FDA Approves Jakafi for Chronic GVHD

The Food and Drug Administration (FDA) has granted approval to Jakafi (ruxolitinib) for the treatment of adult and pediatric patients 12 years and older with steroid-refractory chronic graft-versus-host disease (GVHD) after failure of one or two lines of systemic therapy.

GVHD is a common side effect experienced in patients who have had an allogeneic transplant. With this condition, the donated blood marrow or blood stem cells view the patient’s body as a foreign threat and begin to attack it, appearing as symptoms like skin rash, yellow discoloration of the skin or eyes, nausea, vomiting, diarrhea or abdominal cramping.

“Glucocorticoid-refractory chronic graft-versus-host disease is the leading cause of reduced quality of life and non-relapse mortality in patients surviving longer than two years after allogeneic hematopoietic cell transplantation. I am very happy and thankful that we could observe that ruxolitinib led to significantly greater overall response, failure-free survival, and symptom response compared to currently available therapy in such patients,” said lead study author Dr. Robert Zeiser of the University Medical Center Freiburg Department of Hematology, Oncology and Stem Cell Transplantation in Freiburg, Germany, in an interview with CURE®.

The data that led to Jakafi’s approval came from the phase 3 REACH3 trial, in which the overall response rate (ORR) – or proportion of patients whose disease responded to therapy – was 49.7% after 24 weeks with Jakafi compared with 25.6% with the best available therapy (BAT). On cycle seven day one, the ORR was 70% with Jakafi and 57% with BAT.

The most common blood-related side effects experienced by patients receiving Jakafi were anemia and thrombocytopenia (low blood platelet count), while the most common nonhematologic side effects were infections (pathogen not specified) and viral infection.

“In the U.S., there are over 14,000 people living with chronic GVHD, many of whom face significant complications that may impair daily activities and linger for years,” said Susan Stewart, Executive Director of BMT InfoNet in a release. “The approval of Jakafi is an exciting development for the GVHD community and an important step forward in the treatment of a disease with few options.”

Prior to Jakafi’s approval, the FDA had extended the review period by three months to allow the drug developer, Incyte, to submit more data for review.

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Check back later to learn more about how this approval will affect patients with chronic GVHD.