Posted on January 5th, 2012 by
The US Food and Drug Administration (FDA) has approved Jakafi™ (ruxolitinib) for the treatment of the bone marrow disease myelofibrosis.
Myelofibrosis is a chronic disease characterized by abnormal blood cell development and the formation of scar tissue in the bone marrow. Symptoms of myelofibrosis can include fatigue, shortness of breath, abdominal discomfort, pain under the ribs, feeling full, muscle and bone pain, itching, and night sweats. Most patients with myelofibrosis have an enlarged spleen, and many have an enlarged liver.
For people who are experiencing symptoms from myelofibrosis, previous approaches to treatment have included blood transfusions, drug therapy, radiation therapy, surgery to remove the spleen, or stem cell transplantation.
Jakafi is the first drug to be approved specifically for myelofibrosis. It is taken orally, and inhibits two enzymes—JAK1 and JAK2—that contribute to myelofibrosis.
Two randomized clinical trials that enrolled a total of 528 patients contributed to the approval of Jakafi. The studies enrolled myelofibrosis patients who were experiencing symptoms from their condition and had enlarged spleens. The patients were resistant to other approaches to myelofibrosis treatment or ineligible for stem cell transplantation.
One of the studies compared Jakafi with a placebo (a sugar pill), and the other compared Jakafi with best available therapy.
Compared with the other patients, patients who received Jakafi were more likely to experience a substantial reduction in the size of their spleen, and were also more likely to experience at least a 50 percent reduction in their myelofibrosis symptoms.
Side effects of Jakafi included low blood platelet levels, anemia, fatigue, diarrhea, shortness of breath, headache, dizziness, and nausea.
Jakafi was approved by the FDA for the treatment of intermediate- and high-risk myelofibrosis
Reference: FDA News Release. FDA approves first drug to treat a rare bone marrow disease. November 16, 2011.
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