FDA Approves Jakafi for the Treatment of Patients with Acute Graft-Versus-Host Disease

The Food and Drug Administration (FDA) approved Jakafi (ruxolitinib), the first and only treatment of steroid-refractory acute graft-versus-host disease (GVHD) in adult and pediatric patients aged 12 years and older, according to Incyte — the drug’s manufacturer.

The approval — which marks the third indication for Jakafi in the United States – was based on data from the open-label, single-arm, multicenter REACH1 study, designed to evaluate Jakafi in combination with corticosteroids in patients with steroid-refractory grade 2 to 4 acute GVHD.

Forty-nine of the 71 patients who participated in REACH1 were refractory to steroids alone, 12 patients had received two or more prior anti-GVHD therapies and 10 patients did not otherwise meet the FDA definition of steroid-refractory. Jakafi was administered at 5 mg twice daily, and the dose could be doubled to 10 mg twice daily after three days if there were no signs of toxicity.

The efficacy of Jakafi was evaluated based upon day 28 overall response rate, defined as a complete response, very good partial response or partial response based on the Center for International Blood and Marrow Transplant Research (CIBMTR) criteria. In the 49 patients refractory to steroids alone, overall response rate was 57% with a complete response rate of 31%.

The most frequently reported side effects among all 71 participants in the study were infections (55%) and edema (51%). The most common hematologic abnormalities were anemia (75%), thrombocytopenia (75%) and neutropenia (58%).

“For the first time, patients with steroid-refractory acute GVHD, and the physicians that treat them, have an FDA-approved treatment for this serious disease,” Hervé Hoppenot, chief executive officer of Incyte, said in a press release.

Acute GVHD is a condition that can occur after an allogeneic or donor stem cell transplant where the donated cells initiate an immune response and attack the transplant recipient’s organs, including the skin, gastrointestinal tract and liver.

Nearly 50% of patients with acute GVHD do not respond adequately to corticosteroid therapy (the current standard of care) and can progress to severe disease, with one-year mortality rates of approximately 70%.