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FDA approval of ruxolitinib for the treatment of patients with chronic GvHD

Sep 24, 2021

On September 22, 2021, it was announced that the U.S. Food and Drug Administration (FDA) approved the use of ruxolitinib for the treatment of patients with chronic graft-versus-host disease (cGvHD). The approval is for patients ≥12 years of age who have previously received one or two lines of systemic therapy.1

Ruxolitinib is a first-in-class JAK1/JAK2 inhibitor that is already approved by the FDA for the treatment of polycythemia vera and myelofibrosis in adults, and for steroid-refractory acute GvHD in patients ≥12 years of age. The supplemental new drug application for cGvHD was granted in February 2021, under the priority review program.2

This latest approval was based on results from the REACH3 study (NCT03112603); a phase III, open-label, multicenter study evaluating the efficacy of ruxolitinib compared with best available therapy (BAT) in patients ≥12 years of age with steroid-refractory cGvHD.1 The results of this trial were previously reported on the GvHD Hub and were published in the New England Journal of Medicine in July 2021.3

The main outcome used to support this approval was the overall response rate through Cycle 7 Day 1, which was 13% higher in the ruxolitinib arm compared with the BAT arm (70% vs 57%, respectively). The median duration of response (from first response to progression, death, or new systemic therapies) was twice as long in the ruxolitinib arm compared with the BAT arm (4.2 months vs 2.1 months, respectively). For the ruxolitinib arm, the most common hematologic adverse reactions (occurring in >35% of patients) were anemia and thrombocytopenia, while the most common nonhematologic adverse reactions (occurring in ≥20% of patients) were infections and viral infection.

  1. U.S. Food and Drug Administration. FDA approves ruxolitinib for chronic graft-versus-host disease. Published Sep 22, 2021. Accessed Sep 23, 2021.
  2. Incyte announces FDA approval of Jakafi® (ruxolitinib) for treatment of chronic graft-versus-host disease (GVHD). Published Sep 22, 2021. Accessed Sep 23, 2021.
  3. Zeiser R, Polverelli N, Ram R, et al. Ruxolitinib for glucocorticoid-refractory chronic graft-versus-host disease. N Engl J Med. 2021;385(3):228-238. DOI: 1056/NEJMoa2033122