FDA Approves Ruxolitinib for GVHD Treatment

The FDA approved ruxolitinib (Jakafi) for the treatment of patients 12 years or older with steroid-refractory acute graft-versus-host disease (GVHD), according to Incyte, the manufacturer of the JAK1/JAK2 inhibitor.

This is the first — and only – drug to be approved in this space.

“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,” stated Hervé Hoppenot, chief executive officer, Incyte, in a statement.

“This approval is also an important milestone for Incyte, as it marks the third indication for Jakafi in the United States, further underscoring Incyte’s commitment to delivering innovative medicines for patients in need. We are proud of the impact Jakafi has had on patients’ lives to-date and are dedicated to advancing our ongoing research in JAK inhibition to serve more GVHD patients in the future.”

The approval is based off results from the open-label, single-arm, multicenter REACH1 trial, which examined ruxolitinib in combination with corticosteroids in patients who had steroid-refractory grade II to IV acute GVHD. The trial included 71 patients — 49 who were refractory to steroids alone, 12 who had 2 or more prior GVHD treatments, and 10 who did not meet the FDA criteria for GVHD. Patients were given 5 mg of ruxolitinib twice a day. If patients did not have any toxicities, their dosage could be increased to 10 mg twice a day.

Researchers on the REACH1 trial evaluated the drug’s efficacy based on the day 28 overall response rate (ORR), which was defined as a complete response (CR), very good partial response, or partial response based on the Center for International Blood and Marrow Transplant Research (CIBMTR). For the group that was refractory to steroids alone, there was a 57% ORR, and a 31% CR.

Common adverse events (AEs) seen in the study were infections (55%) and edema (51%). Common laboratory abnormalities were anemia (75%), thrombocytopenia (75%), and neutropenia (58%).

“Every year in the United States, about half of the people who develop acute GVHD do not respond adequately to steroids, making it an extremely challenging disease to treat,” said Madan Jagasia, MBBS, MS, MMHC, a lead investigator on the REACH1 trial and professor of medicine, Vanderbilt University Medical Center, Department of Medicine, Division of Hematology-Oncology and Chief Medical Officer, Vanderbilt-Ingram Cancer Center, in a statement.

“While allogeneic stem cell transplants have the potential to transform people’s lives, the onset of acute GVHD can significantly impact their prognosis. I am excited that we now have Jakafi as a new treatment option for acute GVHD patients that do not respond to corticosteroids who, until now, have had limited choices.”