FDA Approves Ruxolitinib for the Treatment of Previously Treated Chronic Graft-Versus-Host Disease

Patients with chronic graft-versus-host disease who have received prior therapy can now receive treatment with ruxolitinib following its approval by the FDA.

Ruxolitinib (Jakafi) has received approval from the FDA for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host disease (GVHD) following failure of 1 to 2 lines of systemic therapy, according to a press release from drug developer Incyte.

The approval was based on findings from the open-label, multicenter phase 3 REACH3 study (NCT03112603), which compared the use of ruxolitinib to best available therapy in those with steroid-refractory GVHD following allogenic stem cell transplant. Notably, the primary end point of overall response rate (ORR) at 24-weeks was 49.7% in the ruxolitinib arm vs 25.6% in the best available therapy arm (P <.0001). Additionally, the ORR on cycle 7, day 1 was 70% for the experimental cohort and 57% for the control group.

“GVHD is the leading cause of morbidity and mortality in patients following an allogeneic stem cell transplant, yet there historically have been limited treatment options available beyond first-line systemic therapies,” Steven Stein, MD, chief medical officer at Incyte, said in a press release. “Incyte is proud to have contributed to the overall scientific understanding of GVHD through our REACH program, which has led to important treatment advances on behalf of patients and the medical community, including today’s approval of Jakafi for certain people who develop chronic GVHD.”


Incyte announces FDA approval of Jakafi® (ruxolitinib) for treatment of chronic graft-versus-host disease (GVHD). News release. Incyte. September 22, 2021. Accessed September 22, 2021. https://bit.ly/3lPathJ