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.