Jakafi（每次1片，每日2次）抑制JAK 1和JAK2酶（Janus相关激酶），该酶涉及调节血液和免疫功能。骨髓纤维化与JAK 1和JAK2失调相关。
Ruxolitinib，一种激酶抑制剂, 抑制Janus相关激酶(JAKs)JAK1和JAK2，介导对造血和免疫功能重要的若干细胞因子和生长因子信号。JAK信号涉及细胞因子受体对 STATs(信号转导物和转炉激活的补充，激活和随后STATs定位至细胞核导致基因表达的调控。
片：5 mg，10 mg，15 mg，20 mg和25 mg。
1 mg片是红色膜衣，椭圆片一侧凹有“Pfizer”和另一侧“1 XNB”；得到80片瓶: NDC 0069-0145-01。
FDA Approves Incyte’s Jakafi™ (ruxolitinib) for Patients with Myelofibrosis
Signs and symptoms of the disease, in which the bone marrow is replaced by scar tissue resulting in blood cells being made in organs such as the liver and the spleen, are an enlarged spleen, anemia, decreased white blood cells and platelets, fatigue, abdominal discomfort, pain under the ribs, feelings of satiety, muscle and bone pain, itching, and night sweats.
Jakafi, made by Incyte Corp. is a pill taken two times a day, inhibits JAK 1 and 2 (Janus Associated Kinase), which are involved in regulating blood and immunological functioning. Myelofibrosis is associated with the deregulation of JAK 1 and 2.
“Jakafi represents another example of an increasing trend in oncology where a detailed scientific understanding of the mechanisms of a disease allows a drug to be directed toward specific molecular pathways,” Richard Pazdur, MD, Director of the FDA’s Office of Hematology and Oncology Products in the Center for Drug Evaluation and Research, said in a statement.
“The clinical trials leading to this approval focused on problems that patients with myelofibrosis commonly encounter, including enlarged spleens and pain.”
“Today’s FDA approval of Jakafi has the potential to transform the way we treat myelofibrosis,” said Srdan Verstovsek, MD, PhD, Associate Professor in the Department of Leukemia at the University of Texas MD Anderson Cancer Center, the principal investigator of the COMFORT-I pivotal trial.
“In this Phase III clinical trial, we observed significant reductions in spleen size and significant improvements in symptoms. Importantly, these benefits were achieved early on, most within a month, and tended to be durable during treatment. In contrast, most of the patients who received placebo saw their spleens increase and their symptoms worsen.”
The safety and effectiveness of Jakafi was evaluated in two clinical trials with 528 patients. Patients in both trials were resistant or refractory to available myelofibrosis therapy or ineligible for allogeneic bone marrow transplantation. All patients had splenomegaly and were in need of treatment as a result of disease-related symptoms.
Patients in the studies were selected to receive treatment with either Jakafi, placebo, or the best available therapy (hydroxyurea or glucocorticoids).
A greater percentage of patients receiving Jakafi experienced more than a 35% reduction in spleen size when compared with patients receiving placebo or best available therapy. Similarly, more patients receiving Jakafi had more than a 50% reduction in their myelofibrosis-related symptoms than was the case in patients receiving placebo.