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Drug

Ruxolitinib

Approved · FDA / EMA
Emerging researchLate-stage developmentSteady momentum

Also known as Jakafi, Opzelura.

RxNorm1193326ChEMBLCHEMBL1789941ATCD11AH09ATCL01EJ01UNII82S8X8XX8H
6
Research papers
78
Active clinical trials
31
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase 1/2, Open-Label, Dose-Escalation, Safety, Pharmacokinetic, and Pharmacodynamic Study of Oral Nuvisertib (TP-3654) in Patients With Intermediate or High-Risk Primary or Secondary Myelofibrosis

Clinical trial2026-08-03Results expected Q2 2027 · ClinicalTrials.gov

Efficacy and Safety of Ruxolitinib in Neuromyelitis Optica Spectrum Disorders

Clinical trial2026-08-01Withdrawn · ClinicalTrials.gov

Phase II Study Of Combination Ruxolitinib (INCB018424) With Preoperative Chemotherapy For Triple Negative Inflammatory Breast Cancer

Clinical trial2026-07-24Status changed to Completed · ClinicalTrials.gov

CHMP positive opinion: Ruxolitinib Viatris (EMA)

Regulatory2026-07-20EMA

Label change: RUXOLITINIB (NDA202192)

Regulatory2026-07-20FDA

Label change: RUXOLITINIB (NDA217180)

Regulatory2026-07-20FDA

A Phase 3, Randomized, Double-blind, Active-control Study of Pelabresib (DAK539) and Ruxolitinib vs. Placebo and Ruxolitinib in Adult Patients With Myelofibrosis Who Are JAK Inhibitor Naive

Clinical trial2026-07-17Results expected Q2 2028 · ClinicalTrials.gov

Label change: RUXOLITINIB (NDA215309)

Regulatory2026-06-30FDA

Ipsen pens $1.7B deal to acquire Kartos for potential Jakafi add-on therapy for myelofibrosis

Industry2026-06-29Acquisition · Fierce Biotech

JAK inhibition enhances checkpoint blockade immunotherapy in patients with Hodgkin lymphoma.

Research2024-06-21Science (New York, N.Y.)

A phase I study of panobinostat and ruxolitinib in patients with primary myelofibrosis (PMF) and post--polycythemia vera/essential thrombocythemia myelofibrosis (post--PV/ET MF).

Research2019-11-16Leukemia research

Efficacy, safety, and survival with ruxolitinib in patients with myelofibrosis: results of a median 3-year follow-up of COMFORT-I.

Research2015-01-23Haematologica

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Ruxolitinib
Aliases & brands
JakafiOpzelura
RxNorm CUI
1193326
ChEMBL ID
CHEMBL1789941
ATC codes
D11AH09L01EJ01
UNII
82S8X8XX8H
Regulatory jurisdictions
fdaema

Regulatory timeline

31 events

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Approval / market entry confirmed across FDA and EMA (2011-11-16 → 2013-11-07)
Earliest approval
2011-11-16
Latest approval
2026-05-01
Authorities
FDA · EMA
Total events
31
fdaU.S. Food and Drug Administration· 29 events
2026-07-20Label change
Label change: RUXOLITINIB (NDA202192)
Evidence ↗
2026-07-20Label change
Label change: RUXOLITINIB (NDA217180)
Evidence ↗
2026-06-30Label change
Label change: RUXOLITINIB (NDA215309)
Evidence ↗
emaEuropean Medicines Agency· 2 events
2026-07-20CHMP positive opinion
CHMP positive opinion: Ruxolitinib Viatris (EMA)

Indication: Treatment of myelofibrosis (MF), polycythaemia vera (PV) and Graft versus host disease (GvHD).

Evidence ↗
2012-08-23ApprovalMulti-authority
Approval: Jakavi (EMA)
Indication: Myelofibrosis (MF)Jakavi is indicated for the treatment of disease related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic… Show full indication

Myelofibrosis (MF)Jakavi is indicated for the treatment of disease related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis.Polycythaemia vera (PV)Jakavi is indicated for the treatment of adult patients with polycythaemia vera who are resistant to or intolerant of hydroxyurea.Graft versus host disease (GvHD)Acute GvHDJakavi is indicated for the treatment of adults and paediatric patients aged 28 days and older with acute graft versus host disease who have inadequate response to corticosteroids or other systemic therapies (see section 5.1).Chronic GvHDJakavi is indicated for the treatment of adults and paediatric patients aged 6 months and older with chronic graft versus host disease who have inadequate response to corticosteroids or other systemic therapies (see section 5.1). Myelofibrosis (MF)Jakavi is indicated for the treatment of disease related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis.Polycythaemia vera (PV)Jakavi is indicated for the treatment of adult patients with polycythaemia vera who are resistant to or intolerant of hydroxyurea.Graft versus host disease (GvHD)Acute GvHDJakavi is indicated for the treatment of adults and paediatric patients aged 28 days and older with acute graft versus host disease who have inadequate response to corticosteroids or other systemic therapies (see section 5.1).Chronic GvHDJakavi is indicated for the treatment of adults and paediatric patients aged 6 months and older with chronic graft versus host disease who have inadequate response to corticosteroids or other systemic therapies (see section 5.1).

Evidence ↗

Data from the U.S. Food and Drug Administration (U.S. Food and Drug Administration), public domain (CC0).

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

199 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
199
registered trials across all phases
LATEST COMPLETION 2025
78
Active studies
59
Recruiting
39
Late-stage (III+)
69
Completed
52
Discontinued
PHASE DISTRIBUTIONn = 199
Early Phase 12Phase 139Phase 1 / 234Phase 285Phase 2 / 34Phase 324Phase 48Phase N / A3

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

Open Label, Multi-center, Phase IV Study of Ruxolitinib or Ruxolitinib and Panobinostat Combination, for Patients Who Have Completed Prior Global Novartis or Incyte Sponsored Studies

Active, not recruitingPH IVNovartis Pharmaceuticalsn=2792032

A Single-arm Multi-center Study of Ruxolitinib in Chinese Participants With Corticosteroid-refractory Chronic Graft Versus Host Disease After Allogeneic Stem Cell Transplantation

RecruitingPH IVNovartis Pharmaceuticalsn=502031

A Single-arm, Multi-center Study of Ruxolitinib for the Treatment of Chinese Patients With Grade II-IV Corticosteroid-refractory Acute Graft Versus Host Disease

RecruitingPH IVNovartis Pharmaceuticalsn=362028

Opzelura Experience Study

Not yet recruitingPH IVWake Forest University Health Sciencesn=502027

Clinical Study on the Treatment of Pediatric Hemophagocytic Lymphohistiocytosis Based on Cytokine Guided Risk Stratification：A Multicenter Randomized Controlled Study

Unknown statusPH IVThe Children's Hospital of Zhejiang University School of Medicinen=4002025

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase 1 Trial of Combined MEK, STAT3 and PD-1 Inhibition in Metastatic Pancreatic Ductal Adenocarcinoma

CompletedPH IPeter Hosein, MDn=282025

A Phase II Study of Ruxolitinib Pre-, During- and Post-Hematopoietic Stem Cell Transplantation for Patients With Primary or Secondary Myelofibrosis.

CompletedPH IIMassachusetts General Hospitaln=442025

A Randomized, Multicenter, Open-label Phase II Trial to Compare Prophylaxis of Graft Versus Host Disease With Tacrolimus and Mycophenolate Mofetil Versus Ruxolitinib After Post-transplant Cyclophosphamide

CompletedPH IISt. Petersburg State Pavlov Medical Universityn=1282025

Single Patient Study of JAK/STAT Inhibition in CNS Kohlmeier-Degos Disease

CompletedPH I/IINational Heart, Lung, and Blood Institute (NHLBI)n=12025

A Phase 2 Open-Label Study Evaluating Tolerability and Efficacy of Navitoclax Alone or in Combination With Ruxolitinib in Subjects With Myelofibrosis (REFINE)

CompletedPH IIAbbVien=1912025

A Phase 1/2 Study of CPI-0610, a Small Molecule Inhibitor of BET Proteins: Phase 1 (Dose Escalation of CPI-0610 in Patients With Hematological Malignancies) and Phase 2 (Dose Expansion of CPI-0610 With and Without Ruxolitinib in Patients With Myeloproliferative Neoplasms)

CompletedPH I/IIConstellation Pharmaceuticalsn=3362025

Research activity

6 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20122024
Most influential

A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis.

The New England journal of medicine · 2012 · 1,632 cites

Efficacy, safety, and survival with ruxolitinib in patients with myelofibrosis: results of a median 3-year follow-up of COMFORT-I.

Haematologica · 2015 · 195 cites

Long-term outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: survival advantage in comparison to matched historical controls.

Blood · 2012 · 169 cites

JAK inhibition enhances checkpoint blockade immunotherapy in patients with Hodgkin lymphoma.

Science (New York, N.Y.) · 2024 · 139 cites

Efficacy, safety and survival with ruxolitinib in patients with myelofibrosis: results of a median 2-year follow-up of COMFORT-I.

Haematologica · 2013 · 128 cites

A phase I study of panobinostat and ruxolitinib in patients with primary myelofibrosis (PMF) and post--polycythemia vera/essential thrombocythemia myelofibrosis (post--PV/ET MF).

Leukemia research · 2020 · 21 cites
Recent papers

JAK inhibition enhances checkpoint blockade immunotherapy in patients with Hodgkin lymphoma.

Science (New York, N.Y.) · 2024 · 139 cites

A phase I study of panobinostat and ruxolitinib in patients with primary myelofibrosis (PMF) and post--polycythemia vera/essential thrombocythemia myelofibrosis (post--PV/ET MF).

Leukemia research · 2020 · 21 cites

Efficacy, safety, and survival with ruxolitinib in patients with myelofibrosis: results of a median 3-year follow-up of COMFORT-I.

Haematologica · 2015 · 195 cites

Efficacy, safety and survival with ruxolitinib in patients with myelofibrosis: results of a median 2-year follow-up of COMFORT-I.

Haematologica · 2013 · 128 cites

A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis.

The New England journal of medicine · 2012 · 1,632 cites

Long-term outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: survival advantage in comparison to matched historical controls.

Blood · 2012 · 169 cites
Major research themes8
Hodgkin Disease1Immune Checkpoint Inhibitors1Janus Kinase Inhibitors1Nitriles1Nivolumab1Pyrazoles1Pyrimidines1T-Lymphocytes1
Journals, researchers & institutions
Top journals
  • Haematologica2
  • Blood1
  • Leukemia research1
  • Science (New York, N.Y.)1
  • The New England journal of medicine1
Leading researchers
  • Verstovsek S4
  • Arcasoy MO3
  • Catalano JV3
  • DiPersio JF3
  • Gotlib J3
  • Gupta V3
  • Harvey JH Jr3
  • Kantarjian HM3
Leading institutions
free-text, unnormalised
  • The University of Texas MD Anderson Cancer Center2
  • *Oregon Health and Science University1
  • Abramson Cancer Center at the University of Pennsylvania1
  • Cancer Care Centers of South Texas/US Oncology1
  • Columbia Presbyterian Medical Center1
  • Duke University Health System1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.