Plain-English fact sheet
Rusfertide
Also known as PTG-300
Rusfertide is a late-stage experimental peptide designed to control excess red-blood-cell production in polycythemia vera; it is not yet FDA approved.
Quick answer
Rusfertide is a hepcidin-mimetic peptide being reviewed by FDA for adults with polycythemia vera. FDA accepted its application and granted priority review, but application acceptance is not approval.
By the PeptideFactSheets Editorial Team. Use the source links to verify status, evidence, risks, and unknowns; our editorial policyexplains the boundaries. Clinician review appears only when a named reviewer is shown.
What is Rusfertide?
Rusfertide is a manufactured peptide that mimics hepcidin, the hormone that regulates iron availability.
By restricting iron available for red-blood-cell production, it is designed to help control hematocrit and reduce the need for phlebotomy in polycythemia vera.
Why are people interested in it?
It applies peptide design to a blood cancer pathway and has moved from randomized Phase 2 evidence through a completed pivotal trial to regulatory review.
The program is more mature than many frontier peptides, but approval, final labeling, and postmarketing safety remain unresolved.
Current regulatory status
Rusfertide is not FDA approved. FDA accepted a new drug application and granted priority review; the sponsor reports that FDA review is pending.
What is it approved for?
No FDA-approved use. This matters because clinical-trial participation and products marketed online are not the same as an approved medicine.
FDA approval path
NDA under FDA review
Rusfertide has a Phase 3 polycythemia vera program and a company-reported FDA priority-review milestone. Until FDA acts, it remains investigational.
Step 6 of 7
- DiscoveryBasic research
- PreclinicalLab and animal work
- Phase 1Initial human testing
- Phase 2Condition-focused trials
- Phase 3Confirmatory trials
- FDA reviewApplication review
- ApprovedLabel-specific use
What is it being studied for?
Investigational areas
- Treatment of adults with polycythemia vera requiring ongoing hematocrit control
Evidence snapshot
A published randomized-withdrawal Phase 2 trial supports hematocrit control and fewer phlebotomies, and a 293-participant Phase 3 trial has completed its randomized primary period. Final FDA review and complete peer-reviewed pivotal evidence remain decisive.
Potential benefits being researched
- The Phase 2 REVIVE trial found better composite response during randomized withdrawal with rusfertide than placebo.
- Sponsor-reported Phase 3 results supported the regulatory application, but final labeling and FDA conclusions are not yet available.
Potential does not mean proven. Study design, population, endpoint, and regulatory review matter.
Known or possible risks
- Published and sponsor-reported studies identify injection-site reactions, anemia, and fatigue among observed adverse events.
- Changing iron availability can produce iron-related and blood-count effects that require study-specific clinical monitoring.
- The complete approved-use safety profile does not exist unless and until FDA approves a product and label.
What we still do not know
- Whether FDA will approve the application and what exact population and warnings a label would contain
- Long-term effects on thrombosis, disease progression, symptoms, and survival
- Uncommon harms with broader exposure
- How it compares with established polycythemia vera strategies on patient-centered outcomes
Plain-English takeaway
Rusfertide is a credible late-stage peptide program with randomized human evidence and an application under review. It remains investigational until FDA completes that review.
Research and reference links
Use these primary and reputable sources to verify status and read beyond this summary. Trial registries may list studies without proving a benefit.
- 1clinical trialsClinicalTrials.gov: VERIFY Phase 3 trial
Official record for the 293-participant pivotal trial, including endpoints and public results status.
- 2pubmedREVIVE randomized-withdrawal trial
Peer-reviewed Phase 2 evidence for hematocrit control and phlebotomy reduction.
- 3company releaseTakeda: FDA accepts rusfertide application
Company-reported application acceptance and priority-review milestone; not an FDA approval decision.
- 4educationalSEC filing: rusfertide regulatory status
Public company filing confirming submission and pending review of the application.