On September 22, 2021, the U.S. Food and Drug Administration (FDA) approved ruxolitinib (Jakafi®) for chronic graft-versus-host disease (cGVHD) after failure of one or two lines of systemic therapy in adult and pediatric patients aged 12 years and older.

FDA Approves Ruxolitinib for Chronic Graft-Versus-Host Disease

Efficacy was evaluated in REACH-3 (NCT03112603), a randomized, open-label, multicenter clinical trial comparing ruxolitinib to best available therapy (BAT) for corticosteroid-refractory cGVHD after allogeneic stem cell transplantation. The trial randomized 329 patients 1:1 to receive either ruxolitinib 10 mg twice daily or BAT.

The major efficacy outcome was overall response rate (ORR) through cycle 7, day 1. ORR was 70% (95% CI = 63%, 77%) for the ruxolitinib arm and 57% (95% CI = 49%, 65%) for the BAT arm, with a difference of 13% (95% CI = 3%, 23%). The median duration of response, calculated from first response to progression, death, or new systemic therapies for cGVHD, was 4.2 months (95% CI = 3.2, 6.7) and 2.1 months (95% CI - 1.6, 3.2) for the ruxolitinib and BAT arms, respectively. The median time from first response to death or new systemic therapies for cGVHD was 25 months (95% CI = 16.8, not estimable) and 5.6 months (95% CI = 4.1, 7.8) for the ruxolitinib and BAT arms, respectively.

In cGVHD, the most common (> 35%) hematologic adverse reactions to ruxolitinib were anemia and thrombocytopenia. The most common (≥ 20%) nonhematologic adverse reactions were infections and viral infection.

The recommended starting dose of ruxolitinib for cGVHD is 10 mg given orally twice daily.

View full prescribing information for ruxolitinib.

The review that led to the approval was conducted under Project Orbis, an initiative of FDA’s Oncology Center of Excellence (OCE). Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration, Brazilian Health Regulatory Agency, Health Canada, Switzerland’s Swissmedic, and United Kingdom’s Medicines and Healthcare pPoducts Regulatory Agency. The application reviews are ongoing at the other regulatory agencies.

Ruxolitinib’s application was granted priority review and orphan product designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.