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.
“Nearly half of the people who develop chronic GVHD do not respond adequately to steroids—the current standard of care—making this life-threatening condition particularly challenging to treat,” said Robert Zeiser, MD, Section Head, Tumor Immunology and Immune Modulation, University Medical Center Freiburg, Germany, and the principal investigator of the REACH3 trial. “In this clinical trial, treatment with Jakafi demonstrated significantly improved outcomes across a range of efficacy measures compared to best available therapy. This approval represents a significant advancement in the treatment of appropriate patients with chronic GVHD—for both the patients who face a poor prognosis and the healthcare providers who struggle to effectively treat them.”
This new approval was based on the REACH3 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 ORR through cycle 7 of day 1. The ORR was 70% (95% CI, 63%-77%) with ruxolitinib versus 57% (95% CI, 49%-65%) with best available therapy. The median DOR 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 AEs with ruxolitinib in this study were anemia and thrombocytopenia. The most common (≥20%) nonhematologic adverse reactions were infections and viral infection.
The recommended ruxolitinib starting dose for this indication is 10 mg given orally twice daily.