Ruxolitinib is marketed as JAKAFI, an oral kinase inhibitor used for myelofibrosis, polycythemia vera, and steroid-refractory acute or chronic graft-versus-host disease in specified age groups. It inhibits JAK1 and JAK2 signaling pathways relevant to hematopoiesis and immune function. U.S. initial approval was in 2011.
Ruxolitinib remains the standard frontline therapy for intermediate-2 and high-risk myelofibrosis, with phase 3 and real-world data showing spleen and symptom benefits. Cytopenias, transfusion needs and resistance after 2 to 3 years continue to drive interest in new combinations.
CAR-T toxicity management is shifting toward phenotype- and mechanism-based intervention. Key issues include CRS, ICANS, IEC-HS, and long-term risks of cytopenia and infection.
The myelofibrosis therapeutic pipeline now includes over 40 candidates from more than 35 companies, with recent developments including Orphan Drug Designation for CK0804 and multiple Phase III trial initiations. Key clinical milestones include completed enrollment in Karyopharm's SENTRY trial and upcoming Phase I data from Incyte's combination therapy studies.
DelveInsight pipeline reports reveal robust clinical development across three cancer indications, with 45+ companies advancing cutaneous squamous cell carcinoma therapies, 70+ companies developing liver cancer treatments, and multiple hepatocellular carcinoma candidates receiving FDA designations in 2025-2026.
The FDA has accepted the New Drug Application for rusfertide, a first-in-class hepcidin mimetic peptide for polycythemia vera, and granted Priority Review with a PDUFA goal date in the third quarter of 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07588945 |
daGOAT-Guided Prevention of Severe aGVHD After Allo-HSCT |
NOT_YET_RECRUITING | PHASE3 |
| NCT07588139 |
A Study to Evaluate Dermal Open-Flow Microperfusion and Plasma Pharmacokinetic Study of Multiple Doses of Oral Povorcitinib or Topical Ruxolitinib Cream in Healthy Adult Participants |
RECRUITING | PHASE1 |
| NCT07566897 |
A Phase I Study to Evaluate the Safety and Efficacy of L19IL2 in Combination With Ruxolitinib in Patients With Advanced Solid Tumors |
NOT_YET_RECRUITING | PHASE1 |
| NCT07548983 |
Ruxolitinib With Azacitidine Maintenance for the Treatment of Patients With Acute Myeloid Leukemia Undergoing Reduced Intensity Allogeneic Stem Cell Transplantation |
NOT_YET_RECRUITING | PHASE1 |
| NCT07521046 |
Tolerability of Ropeginterferon Alfa-2b Add-on to Ongoing Ruxolitinib Therapy in Myelofibrosis (RopeRux in Myelofibrosis) |
NOT_YET_RECRUITING | PHASE1 |
| NCT07498205 |
Comparing Momelotinib and Ruxolitinib in People With Untreated Myelofibrosis and Low Blood Cell Counts |
NOT_YET_RECRUITING | PHASE4 |
| NCT07424222 |
Ruxolitinib for Immune Effector Cell Associated Hemophagocytic Lymphohistiocytosis-like Syndrome (RISE) |
NOT_YET_RECRUITING | PHASE1 |
| NCT07359859 |
A Study of Ruxolitinib for Preventing Graft-Versus-Host Disease in People With a Hematologic Malignancy Who Will Receive a Stem Cell Transplant |
RECRUITING | PHASE2 |
| NCT07357727 |
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF) |
RECRUITING | PHASE3 |
| NCT07356245 |
Ruxolitinib Maintenance Post-Hematopoietic Stem Cell Transplant T-Cell Lymphoma |
RECRUITING | PHASE2 |
