FDA Grants Priority Review for Ruxolitinib (Jakafi®) as a Treatment for Patients with Acute Graft-Versus-Host Disease
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
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
Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the
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
Important Safety Information
Jakafi can cause serious side effects, including:
Low blood counts: Jakafi® (ruxolitinib) may cause your platelet, red blood cell, or white blood cell counts to be lowered. If you develop bleeding, stop taking Jakafi and call your healthcare provider. Your healthcare provider will perform blood tests to check your blood counts before you start Jakafi and regularly during your treatment. Your healthcare provider may change your dose of Jakafi or stop your treatment based on the results of your blood tests. Tell your healthcare provider right away if you develop or have worsening symptoms such as unusual bleeding, bruising, tiredness, shortness of breath, or a fever.
Infection: You may be at risk for developing a serious infection during treatment with Jakafi. Tell your healthcare provider if you develop any of the following symptoms of infection: chills, nausea, vomiting, aches, weakness, fever, painful skin rash or blisters.
Skin cancers: Some people who take Jakafi have developed certain types of non-melanoma skin cancers. Tell your healthcare provider if you develop any new or changing skin lesions.
Increases in Cholesterol: You may have changes in your blood cholesterol levels. Your healthcare provider will do blood tests to check your cholesterol levels during your treatment with Jakafi.
The most common side effects of Jakafi include: low platelet count, low red blood cell counts, bruising, dizziness, headache.
These are not all the possible side effects of Jakafi. Ask your pharmacist or healthcare provider for more information. Tell your healthcare provider about any side effect that bothers you or that does not go away.
Before taking Jakafi, tell your healthcare provider about: all the medications, vitamins, and herbal supplements you are taking and all your medical conditions, including if you have an infection, have or had tuberculosis (TB), or have been in close contact with someone who has TB, have or had hepatitis B, have or had liver or kidney problems, are on dialysis, had skin cancer or have any other medical condition. Take Jakafi exactly as your healthcare provider tells you. Do not change or stop taking Jakafi without first talking to your healthcare provider. Do not drink grapefruit juice while on Jakafi.
Women should not take Jakafi while pregnant or planning to become pregnant, or if breast-feeding.
Except for the historical information set forth herein, the matters set forth in this release contain predictions, estimates and other forward-looking statements, including statements regarding whether or when ruxolitinib might be approved in the U.S. for patients with acute GVHD who have had an inadequate response to corticosteroids. These forward-looking statements are based on Incyte’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to the efficacy or safety of ruxolitinib for the treatment of patients with acute GVHD who have had an inadequate response to corticosteroids, the results of additional data and additional analyses of data from the REACH1 study, actions taken by regulatory authorities, and other risks detailed from time to time in Incyte’s reports filed with the