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Ruxolitinib

Ruxolitinib was the first JAK inhibitor: it shrinks the spleen and relieves symptoms in myelofibrosis and controls blood counts in polycythaemia vera, without eliminating the disease clone.

COMFORT-I/II (2011) showed spleen volume and symptom benefit versus placebo/best available therapy with a later-demonstrated survival advantage; RESPONSE (2014) established it in hydroxyurea-resistant PV. Also approved in acute and chronic graft-versus-host disease (REACH2/3). Anaemia, thrombocytopenia, infections and a discontinuation syndrome are the main issues.

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Ruxolitinib
PubChem
Modality
Small-molecule JAK1/JAK2 inhibitor
Mechanism
ATP-competitive inhibition of JAK1 and JAK2, dampening JAK-STAT signalling regardless of JAK2/CALR/MPL driver.
Brand / code
Jakafi / Jakavi
Medicare
Part D (self-administered)

Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026). Jakafi is a Part D specialty drug; generic entry is not expected before 2028.

Commercial insurance
covered with prior authorisation

Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy.

Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.

Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.

NICE recommendedNICE TA386 · 2016SMC: accepted
Appraised for
Disease-related splenomegaly or symptoms in myelofibrosis (intermediate-2 or high risk)
Notes
Polycythaemia vera resistant or intolerant to hydroxycarbamide appraised 2023. Steroid-refractory acute and chronic GVHD appraised 2023-24.
NHS England
Routinely funded for the appraised indication (or via managed access)

Sources: NICE TA386 · SMC advice: ruxolitinib. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

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Approvals

RegionYearIndication
US2011Intermediate/high-risk myelofibrosis
US2014Polycythaemia vera after hydroxyurea
US2019Steroid-refractory acute GVHD
US2021Chronic GVHD after failure of 1-2 lines
EU2012Myelofibrosis

Trials

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ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
332 studies22 recruiting78 Phase 226 Phase 3
Search “Ruxolitinib” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Ruxolitinib
intervention: Ruxolitinib
Open on ClinicalTrials.gov →

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Key papers

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Latest papers

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Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Ruxolitinib" OR ABSTRACT:"Ruxolitinib" OR TITLE:"Jakafi" OR ABSTRACT:"Jakafi" OR TITLE:"Jakavi" OR ABSTRACT:"Jakavi") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Ruxolitinib, not a curated reading list.

Connected

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