On September 22, 2021, the Food and Drug Administration approved ruxolitinib (Jakafi, Incyte Corp.) for chronic graft-versus-host disease (cGVHD) after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older.
Efficacy was evaluated in REACH-3 (NCT03112603), a randomized, open-label, multicenter clinical trial of ruxolitinib compared 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 used to support approval was overall response rate (ORR) (2014 NIH Response Criteria) through cycle 7 day 1. The ORR was 70% (95% CI 63%, 77%) for the ruxolitinib arm and 57% (95% CI 49%, 65%) for the BAT arm with a difference in response rate of 13% (95% CI 3%, 23%). The median durations of response, calculated from first response to progression, death, or new systemic therapies for chronic GVHD, were 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 times from first response to death or new systemic therapies for cGVHD were 25 months (95% CI 16.8, NE) and 5.6 months (95% CI 4.1, 7.8) for the ruxolitinib and BAT arms, respectively.