FDA Grants Priority Review to Jakafi for Acute Graft-Versus-Host-Disease

The Food and Drug Administration (FDA) has granted a priority review to the supplemental new drug application for Jakafi (ruxolitinib) to treat patients with acute graft-versus-host-disease (GVHD) who have had an inadequate response to corticosteroids.

GVHD is a potentially serious complication of allogeneic stem cell transplantation — when a patient receives stem cells from a donor or donated umbilical cord blood. When a patient receives donated stem cells containing T-cells (a type of white blood cell that help protect the body by recognizing foreign invaders), the donor cells may also attack the patient’s healthy tissues and organs. This in turn, can impair the tissue or organ’s function or may cause it to fail altogether, known as GVHD.

“Patients with acute GVHD face significant morbidity and mortality risk, underscoring the urgent need for new treatment options,” Steven Stein, M.D., chief medical officer of Incyte, the drug’s manufacturer, said in a press release.

In the supplemental new drug application, Incyte included data from the REACH1 study — designed to evaluate Jakafi in combination with corticosteroids in 71 patients with acute GVHD who have had an inadequate response to corticosteroids.

Topline results from the ongoing trial demonstrated an overall response rate of 55 percent (39 patients experienced a response) at day 28, the primary endpoint. In addition, the best overall response rate was 73 percent (52 patients).

“If approved, ruxolitinib will be the first and only treatment available in the U.S. for patients with acute GVHD who have not responded adequately to corticosteroid therapy,” Stein said. “The acceptance of our (supplemental new drug application) is a critical milestone for Incyte and the GVHD community, and we look forward to working with the FDA to bring this innovative therapy to patients who currently have limited treatment options as soon as we are able.”