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Fedratinib

Phase 3

Primary Myelofibrosis | Small molecule | Oncology |Bristol-Myers Squibb Company|Last Updated: Oct 21, 2025

Target and mechanism

Molecular targetJAK2, FLT3
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLEDDMC
Total Trials2
Total Enrollment233

FDA Designations

No designations recorded

Clinical trial landscape

Fedratinib · 7 trials · 5 indications

Phase 3 1Phase 1 6
NCT03952039An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previously Treated With RuxolitinibPrimary Myelofibrosis
COMPLETED202 Analytics
PHASE3COMPLETED
An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib
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Study Endpoints

Primary Endpoints

Spleen Volume Response Rate (RR)
From Screening to end of Cycle 6 (1 cycle = 28 days), approximately 196 days

Percentage of participants who have ≥ 35% spleen volume reduction (SVR) at end of cycle 6 A Cochran-Mantel-Haenszel (CMH) test to adjust for planned stratification factors (spleen size by palpation, platelet counts and refractory/relapsed or intolerance to ruxolitinib treatment). The RRs and 95% confidence intervals (CI) will be provided for each arm as well as for the difference in RRs and 95% confidence interval of the difference for fedratinib to BAT.

Fedratinib Pharmacokinetics: Estimation of maximum observed plasma concentration (Cmax)
Up to 12 days
Fedratinib Pharmacokinetics: Estimation of area under the curve (AUC) calculated from time zero to t, where t is the timepoint of the last quantifiable concentration (AUC(0-T))
Up to 12 days
Fedratinib Pharmacokinetics: Estimation of AUC calculated from time zero extrapolated to infinite time (AUC(INF))
Up to 12 days
Pharmacokinetic - Maximum observed plasma concentration (Cmax) for Fedratinib
Up to 216 hours following the last dose of fedratinib

Maximum observed plasma concentration

Pharmacokinetic - Area under the plasma concentration-time curve (AUC) for Fedratinib
Up to 216 hours following the last dose of fedratinib

Area under the plasma concentration-time curve

Maximum Tolerated Dose (MTD)
Up to Cycle 1 (each cycle is 28 days)

is the highest dose that causes DLTs in not more than 33% of the subjects treated with fedratinib in the first cycle with at least 3 evaluable subjects treated at this dose.

Recommended Phase 2 dose (RP2D)
Up to Cycle 1 (each cycle is 28 days)

is a recommended Phase 2 dose that is determined as safe and tolerable by the Safety Review Committee based on the data from the first cycle with at least 3 evaluable subjects treated at each dose of the Phase 1 part.

Response Rate (RR)
Up to Cycle 6 (each cycle is 28 days)

Proportion of subjects who have ≥ 35% SVR at end of Cycle 6 from baseline

Pharmacokinetic - Cmax (Digoxin, Rosuvastatin and Metformin
96 hours

Maximum observed plasma concentration

Pharmacokinetic - AUC (Digoxin, Rosuvastatin and Metformin
96 hours

Area under the curve

Pharmacokinetic - CLr
48 hours

Renal Clearance

Analysis of Glucose AUC after OGTT using linear trapezoidal method
3 hours

Area under the curve

Fedratinib Pharmacokinetic (PK): AUC0-t
Up to approximately 8 days.

Estimation of AUC from time zero to the last measured time point

Fedratinib Pharmacokinetic (PK): AUC0-∞
Up to approximately 8 days.

Estimation of AUC from time zero extrapolated to infinity

Fedratinib Pharmacokinetic (PK): Cmax
Up to approximately 8 days.

Estimation of maximum observed plasma concentration

Fedratinib Pharmacokinetic (PK): Tmax
Up to approximately 8 days.

Estimation of time to reach Cmax

Fedratinib Pharmacokinetic (PK): t1/2
Up to approximately 8 days.

Estimation of terminal elimination half-life

Fedratinib Pharmacokinetic (PK): CL/F
Up to approximately 8 days.

Estimation of apparent total plasma clearance when dosed orally

Fedratinib Pharmacokinetic (PK): Vz/F
Up to approximately 8 days.

Estimation of apparent volume of distribution when dosed orally

Fedratinib Pharmacokinetic - Cmax
Up to approximately 8 days.

Maximum observed plasma concentration

Fedratinib Pharmacokinetic - Tmax
Up to approximately 8 days.

Time to maximum observed plasma concentration

Fedratinib Pharmacokinetic - AUC0-t
Up to approximately 8 days.

Area under the curve from time zero to the last quantifiable concentration

Fedratinib Pharmacokinetic - AUC0-∞
Up to approximately 8 days.

Area under the curve from time zero extrapolated to infinity

Fedratinib Pharmacokinetic - t1/2
Up to approximately 8 days.

Terminal elimination half-life

Fedratinib Pharmacokinetic - CL/F
Up to approximately 8 days.

Apparent total plasma clearance when dosed orally

Fedratinib Pharmacokinetic - Vz/F
Up to approximately 8 days.

Apparent total volume of distribution when dosed orally

Secondary Endpoints

Symptom Response Rate (SRR)
From Cycle 1 Dose 1 to end of Cycle 6, approximately 168 days
Spleen Volume Response Rate (RR25)
From Screening to end of Cycle 6 (1 cycle = 28 days), approximately 196 days
Number of Participants With All Grade Treatment Emergent Adverse Events (AEs) and Grade 3 to 4 Treatment Emergent AEs
From Cycle 1 Dose 1 to end of Cycle 6, approximately 168 days
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Fedratinib 400mg/dayEXPERIMENTALWill include up to 128 subjects receiving fedratinib 400 mg self-administered Investigational Product (IP) on an outpatient basis, once daily preferably together with food during an evening meal at the same time each day in consecutive 4-week (28-day) cycles.
Best Available Therapy (BAT)ACTIVE_COMPARATORBest-available Investigator-selected therapy included a number of available compounds to treat MF and/or its symptoms and was chosen by the investigator for each subject. Therapy changed at different times during the treatment period. No investigational agents (e.g. not approved for the treatment of any indication) were allowed. BAT also included the choice of no treatment.
Treatment 1AEXPERIMENTAL -
Treatment 1BEXPERIMENTAL -
Treatment 2AEXPERIMENTAL -
Treatment 2BEXPERIMENTAL -
Treatment 2CEXPERIMENTAL -
Treatment AdministrationEXPERIMENTAL* Day 1: Single dose of 100 mg fedratinib * Days 10 to 23, inclusive: Single dose of 400 mg fluconazole on Day 10 and once daily (QD) doses of 200 mg fluconazole on Days 11 to 23, inclusive * Day 18: Single dose of 100 mg fedratinib coadministered with the 200-mg fluconazole dose.
Fedratinib AdministrationEXPERIMENTALThe fedratinib dose is 300 or 400 mg/day PO (3 or 4 x 100 mg capsules) to be self-administered orally once daily continuously on an outpatient basis, preferably together with food during an evening meal, the same time each day.
Fedratinib in moderate hepatic impairment subjectsEXPERIMENTALA single oral dose of 300 mg of fedratinib will be given to subjects with moderate hepatic impairment
Fedratinib in severe hepatic impairment subjectsEXPERIMENTALA single dose of 200 mg of fedratinib will be given to subjects with severe hepatic impairment
Fedratinib in healthy vs moderate hepatic impairment subjectsEXPERIMENTALA single oral dose of 300 mg of fedratinib will be given to healthy subjects with normal hepatic function.
Fedratinib in healthy vs severe hepatic impairment subjectsEXPERIMENTALA single oral dose of 200 mg of fedratinib will be given to healthy subjects with normal hepatic function.
Fedratinib plus RifampinEXPERIMENTALA single dose of fedratinib on Day 1. On Days 9 through 18, once-daily (QD) doses of rifampin. On Day 17, a single dose of fedratinib will be concomitantly administered with rifampin.
Fedratinib plus EfavirenzEXPERIMENTALA single dose of fedratinib on Day 1. On Days 9 through 18, once-daily (QD) doses of efavirenz. On Day 17, a single dose of fedratinib will be concomitantly administered with efavirenz.

Interventions

NameTypeDescription
FEDRATINIBDRUGA potent and selective inhibitor of JAK2 kinase activity
Best Available Therapy (BAT)DRUGBest available therapy (BAT)
FluconazoleDRUGFluconazole
DigoxinDRUGOral
RosuvastatinDRUGOral
MetforminDRUGOral
RifampinDRUGRifampin
EfavirenzDRUGEfavirenz
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites107

Inclusion Criteria: 1. Subject is at least 18 years of age at the time of signing the informed consent form (ICF) 2. Subject has an Eastern Cooperative Oncology Group (ECOG) Performance Score (PS) of 0, 1 or 2 3. Subject has diagnosis of primary myelofibrosis (PMF) according to the 2016 World Healt...

Countries:AustraliaAustriaBelgiumChinaCzechiaFranceGermanyHungaryIrelandItalyNetherlandsPolandRussiaSouth KoreaSpainUnited KingdomUnited StatesJapan
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Frequently asked questions about Fedratinib

What is Fedratinib used for?

Fedratinib is being studied for use in primary myelofibrosis and in healthy volunteers. In clinical trials, it is evaluated as a treatment for primary myelofibrosis, while healthy volunteer studies assess its pharmacokinetics, tolerability, and drug interactions. It is an investigational small molecule in Phase 1 clinical development.

What does Fedratinib target?

Fedratinib is a kinase inhibitor, belonging to the -tinib class of small molecules. It targets kinases, which are enzymes involved in cellular signaling. Its development focuses on conditions like primary myelofibrosis, where kinase activity plays a role in disease pathology.

Who makes Fedratinib?

Fedratinib is developed by Bristol-Myers Squibb Company, traded on the New York Stock Exchange under the ticker BMY. The company is conducting clinical trials to evaluate the drug's safety, tolerability, and pharmacokinetics in various patient populations.

What phase is Fedratinib in?

Fedratinib is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. The ongoing and completed trials focus on pharmacokinetics, tolerability, and drug interactions in healthy volunteers and subjects with hepatic impairment.

What clinical trials is Fedratinib in?

Fedratinib has been studied in several Phase 1 trials, including NCT03983161, which evaluated pharmacokinetics in subjects with moderate and severe hepatic impairment, and NCT04231435, which examined its effect on transporter probe substrates. Other completed trials include NCT04702464 and NCT05051553, both in healthy volunteers.

Is Fedratinib the same as other drugs?

Fedratinib is a distinct small molecule kinase inhibitor developed by Bristol-Myers Squibb. It is not known to be the same as any other drug. Its unique chemical structure and target profile differentiate it from other kinase inhibitors in development.