FDA Approves First Treatment for Steroid-Refractory Acute Graft-Versus-Host Disease

Officials with the FDA approved ruxolitinib (Jakafi, Incyte), the first treatment for patients 12 years and older with steroid-refractory acute graft-versus-host disease.

Officials with the FDA approved ruxolitinib (Jakafi, Incyte), the first treatment for patients 12 years and older with steroid-refractory acute graft-versus-host disease (GVHD), according to a press release.

Ruxolitinib was previously granted Breakthrough Therapy Designation and Orphan Drug Designation by the FDA for this indication.

GVHD can occur after an allogeneic stem cell transplant when the donated cells initiate an immune response and attack the transplant recipient’s orgrans. The condition, which can be acute or chronic, can affect multiple organ systems including the skin, gastrointestinal tract, and liver. Patients with steroid-refractory acute GVHD can progress to severe disease that leads to significant morbidity and mortality, with 1-year mortality rates of approximately 70%, according to Incyte.

The approval was based on data from the REACH1 clinical trial evaluating ruxolitinib in combination with corticosteroids in patients with steroid-refractory grade 3 and 4 acute GVHD.

For the study, 71 patients were recruited into the trial and ruxolitinib was administered at 5 mg twice daily, with the dose increased to 10 mg twice daily after 3 days in the absence of toxicity. Of these patients, 49 were refractory to steroids alone, 12 had received 2 or more prior anti-GVHD therapies, and 10 did not otherwise meet the FDA definition of steroid-refractory.

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

Adverse effects most frequently reported in the study were infections and edema and the most common laboratory abnormalities were anemia, thrombocytopenia, and neutropenia.

“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,” lead investigator Madan Jagasia, MBBS, MS, MMHC, professor of medicine at Vanderbilt University Medical Center, department of medicine, division of hematology-oncology, and chief medical officer at Vanderbilt-Ingram Cancer Center, said 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 onw, have had limited choices.”

The REACH clinical program also includes phase 3 trials in patients with steroid-refractory acute GVHD (REACH2) and steroid-refractory chronic GVHD (REACH3). Results from both these trials are expected before the end of 2019, according to Incyte.


FDA Approves Jakafi (ruxolitinib) for the Treatment of Patients with Acute Graft-Versus-Host Disease [news release]. Incyte. 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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