FDA Approves Jakafi® (ruxolitinib) for the Treatment of Patients with Uncontrolled Polycythemia Vera
The first and only
FDA-approved treatment for patients with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea
- PV is a rare blood cancer associated with overproduction of blood cells that can cause serious cardiovascular complications, such as stroke and heart attack1
- Jakafi is the only treatment for patients with PV shown to provide consistent hematocrit control, spleen volume reduction and complete hematological remission by targeting the overactive JAK pathway, a critical component of this disease
A bottle of Jakafi® (ruxolitinib) 10mg tablets (Photo: Business Wire)
“The approval of Jakafi represents an important advance for patients
with uncontrolled PV. For the first time we are able to provide these
patients a treatment that has been shown to provide effective and
consistent control of their blood counts and reduce spleen volume,” said
Srdan Verstovsek, M.D., Ph.D., Professor,
PV is a myeloproliferative neoplasm (MPN) and is typically characterized by elevated hematocrit, the volume percentage of red blood cells in whole blood, which can lead to a thickening of the blood and an increased risk of blood clots, as well as an elevated white blood cell and platelet count1. PV may occur at any age but often presents later in life, with a median age at diagnosis of 60 years1,2.
Approximately 100,000 patients in the U.S. are living with PV3. Current standard treatment for PV is phlebotomy (the removal of blood from the body) plus aspirin. When phlebotomy can no longer control PV, chemotherapy such as hydroxyurea, or interferon, is utilized4,5. Approximately one in four (~25,000) patients with PV are considered uncontrolled6,7 because they have an inadequate response to or are intolerant of hydroxyurea, the most commonly used chemotherapeutic agent for the treatment of PV.
Patients with PV who fail to consistently maintain appropriate blood count levels, including appropriate hematocrit levels, have an approximately four times higher risk of major thrombosis (blood clots) or cardiovascular death8.Patients with PV can also suffer from an enlarged spleen, and a significant symptom burden which may be attributed to thickening of the blood and a lack of oxygen to parts of the body9. These symptoms commonly include fatigue, itching, night sweats, bone pain, fever, and weight loss5.
“Being diagnosed with a serious disease affects a person in a way that
cannot be predicted,” said
Jakafi is also the first and only
“The team at
The approval of Jakafi for the treatment of patients with polycythemia
vera who have had an inadequate response to or are intolerant of
hydroxyurea was based on data from the pivotal Phase III RESPONSE trial,
which was conducted under a Special Protocol Assessment from the
About Jakafi® (ruxolitinib)
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 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 experience unusual bleeding, bruising, fatigue, 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.
The most common side effects of Jakafi include: anemia, low platelet count, 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 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.
Patient Assistant Program: IncyteCARES
Except for the historical information set forth herein, the matters set
forth in this press release, including without limitation statements
with respect to the potential efficacy, safety and therapeutic value of
Jakafi® (ruxolitinib) in uncontrolled polycythemia vera,
contain predictions and estimates and are forward-looking statements
within the meaning of the "safe harbor" provisions of the Private
Securities Litigation Reform Act of 1995. 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 Jakafi, the results of further research and
development, other market or economic factors, competitive and
technological advances, and other risks detailed from time to time in
Leukemia & Lymphoma Society. Polycythemia Vera Facts 2012. Available at:
|2||Tefferi A, Rumi E, Finazzi G, et al. Leukemia. 2013;27:1874-81.|
|3||Data on file. Incyte Corporation|
|4||Vannucchi AM. Blood 2014;124(22):3212-20.|
|5||Passamonti F. Blood 2012;120(2):275-84.|
|6||Barosi G, Birgegard G, Finazzi G, et al. Br J Haematol. 2010;148:961-3.|
|7||Alvarez-Larrán A, Pereira A, Cervantes F, et al. Blood. 2012;119:1363-9|
|8||Marchioli R, et al. N Engl J Med. 2013;368:22-33.|
National Institutes of Health http://www.nhlbi.nih.gov/health/health-topics/topics/poly/signs
|10||Vannucchi AM et al CA Cancer J Clin 2009;59:171-191|
Pamela M. Murphy
Vice President, Investor Relations/Corporate Communications
Sam Brown Inc.