FDA Grants Priority Review for Ruxolitinib (Jakafi) for Acute Graft-Versus-Host Disease

• The FDA previously granted ruxolitinib Breakthrough Therapy Designation for the treatment of acute GVHD
• Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States
• Jakafi is also indicated for treatment of people with intermediate or high-risk myelofibrosis

Incyte Corporation (Nasdaq:INCY) announced on 10/25/18 that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review its supplemental New Drug Application (sNDA) for ruxolitinib (Jakafi) for the treatment of patients with acute graft-versus-host-disease (GVHD) who have had an inadequate response to corticosteroids. The FDA grants Priority Review to medicines that have the potential to provide significant improvements in the treatment of a serious disease. This designation shortens the FDA review period to six months compared to 10 months for Standard Review.

The sNDA submission is based on data from the REACH1 study evaluating ruxolitinib in combination with corticosteroids in patients with acute GVHD who have had an inadequate response to corticosteroids. Topline results from this ongoing trial demonstrated an overall response rate of 55 percent (n=39/71) at Day 28, the primary endpoint. In addition, the best overall response rate was 73 percent (n=52/71).

“Patients with acute GVHD face significant morbidity and mortality risk, underscoring the urgent need for new treatment options,” said Steven Stein, M.D., Chief Medical Officer, Incyte. “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. The acceptance of our sNDA 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.”

GVHD is a condition that can occur after an allogeneic transplant (the transfer of genetically dissimilar blood stem cells) and is a significant cause of morbidity and mortality in transplant recipients. In GVHD, the donated bone marrow or peripheral blood stem cells view the recipient’s body as foreign and attack the body. There are two forms of GVHD, acute and chronic, which can affect multiple organ systems including the skin, gastrointestinal (digestive) tract and liver.

The FDA previously granted ruxolitinib Breakthrough Therapy Designation for the treatment of acute GVHD, which is designed to expedite the development and review of drugs for serious conditions that have shown encouraging early clinical results and may demonstrate substantial improvements over available medicines. Additionally, the FDA has granted ruxolitinib Orphan Drug Designation for the treatment of GVHD, a designation granted to investigational compounds intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people.

Jakafi is also indicated for treatment of people with intermediate or high-risk myelofibrosis (MF), including primary MF, post–polycythemia vera MF, and post–essential thrombocythemia MF.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.