Ruxolitinib Approved for Chronic Graft-Versus-Host Disease

The FDA has approved ruxolitinib for the treatment of chronic graft-versus-host disease (cGVHD) in adult and pediatric patients 12 years and older whose disease is refractory to one or two lines of systemic therapy.

This decision is supported by efficacy data from the REACH-3 trial comparing 10 mg daily ruxolitinib to best available therapy (BAT) for 329 patients whose cGVHD was refractory to corticosteroids after allogeneic stem cell transplantation. Overall response rate (ORR), as defined by 2014 NIH Response Criteria, was 70% (95% CI 63-77%) for patients treated with ruxolitinib, compared with 57% (95% CI 49- 65%) in the BAT arm. Median duration of response from first response to progression, death, or new systemic therapy for cGVHD was 4.2 months (95% CI 3.2-6.7) in the ruxolitinib group and 2.1 months (95% CI 1.6-3.2) for patients receiving BAT. The median time from first response to death or new systemic therapy in the ruxolitinib and BAT arms was 25 months (95% CI 16.8 to not estimable) and 5.6 months (95% CI 4.1-7.8), respectively.

Anemia and thrombocytopenia occurred in >35% of patients receiving ruxolitinib. The most common nonhematologic AEs, occurring in ≥20% of patients, were infections.

Recommended starting dose of ruxolitinib for the treatment of cGVHD is 10 mg orally twice daily.

Source: FDA press release, September 22, 2021.