Hormonal

Rusfertide

Also known as: PTG-300, Rusfertide injection

Clinical Trials
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Key Facts: Rusfertide

Category
Hormonal
FDA Status
Not FDA Approved
Clinical Status
NDA Submitted (Jan 2026) - FDA Priority Review, PDUFA date Q3 2026. Holds Breakthrough Therapy, Orphan Drug, Fast Track, and Priority Review designations.
Administration
Subcutaneous injection, once weekly
Typical Dose
40-80 mg weekly
Frequency
Once weekly
Duration
Ongoing - designed for chronic use
Also Known As
PTG-300, Rusfertide injection

Mechanism of Action

Hepcidin is the hormone that decides how much iron enters the bloodstream by controlling the iron exporter ferroportin. When hepcidin is high, ferroportin is degraded, so iron stays locked in storage and gut cells instead of feeding red blood cell factories in the marrow. Rusfertide is a synthetic hepcidin mimetic that imposes this iron restriction on purpose, starving the overactive marrow in PV of the iron it needs to overproduce red cells. The result is better-controlled hematocrit without constant phlebotomy. This is a well-characterized, on-target mechanism, not a speculative hypothesis.

Research Summary

The randomized phase 2 REVIVE trial, published in the New England Journal of Medicine, showed rusfertide kept hematocrit under control and sharply cut phlebotomy in PV patients. The pivotal phase 3 VERIFY trial (293 patients) then met its primary endpoint and all four key secondary endpoints: 77% of rusfertide-treated patients were free of phlebotomy eligibility during weeks 20 to 32 versus 33% on placebo, with results presented at the 2025 ASCO Annual Meeting and durability data through week 52 reported afterward. On that strength, the FDA accepted the new drug application under priority review with a target decision date in 2026, and it holds Breakthrough Therapy designation. Reported side effects are generally injection-site reactions and manageable, though long-term safety in a chronic blood cancer is still being followed. This is one of the few peptides on this list with genuine, high-quality randomized human evidence rather than animal-only data.

Trial Progress:Preclinical
Pre
I
II
III
IV
FDA

Dosing Information

Phase 3 Clinical Trials·Active Phase 3 trials with FDA Breakthrough Therapy designation

Typical Dosing

Community experience

Common Dose

40-80 mg weekly

Range

10-120 mg weekly (trial range)

Frequency

Once weekly

Dose titrated based on hematocrit response. In REVIVE trial, most patients stabilized at 40-80 mg weekly. Designed as chronic therapy. Being studied primarily for polycythemia vera but has potential application for TRT-induced erythrocytosis (elevated hematocrit from testosterone therapy).

Research Dosing

Scientific studies

Doses from clinical trials only

Duration

Ongoing - designed for chronic use

Administration

Subcutaneous injection, once weekly

Timing & Administration

Best Time to Take

Once weekly, consistent day

Once weekly

Food Recommendation

With or without food

Why This Timing?

Long-acting hepcidin mimetic with weekly dosing schedule. Designed for once-weekly subcutaneous injection for convenience and compliance.

Possible Side Effects

Not everyone experiences these effects. Individual responses vary based on dosage, duration, and personal factors.

  • Injection site reactions (most common, mild)
  • Iron deficiency (expected mechanism of action)
  • Fatigue
  • Dizziness
  • Headache
  • Nausea
  • Generally well-tolerated in Phase 2 trials
  • Long-term safety data still being collected in Phase 3

References

Research This Peptide Further

Frequently Asked Questions

What does Rusfertide do?

Rusfertide is an injectable peptide that mimics hepcidin, the body's master hormone for controlling iron, and it is being developed to manage polycythemia vera (PV), a blood cancer that makes too many red blood cells. By limiting iron availability, it throttles red blood cell production and reduces the need for repeated blood draws (phlebotomy). It is a late-stage investigational drug, not a research-only or gray-market peptide: it cleared a positive phase 3 trial and is under FDA priority review.

How does Rusfertide work?

Hepcidin is the hormone that decides how much iron enters the bloodstream by controlling the iron exporter ferroportin. When hepcidin is high, ferroportin is degraded, so iron stays locked in storage and gut cells instead of feeding red blood cell factories in the marrow. Rusfertide is a synthetic hepcidin mimetic that imposes this iron restriction on purpose, starving the overactive marrow in PV of the iron it needs to overproduce red cells. The result is better-controlled hematocrit without constant phlebotomy. This is a well-characterized, on-target mechanism, not a speculative hypothesis.

Is Rusfertide FDA approved?

No, Rusfertide is not currently FDA approved. Current status: NDA Submitted (Jan 2026) - FDA Priority Review, PDUFA date Q3 2026. Holds Breakthrough Therapy, Orphan Drug, Fast Track, and Priority Review designations.

What are the side effects of Rusfertide?

Reported side effects include: Injection site reactions (most common, mild), Iron deficiency (expected mechanism of action), Fatigue, Dizziness, Headache. Individual responses vary based on dosage, duration, and personal health factors.

What is the typical dose of Rusfertide?

Community-reported common dose: 40-80 mg weekly (Once weekly). Range: 10-120 mg weekly (trial range). Administration: Subcutaneous injection, once weekly. Currently investigational. Not yet FDA approved. Only available through clinical trials. All dosing from published trial data. Not medical advice.

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Desmopressin

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Desmopressin (dDAVP) is a synthetic tweak of the natural hormone vasopressin, redesigned to keep the water-retaining effect while dropping most of the blood-pressure effect. It is a long-established FDA-approved drug used for central diabetes insipidus, bedwetting and nocturia, and certain bleeding disorders like von Willebrand disease and mild hemophilia A. This is settled medicine, not an experimental peptide, and its main real-world danger is straightforward: it can drop your blood sodium dangerously low if you drink too much fluid on it.

Hormonal

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