FDA Approves Jakafi® (ruxolitinib) for the Treatment of Patients with Acute Graft-Versus-Host Disease
Jakafi is the first and only
“For the first time, patients with steroid-refractory acute GVHD, and
the physicians that treat them, have an
The approval was based on data from REACH1, an open-label, single-arm,
multicenter study of Jakafi in combination with corticosteroids in
patients with steroid-refractory grade II-IV acute GVHD. Of the 71
patients recruited into REACH1, 49 patients were refractory to steroids
alone, 12 patients had received two or more prior anti-GVHD therapies
and 10 patients did not otherwise meet the
The efficacy of Jakafi was evaluated based upon Day 28 overall response
rate (ORR), defined as a complete response (CR), very good partial
response or partial response based on the
GVHD is a condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor) where the donated cells initiate an immune response and attack the transplant recipient’s organs, leading to significant morbidity and mortality. There are two major forms of GVHD, acute and chronic, that can affect multiple organ systems including the skin, gastrointestinal (digestive) tract and liver. Patients who develop steroid-refractory acute GVHD can progress to severe disease, with one-year mortality rates of approximately 70 percent.1
“Every year in
Jakafi will be made available to appropriate patients with
steroid-refractory acute GVHD immediately.
The REACH clinical trial program is evaluating Jakafi in patients with
steroid-refractory GVHD. The REACH program includes the
The REACH clinical program also includes the collaborative
About Jakafi® (ruxolitinib)
Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the U.S.
Jakafi is also indicated for treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea as well as intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF in adults.
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: for certain types of MF and PV - low platelet count, low red blood cell count, bruising, dizziness, and headache; and for acute GVHD – low red blood cell counts, low platelet counts, low white blood cell counts, infections and fluid retention.
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, have a high level of fat in your blood (high blood cholesterol or triglycerides), 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.
Women should not take Jakafi while pregnant or planning to become pregnant. Do not breast-feed during treatment with Jakafi and for 2 weeks after the final dose.
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 efforts to
advance the Company’s ongoing research in JAK inhibition and to help
more GVHD patients in the future, the potential efficacy, safety and
therapeutic value of Jakafi® (ruxolitinib) in patients with
steroid-refractory GVHD and expected timing of initial results of the
REACH2 and REACH3 trials. 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 those
risks detailed from time to time in Incyte’s reports filed with the
1 Shapira MY, Klimov A, Vipul S, et al. Regional intra-arterial steroid treatment in 120 patients with steroid-resistant or -dependent GvHD. Bone Marrow Transplant. 2017;52(10):1416-1422.
Michael Booth, DPhil