On September 22, 2021, the FDA approved a new indication for ruxolitinib (Jakafi; Incyte) for the treatment of patients aged ≥12 years with chronic graft-versus-host disease (GVHD) after 1 or 2 lines of systemic therapy. Ruxolitinib received an orphan drug designation for this indication.
Ruxolitinib was previously approved for several indications. This new approval was based on the REACH-3 study, a randomized, open-label, multicenter clinical trial of ruxolitinib versus best available therapy for corticosteroid-refractory chronic GVHD after allogeneic stem-cell transplant.
The study included 329 patients who were randomized in a 1:1 ratio to ruxolitinib 10 mg twice daily or to best available therapy. The major efficacy outcome was overall response rate (ORR) through cycle 7 of day 1. The ORR was 70% (95% confidence interval [CI], 63%-77%) with ruxolitinib versus 57% (95% CI, 49%-65%) with best available therapy. The median duration of response was 4.2 months with ruxolitinib and 2.1 months with best available therapy. The median time from first response to death or initiation of new systemic therapy for chronic GVHD was 25 months versus 5.6 months, respectively.
The most common (>35%) hematologic adverse events with ruxolitinib in this study were anemia and thrombocytopenia. The most common (≥20%) nonhematologic adverse reactions were infections and viral infection.