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FDA Approves New Treatment Option for Common and Serious Complication of Life-saving Stem Cell Transplants

Rye Brook, N.Y., September 24, 2021 — The U.S. Food and Drug Administration (FDA) this week approved ruxolitinib (Jakafi) in patients 12 years or older for the treatment chronic graft-versus-host-disease (GVHD) that has not responded to previous treatment. GVHD is a common and serious complication of donor-derived stem cell transplants, which are used to treat multiple myeloma and forms of leukemia and lymphoma.

GVHD happens when the donor’s T cells (the graft) see the patient’s healthy cells (the host) as foreign and attack them. The chronic form of GVHD typically occurs 100 days after transplant and can cause serious damage, including inflammation and scarring of the skin, mouth, eyes, joints, liver, lung, esophagus and the GI tract.

Jakafi works by targeting the JAK protein, which is a key player in the development of GVHD. LLS provided vital early funding that helped scientists understand the role of the JAK in blood cancer. This research laid the foundation for ruxolitinib treatment not only of GVHD, but also for the treatment of the blood cancers myelofibrosis and polycythemia vera.

The FDA approval of ruxolitinib for chronic GVHD was based on findings from the REACH3 study, which compared ruxolitinib to the best available therapy. The overall response rate after 24 weeks of treatment, including partial and complete response, was 70% in patients treated with ruxolitinib compared with 57% in patients treated with other therapies. The most common side effects of ruxolitinib for chronic GVHD include low red blood cell or platelet counts and infections including viral infections.

Almost half of the patients who develop chronic GVHD do not respond adequately to the current standard of care (steroids). Ruxolitinib gives patients an important new option to help with this frequent and sometimes fatal complication of stem cell transplants.