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


Ruxolitinib is the first and only FDA-approved treatment for this indication, according to Incyte, the drug's manufacturer.

Officials with the FDA have approved Incyte’s ruxolitinib (Jakafi) for the treatment of steroid-refractory acute graft-versus-host disease (GVHD) in adult and pediatric patients, age 12 years and older, the company announced today.

Ruxolitinib is the first and only FDA-approved treatment for this indication, according to Incyte.

“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,” said Hervé Hoppenot, Chief Executive Officer, Incyte, in a prepared statement.

The approval was based on data from REACH1, an open-label, single-arm, multicenter study of ruxolitinib in combination with corticosteroids in patients with steroid-refractory grade II-IV acute GVHD. Forty-nine of 71 patients recruited into REACH1 were refractory to steroids alone, 12 patients had received 2 or more prior anti-GVHD therapies and 10 patients did not otherwise meet the FDA definition of steroid-refractory. Ruxolitinib was administered at 5 mg twice daily, and the dose could be increased to 10 mg twice daily after three days in the absence of toxicity.

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

The most frequently reported adverse reactions among all 71 study participants were infections (55%) and edema (51%), and the most 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 prepared 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,” Jagasia added.

The FDA previously granted Incyte’s ruxolitinib application Breakthrough Therapy Designation and Orphan Drug Designation for the treatment of patients with steroid-refractory acute GVHD, and the supplemental New Drug Application (sNDA) was reviewed under the FDA’s Priority Review program.

Ruxolitinib will be made available to appropriate patients with steroid-refractory acute GVHD immediately, according to Incyte.


FDA Approves Jakafi® (ruxolitinib) for the Treatment of Patients with Acute Graft-Versus-Host Disease [news release]. Wilmington, DE; May 24, 2019: Incyte website. https://investor.incyte.com/news-releases/news-release-details/fda-approves-jakafir-ruxolitinib-treatment-patients-acute-graft?rel=0. Accessed May 24, 2019.

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