EMA/99179/2015
EMEA/H/C/002464
EPAR summary of the public
Jakavi
This is a summary of Jakavi's European Public Assessment Report (EPAR). It explains how the Human Drugs Committee (CHMP) evaluates drugs to reach their opinion, and approves the grant of marketing approval and its recommendations for Jakavi's conditions of use.
What is Jakavi?
Jakavi is a drug containing the active substance ruxolitinib. It can be tablets (5, 10, 15 and 20 mg).
What is Jakavi used for?
Jakavi is used to treat the following conditions:
Adult bone marrow fibrosis, such as fever, night sweats, bone pain and weight loss, with splenomegaly (splenomegaly) or disease-related symptoms.MyelofibrosisIt is a disease in which the bone marrow becomes very dense and stiff and produces abnormal, immature blood cells. Jakavi can be used in three types of diseases: primary myelofibrosis (also known as chronic idiopathic myelofibrosis, the cause is unknown), which occurs later inPolycythemia(where the disease is associated with excessive red blood cell production) and late primaryThrombocytopeniaMyelofibrosis (where the disease is associated with overproduction of platelets, which are components that help blood clot).
Polycythemia in adults who are resistant or intolerant to hydroxyurea drugs. Hyperglycemia is a disease that primarily causes excessive red blood cells, which "blood thickens" and occasionally form blood clots, which can cause blood to flow to the organs.
This medicine is only available by prescription.
How is Jakavi used?
Only experienced doctors in the treatment of cancer patients can start using Jakavi treatment. The patient's complete blood count must be collected prior to initiation of treatment and monitored during treatment.
In myelofibrosis, the recommended starting dose is twice daily, up to 20 mg, based on platelet count. In erythrocytosis, the recommended starting dose is 10 mg twice daily.
If the treatment is not effective, the dose can be increased by 5 mg to 25 mg twice a day.
In some cases, lower doses should be used, including patients with reduced liver function or severe renal function, as well as patients taking certain other medications. Should stop treatment
If the blood level of the patient's platelets or neutrophils (a white blood cell) is below a certain threshold, or the spleen size or symptoms do not improve after six months. When the level of hemoglobin is very low, treatment of polycythemia should also be stopped.
How does Jakavi work?
The active substance ruxolitinib in Jakavi acts by blocking a group of enzymes called Janus kinase (JAK), which are involved in the production and growth of blood cells. In myelofibrosis and polycythemia, JAK activity is too high, leading to abnormal blood cell production. These blood cells migrate to organs including the spleen, causing them to become larger. By blocking JAK, Jakavi reduces the abnormal production of blood cells, thereby reducing the symptoms of the disease.
How did Jakavi learn?
In myelofibrosis, Jakavi conducted a survey of two major studies involving 528 patients. The first study compared Jakavi with placebo (virtual therapy). The second study compared Jakavi with the best treatments, including different types of drugs such as anticancer drugs, hormones and immunosuppressive agents. The primary measure of effectiveness was the proportion of patients with a spleen size reduction of at least 35% six months after the first study and one year after the second study.
In erythrocytosis, Jakavi conducted a study in a study involving 222 patients who were resistant or intolerant to hydroxyurea. This study compared Jakavi with the best treatment plan and observed the percentage improvement in the patient's condition, measured as no or no more than one venous incision (a method of removing excess blood from the body), and the spleen reduction treatment was 8 After the month, the body weight is at least 35%.
What benefits did Jakavi show during the study?
In bone marrow fibrosis, Jakavi is more effective than placebo and is the best treatment for reducing the size of the spleen. In the first study, 42% of patients treated with Jakavi (65 out of 155 patients) achieved a reduction in spleen size, compared with less than 1% of patients in the placebo group (of 153 patients) 1)). In the second study, 29% of patients treated with Jakavi (41 out of 144 patients) achieved a reduction in spleen size, compared with 0% of patients receiving optimal treatment (0 out of 72 patients) name). In erythrocytosis, 21% of patients treated with Jakavi (23 of 110) showed improvement after 8 months of treatment, and 1 of the patients who received the best treatment (1 of 112) example).
What are the risks associated with Jakavi?
In bone marrow fibrosis, Jakavi's most common side effects (more than 1 in 10 patients) are thrombocytopenia (low platelet count), anemia (low red blood cell count), and neutropenia (low neutrophil levels) Urinary tract infection (infection with urine-bearing structure, bleeding, bruising, weight gain, hypercholesterolemia (high blood cholesterol levels), dizziness, headache and elevated liver enzyme levels.
In erythrocytosis, the most common side effects of Jakavi (more than 1 in 10 patients) are thrombocytopenia (low platelet count), anemia (low red blood cell count), hemorrhage, bruises, hypercholesterolemia (high blood cholesterol) Level), hypertriglyceridemia (high blood lipid levels), dizziness, elevated liver enzymes, and high blood pressure.
Jakavi must not be used by women who are pregnant or breastfeeding. For a complete list of all the side effects and limitations of Jakavi, see the package flyer.
Why is Jakavi approved?
CHMP decided that Jakavi's interests outweighed its risks and suggested obtaining a marketing license. In myelofibrosis, CHMP believes that the reduction in spleen size and symptoms in patients taking Jakavi is of clinical importance. The Committee noted that the quality of life of patients treated with Jakavi has improved, but the effects of the drug have yet to prolong the life of the patient or delay the progression of the disease or the onset of leukemia. With regard to safety, the Committee believes that the risk of infection is acceptable, but should be monitored further and that other known risks, such as bleeding or a reduction in blood counts, can be properly managed.
In erythrocytosis, CHMP believes that Jakavi is beneficial for patients who are not responding or intolerant to hydroxyurea treatment, and that safety is acceptable. However, the long-term effects of the drug require further research.
What measures are being taken to ensure the safe and effective use of Yakuwi?
A risk management plan has been developed to ensure that Jakavi is used as safely as possible. According to the program, safety information is included in the product feature summary and Jakavi package inserts, including appropriate precautions for health care professionals and patients.
In addition, Jakavi's company is expanding its main research on myelofibrosis and will provide annual data on the impact of Jakavi on patient survival and survival without deteriorating or leukemia. In erythrocytosis, the company will expand its main research to provide long-term data on the safety and effectiveness of Jakavi.
Additional information about Jakavi
On August 23, 2012, the European Commission approved the entire EU listing permit for Jakavi.
The complete EPAR for Jakavi can be found on the Agency's website: ema.europa.eu/Find medicine / Human medicine / European public assessment reports. For more information on using Jakavi treatment, please read the package insert (also part of EPAR) or contact your doctor or pharmacist.
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