FDA Approves Ruxolitinib for Chronic Graft-Versus-Host Disease

Graft-versus-host disease.

The FDA has approved ruxolitinib (Jakafi®, Incyte) for adult and pediatric patients 12 year of age and older with chronic graft-versus-host disease (cGVHD) after failure of one or two lines of systemic therapy.

“Chronic graft-versus-host disease, a major complication of allogeneic stem cell transplantation, becomes glucocorticoid-refractory or glucocorticoid-dependent in approximately 50% of patients,” wrote Robert Zeiser, MD, Professor and Head of the Tumor Immunology and Immune Modulation at the University of Freiberg Medical Center in Germany, and colleagues, in their published results of the REACH-3 clinical trial (NCT03112603), on which the approval was based. “In retrospective surveys, ruxolitinib, a Janus kinase inhibitor, showed potential efficacy in patients with glucocorticoid-refractory or -dependent cGVHD.”

This randomized, open-label, multicenter phase 3 clinical trial enrolled 329 patients, who were randomized 1:1 to receive either 10 mg ruxolitinib twice daily or best available therapy. The primary end point, which the approval was based on, was overall response rate through Cycle 7, Day 1 of treatment.

Ruxolitinib produced an overall response rate of 70%, compared with 57% in the best available therapy treatment arm. The median durations of response were 4.2 months and 2.1 months, respectively. The median times from first response to death or new systemic therapies for cGVHD were 25.0 months and 5.6 months, respectively.

In patients with cGVHD, the most common hematologic adverse reactions occurring in more than 35% of patients were anemia and thrombocytopenia; the most common nonhematologic adverse reactions occurring in at least 20% of patients were infections and viral infections.

“Among patients with glucocorticoid-refractory or -dependent cGVHD, ruxolitinib led to significantly greater overall response, failure-free survival, and symptom response,” concluded Dr. Zeiser and colleagues in their July 2021 article published in The New England Journal of Medicine.

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

For More Information

Zeiser R, Polverelli N, Ram R, et al (2021). Ruxolitinib for glucocorticoid-refractory chronic graft-verus-host disease. N Engl J Med, 385(3):228-238. DOI:10.1056/NEJMoa2033122

Clinicaltrials.gov (2021). A study of ruxolitinib vs best available therapy (BAT) in patients with steroid-refractory chronic graft vs host disease (GvHD) after bone marrow transplantation. NLM identifier: NCT03112603.

Jakafi® (ruxolitinib) prescribing information (2021). Incyte. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/202192s023lbl.pdf

US Food & Drug Administration (2021). FDA approves ruxolitinib for chronic graft-versus-host disease. Available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-ruxolitinib-chronic-graft-versus-host-disease

Image credit: Mark ong. Licensed under CC BY-SA 4.0

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