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Rusfertide

Phase 3FDA approved

Polycythemia Vera | Small molecule | Hematology |Takeda Pharmaceutical Company Limited|Trials Updated: Oct 2, 2026

Rusfertide development status

FDA approval on record Aug 28, 2026 as Mimrylo
Approval holderTakeda Pharms Am
ApplicationNDA220605
Highest phase Phase 3 run by Protagonist Therapeutics, Inc. (NCT05210790)
Phase scored for TAKPhase 2
Registered trials 9 across 2 sponsors since Jan 2019

Rusfertide target and mechanism

Molecular targethepcidin
Target class-Tide (Peptide)
ModalitySmall molecule

Also known as TAK-121, PTG-300

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials2
Total Enrollment33

FDA Designations

PRIORITY_REVIEW

Rusfertide clinical trials

Rusfertide · 2 trials · 1 indication

Phase 2 2
NCT07765602A Study of Rusfertide in Adults With Polycythemia Vera in ChinaPolycythemia Vera
NOT YET_RECRUITING24 Analytics
NCT07648030A Study of Rusfertide in Japanese Adults With Polycythemia VeraPolycythemia Vera
RECRUITING9 Analytics
PHASE2NOT YET_RECRUITING
A Study of Rusfertide in Adults With Polycythemia Vera in China
Polycythemia VeraUnlock trial analytics
PHASE2RECRUITING
A Study of Rusfertide in Japanese Adults With Polycythemia Vera
Polycythemia VeraUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of Participants Achieving a Response Starting at Week 20 Through Week 32
Week 20 through Week 32

Response is defined as the absence of phlebotomy eligibility. Phlebotomy eligibility is defined as: A confirmed hematocrit ≥45%, which is ≥3% (absolute) higher than the baseline hematocrit or hematocrit ≥48%. Confirmation is defined as 2 consecutive hematocrit assessments that are ≥45% and at least 3% higher than the baseline hematocrit.

Secondary Endpoints

Change From Baseline in Hematocrit Over Time
From Baseline to Week 32
Percentage of Participants Maintaining Hematocrit <45% During Week 0 to Week 32 and Week 0 to Week 52
From Baseline to Weeks 32 and 52
Median Time to First Hematocrit ≥45% After Enrollment
From Baseline to Week 52
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
RusfertideEXPERIMENTALParticipants will receive rusfertide (TAK-121), subcutaneously (SC) once weekly for 52 weeks.

Interventions

NameTypeDescription
RusfertideDRUGRusfertide injections administered SC.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo

* Inclusion Criteria 1. Chinese male and female participants aged 18 years or older at the time of signing of informed consent. 2. Participant understands the trial procedures, is willing and able to adhere to trial requirements, and agrees to participate in the trial by providing written infor...

Countries:Japan
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Competitive Landscape -Polycythemia Vera 19 trials

Recent Changes (Last 90 Days)

LOWOct 2, 2026NCT07648030lastUpdatePostDate: changed
LOWOct 2, 2026NCT07648030lastUpdatePostDate: changed
LOWSep 2, 2026NCT07648030lastUpdatePostDate: changed
LOWSep 2, 2026NCT07648030lastUpdatePostDate: changed
LOWSep 2, 2026NCT07648030lastUpdatePostDate: changed
LOWAug 28, 2026NCT07765602lastUpdatePostDate: changed
LOWAug 28, 2026NCT07765602lastUpdatePostDate: changed
LOWAug 14, 2026NCT07765602NEW_TRIAL: changed
LOWAug 14, 2026NCT07765602NEW_TRIAL: changed
LOWAug 5, 2026NCT07648030lastUpdatePostDate: changed
LOWJul 30, 2026NCT07648030Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 30, 2026NCT07648030Status: NOT_YET_RECRUITING → RECRUITING

Frequently asked questions about Rusfertide

What is Rusfertide used for?

Rusfertide is an investigational small molecule being studied for the treatment of Polycythemia Vera, a hematologic condition. It is currently in Phase 2 clinical development and has not been approved by the FDA. Rusfertide is being evaluated in adult patients with Polycythemia Vera.

What does Rusfertide target?

Rusfertide targets hepcidin, a peptide hormone that regulates iron metabolism. By modulating hepcidin, Rusfertide may help control red blood cell production in Polycythemia Vera. It is classified as a -tide (peptide) modality, though it is a small molecule.

Who is developing Rusfertide?

Rusfertide is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. The company is conducting Phase 2 clinical trials for Rusfertide in Polycythemia Vera. Takeda is responsible for the drug's clinical development program.

What phase is Rusfertide in?

Rusfertide is in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. The drug has been granted Priority Review designation by the FDA, which may expedite the review process if it reaches that stage. Currently, Rusfertide is being studied in clinical trials.

What clinical trials is Rusfertide in?

Rusfertide is being studied in two Phase 2 clinical trials. NCT07765602 is a study in adults with Polycythemia Vera in China, with an enrollment of 24 participants, and is not yet recruiting. NCT07648030 is a study in Japanese adults with Polycythemia Vera, enrolling 9 participants, and is currently recruiting.

Is Rusfertide the same as hepcidin?

No, Rusfertide is not the same as hepcidin. Rusfertide is a drug that targets hepcidin, a peptide hormone involved in iron regulation. Rusfertide is a small molecule that modulates hepcidin activity, potentially affecting red blood cell production in Polycythemia Vera.