FDA Approves Ruxolitinib for Graft-Versus-Host-Disease


The FDA has approved ruxolitinib to treat adults with steroid-refractory chronic graft-versus-host disease.

The FDA approved ruxolitinib (Jakafi) for patients with steroid-refractory chronic graft-versus-host disease (GVHD) aged 12 years or older whose disease has not responded to 1 or 2 lines of previous therapy.1

The approval is based on findings from the phase 3, randomized REACH3 study, in which ruxolitinib generated a significantly higher overall response rate (ORR) in comparison to the best available therapy (BAT) offered to the control arm of the study. The primary end point at week 24 showed an ORR at 49.7% for the investigative arm and 25.6% for the control arm in patients with steroid-refractory chronic GVHD.

“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 principal investigator Robert Zeiser, MD, Department of Hematology, Oncology, and Stem Cell Transplantation, University Medical Center Freiburg, in a press release. “In this clinical trial, treatment with Jakafi demonstrated significantly improved outcomes across a range of efficacy measures compared to best available therapy.”

The findings also showed a relationship between ruxolitinib and a longer median failure-free survival (FFS) than existed with BAT at week 24. The JAK2 inhibitor had not yet reached FFS whereas BAT FFS had been reached at 5.7 months (HR, 0.370; 95% CR, 0.268-0.510; P < .0001).

Furthermore, compared with BAT, ruxolitinib elicited a more substantial improvement of symptoms at week 24. As per the modified Lee Symptom Scale (mLSS), 24% of participants in the investigational arm of the study reported a significant decrease in symptoms while only 11% of participants in the control arm reported similarly (OR, 2.62; 95% CI, 1.42-4.82; P = .0011). The best reported ORR in patients receiving ruxolitinitb was 76.4%.

While rates of death leading to the data cutoff proved to be comparable between both the investigative and control arms (18.8% vs 16.5%, respectively), serious toxicities emerged in 36.7% of those receiving BAT as opposed to 33.3% with those receiving ruxolitinib.

Participants in the investigative arm reported a slightly higher rate of all-grade adverse events (AEs) at 97.6% whereas those receiving chemotherapy reported 91.8%. Even so, rates of grade 3 or higher toxicities were found in 57.0% of the ruxolitinib participants and 57.6 of the BAT participants.

The most notable AEs reported were low red or white blood cell counts and infections, including viral infections.

“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,” Zeizer concluded.


  • Incyte announces acceptance and priority review of SNDA for Jakafi (ruxolitinib) as a treatment for patients with chronic graft-versus-host disease. News release. Incyte. February 22, 2021. Accessed February 22, 2021. http://bwnews.pr/3pFaDs1.

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