA Approved to Manage Erythrocytosis in Polycythemia Vera?

[billsman]

Some may be interested to know that the FDA approved rusfertide as a breakthrough treatment for PV. Not sure that's going to help folks like me with chronically low ferritin. From what I can find out thus far, the out-of-pocket cost would be $120k per year.

But at least there is a hepcidin mimetic available. And hopefully others are forthcoming.

 

[Nelson Vergel]

Hi @billsman

It is not approved yet. It was given a breakthrough designation:

FDA Grants Breakthrough Therapy Designation to Rusfertide for Erythrocytosis in Polycythemia Vera

Rusfertide earned an FDA breakthrough designation for polycythemia vera based on findings from the phase 3 VERIFY study.

www.onclive.com

What is Breakthrough Therapy Designation and how it affects a drug approval process?
Breakthrough Therapy Designation is a special status granted by the FDA to drugs that demonstrate preliminary evidence of substantial improvement over existing therapies for serious or life-threatening conditions, accelerating their development and review process.[1][2][3][4]
Definition and Criteria
· Breakthrough Therapy Designation is given to drugs that target serious diseases and show, through early clinical data, significant efficacy or safety advantages over current treatments.[2][3][1]
· The designation requires robust preliminary clinical evidence demonstrating improvement on clinically meaningful endpoints such as survival rates, major symptoms, or adverse event profiles.[3][5]
· Sponsors request this designation, usually before the end of Phase II clinical trials, and the FDA commits to responding within 60 days.[4][2]
Impact on Drug Approval Process
· Accelerated Development: Designated drugs benefit from streamlined clinical trials, sometimes with smaller or alternative study designs, and rolling review, meaning portions of the New Drug Application (NDA) or Biologics License Application (BLA) can be submitted as they are ready rather than waiting for complete submissions.[1][2]
· Expedited Review: The FDA provides intensive guidance, organizational commitment from senior staff, and priority review, cutting the review time from 10 months to 6 months.[2][4]
· Enhanced Collaboration: There are more frequent and high-level interactions between the FDA and sponsors, which facilitates faster problem solving and data collection strategies during clinical development.[4][2]
· Faster Access for Patients: By reducing both the drug development and review timelines, life-saving or transformative therapies reach patients sooner.[6][2]
· Legal Standards: The designation does not change the legal evidentiary requirements for approval; drugs must still demonstrate efficacy and safety to FDA standards, but they are guided and supported throughout the process.[7]
Summary Table: Key Features

Feature	Breakthrough Therapy Designation	Citation
Disease Targets	Serious or life-threatening	[2]
Required Data	Preliminary clinical evidence	[1]
FDA Guidance	Intensive, senior-level	[2]
Review Benefits	Rolling & priority review	[2][4]
Timeline Reduction	Up to 30% faster development	[2]

Breakthrough Therapy Designation can significantly shorten drug development timelines and increase patient access to innovative therapies, while maintaining the FDA’s safety standards.[6][2][4]



1. https://en.wikipedia.org/wiki/Breakthrough_therapy
2. https://www.precisionformedicine.com/blog/what-is-an-fda-breakthrough-therapy-designation
3. https://www.ozmosi.com/fda-accelerated-approval-drug-development/
4. https://www.fightcancer.org/policy-resources/fdas-expedited-drug-approval-programs
5. https://medelis.com/fda-breakthrough-therapy-designation-a-pathway-for-expedited-drug-development/
6. https://friendsofcancerresearch.org/about-breakthrough-therapies/
7. https://www.healthaffairs.org/do/10.1377/hpb20140515.807039/
8. https://www.nber.org/system/files/working_papers/w30712/w30712.pdf
9. https://www.gene.com/stories/breakthrough-designation-explained
10. https://www.parexel.com/insights/new-medicines-novel-insights/advancing-rare-disease-drug-development/effective-regulatory-strategies/timing-and-data-are-critical-winning-breakthrough-therapy-designation-rare-disease-drugs
11. https://pmc.ncbi.nlm.nih.gov/articles/PMC3745545/
12. https://pmc.ncbi.nlm.nih.gov/articles/PMC10324637/

13. https://www.goodrx.com/drugs/medication-basics/fda-breakthrough-therapy

 

[billsman]

Hi @billsman

It is not approved yet. It was given a breakthrough designation:

FDA Grants Breakthrough Therapy Designation to Rusfertide for Erythrocytosis in Polycythemia Vera

Rusfertide earned an FDA breakthrough designation for polycythemia vera based on findings from the phase 3 VERIFY study.

www.onclive.com

What is Breakthrough Therapy Designation and how it affects a drug approval process?
Breakthrough Therapy Designation is a special status granted by the FDA to drugs that demonstrate preliminary evidence of substantial improvement over existing therapies for serious or life-threatening conditions, accelerating their development and review process.[1][2][3][4]
Definition and Criteria
· Breakthrough Therapy Designation is given to drugs that target serious diseases and show, through early clinical data, significant efficacy or safety advantages over current treatments.[2][3][1]
· The designation requires robust preliminary clinical evidence demonstrating improvement on clinically meaningful endpoints such as survival rates, major symptoms, or adverse event profiles.[3][5]
· Sponsors request this designation, usually before the end of Phase II clinical trials, and the FDA commits to responding within 60 days.[4][2]
Impact on Drug Approval Process
· Accelerated Development: Designated drugs benefit from streamlined clinical trials, sometimes with smaller or alternative study designs, and rolling review, meaning portions of the New Drug Application (NDA) or Biologics License Application (BLA) can be submitted as they are ready rather than waiting for complete submissions.[1][2]
· Expedited Review: The FDA provides intensive guidance, organizational commitment from senior staff, and priority review, cutting the review time from 10 months to 6 months.[2][4]
· Enhanced Collaboration: There are more frequent and high-level interactions between the FDA and sponsors, which facilitates faster problem solving and data collection strategies during clinical development.[4][2]
· Faster Access for Patients: By reducing both the drug development and review timelines, life-saving or transformative therapies reach patients sooner.[6][2]
· Legal Standards: The designation does not change the legal evidentiary requirements for approval; drugs must still demonstrate efficacy and safety to FDA standards, but they are guided and supported throughout the process.[7]
Summary Table: Key Features

​

Feature	Breakthrough Therapy Designation	Citation
Disease Targets	Serious or life-threatening	[2]
Required Data	Preliminary clinical evidence	[1]
FDA Guidance	Intensive, senior-level	[2]
Review Benefits	Rolling & priority review	[2][4]
Timeline Reduction	Up to 30% faster development	[2]

Breakthrough Therapy Designation can significantly shorten drug development timelines and increase patient access to innovative therapies, while maintaining the FDA’s safety standards.[6][2][4]



1. https://en.wikipedia.org/wiki/Breakthrough_therapy
2. https://www.precisionformedicine.com/blog/what-is-an-fda-breakthrough-therapy-designation
3. https://www.ozmosi.com/fda-accelerated-approval-drug-development/
4. https://www.fightcancer.org/policy-resources/fdas-expedited-drug-approval-programs
5. https://medelis.com/fda-breakthrough-therapy-designation-a-pathway-for-expedited-drug-development/
6. https://friendsofcancerresearch.org/about-breakthrough-therapies/
7. https://www.healthaffairs.org/do/10.1377/hpb20140515.807039/
8. https://www.nber.org/system/files/working_papers/w30712/w30712.pdf
9. https://www.gene.com/stories/breakthrough-designation-explained
10. https://www.parexel.com/insights/new-medicines-novel-insights/advancing-rare-disease-drug-development/effective-regulatory-strategies/timing-and-data-are-critical-winning-breakthrough-therapy-designation-rare-disease-drugs
11. https://pmc.ncbi.nlm.nih.gov/articles/PMC3745545/
12. https://pmc.ncbi.nlm.nih.gov/articles/PMC10324637/

13. https://www.goodrx.com/drugs/medication-basics/fda-breakthrough-therapy

You're correct. Thanks for pointing that out.

 

[madman]

Some may be interested to know that the FDA approved rusfertide as a breakthrough treatment for PV. Not sure that's going to help folks like me with chronically low ferritin. From what I can find out thus far, the out-of-pocket cost would be $120k per year.

But at least there is a hepcidin mimetic available. And hopefully others are forthcoming.

* Rusfertide (Takeda) is currently being investigated in the phase 3 VERIFY trial (NCT05210790), with an estimate study complete date of June 2025.3 The hepcidin mimetic has already received breakthrough therapy, orphan drug, and fast track designations from the FDA.




post # 11 (1st thread)!

Thread 'Rusfertide Decreases Need for Therapeutic Phlebotomy in Polycythemia Vera'

Jun 25, 2023

New Treatment Could End the Phlebotomy Cycle for Men with High Hematocrit​

If you're a man on testosterone replacement therapy (TRT) who's been told you need regular blood draws to manage your hematocrit levels, a breakthrough treatment currently in clinical trials could change everything. Recent research on a drug called rusfertide shows promise for maintaining healthy hematocrit levels without the constant need for therapeutic phlebotomies.

The Problem You Know Too Well​

Men on TRT frequently develop elevated hematocrit—a condition where your blood becomes too thick due to an...

• madman
• polycythemia vera; rusfaertide; hepcidin mimetic
• Replies: 7
• Forum: Health & Wellness

 

[Nelson Vergel]

More info: Rusfertide Leads to Promising Phlebotomy Reduction in Polycythemia Vera

 

[billsman]

So I am at a point where my iron and ferritin are chronically low. I take xyotsed 100 mg per week. I do TP every 2 months. Something has to give.

My question: other than gels, are there other TRT that do not produce erythrocytosis?

 

[ignition]

So I am at a point where my iron and ferritin are chronically low. I take xyotsed 100 mg per week. I do TP every 2 months. Something has to give.

My question: other than gels, are there other TRT that do not produce erythrocytosis?

Oral: Kyzatrex, Jatenzo, Tlando.

And general comment, I hope to be happily surprised, but I don't think we will see Rusfertide use for TRT driven erythrocytosis for some time to come.

My hem/onc and cancer center had the trial and I asked about it. He was emphatic that not having polycythemia vera, I was ineligible. It has only been trialed for polycythemia vera specifically. It doesn't have to be, but it has been developed as a very niche specialty drug.

I also wonder what kind of price it will have.

Sure hope it becomes readily available for other causes of erythrocytosis!

 

[madman]

So I am at a point where my iron and ferritin are chronically low. I take xyotsed 100 mg per week. I do TP every 2 months. Something has to give.

My question: other than gels, are there other TRT that do not produce erythrocytosis?

Oral TU (Jatenzo, Tlando or Kyzatrex) or nasal T-gel (Natesto).

Transdermal (gels/cream) can still easily cause issues with elevated HCT if you push your FT too high!

Oral TU would be the best option here and would give you the most bang for your buck especially when it comes to minimizing side-effects.

Post in thread 'Oral Testosterone Therapy in Men with Low Testosterone: A Comprehensive Review'

Nov 23, 2024

Understanding Testosterone and Muscle Preservation - Excel Male Men's Health Forum

Never heard about oral TU LOL! In this episode of The Dr. Gabrielle Lyon Show, Dr. Lyon sits down with Shalin Shah, CEO of Marius Pharmaceuticals, to explore the science, benefits, and future of testosterone therapy. Shalin has been a driving force behind bringing Kyzatrex, an FDA-approved...

www.excelmale.com

00:29:13 - Oral vs Injectable Testosterone: Benefits and Comparisons

01:05:55 - Kyzatrex: A New Approach to Testosterone Delivery




If your goal is to minimize chances of driving up hematocrit let alone hitting a high-end short-lived daily peak twice daily then 400 mg BID is most likely the way to achieve such unless you are one of those hyper responders who will fare well on a lower daily dose...

• madman

 

[billsman]

Oral: Kyzatrex, Jatenzo, Tlando.

And general comment, I hope to be happily surprised, but I don't think we will see Rusfertide use for TRT driven erythrocytosis for some time to come.

My hem/onc and cancer center had the trial and I asked about it. He was emphatic that not having polycythemia vera, I was ineligible. It has only been trialed for polycythemia vera specifically. It doesn't have to be, but it has been developed as a very niche specialty drug.

I also wonder what kind of price it will have.

Sure hope it becomes readily available for other causes of erythrocytosis!

I'm afraid you're correct about rusfertide. However, my hope is that there will be competitors in the future.

I tried Jatenzo in the past and it didn't produce good levels for me. I'm not looking for supra-levels -- just 500s. I've been on TRT for over 25 years, but I've only had high crit/low iron for the past 4 years, which seems to be made worse by my use of xyosted. Unfortunately, the gels didn't work for me. I was on hCG for 10 years to have kids, but my E2 was high and I was dosing anastrozole was challanging. Tried IM test, but that's when the high crit really took off, which I know is often the case. I just don't feel like I have a lot of options now. But I've got to get my iron up -- the fatigue of awful.