Jakafi Approved for Rare Bone-Marrow Disease
First drug in U.S. sanctioned for myelofibrosis
WEDNESDAY, Nov. 16 (HealthDay News) -- Jakafi (ruxolitinib) has been approved by the U.S. Food and Drug Administration as the first drug to treat myelofibrosis, a rare disease of the bone marrow, the agency said Wednesday.
In cases of myelofibrosis, healthy bone marrow is replaced by scar tissue, causing blood cells to be made in the liver, spleen and other organs. Symptoms often include an enlarged spleen, anemia, a decrease in white blood cells and platelets, fatigue, abdominal discomfort, pain under the ribs, muscle and bone pain, itching and night sweats, the FDA said in a news release.
Jakafi inhibits the actions of two enzymes that are involved in regulating blood, the agency said. The drug was evaluated in clinical studies involving 528 people, all of whom had an enlarged spleen.
Observed side effects included low blood platelet levels, anemia, fatigue, diarrhea, shortness of breath, headache, dizziness, nausea and confusion.
Jakafi was approved as an orphan drug, since myelofibrosis affects fewer than 200,000 people in the United States, the FDA said.
The drug is produced by Incyte Corp., based in Wilmington, Del.
The FDA has more about this approval.Scott Roberts Related Articles
- New System Targets Germs in Donated Blood Plasma
December 17, 2014
- First Newborn Screening Test Approved for Rare Immune Disorder
December 16, 2014
Learn More About Sharp
Sharp HealthCare is San Diego's health care leader with seven hospitals, two medical groups and a health plan. Learn more about our San Diego hospitals, choose a Sharp-affiliated San Diego doctor or browse our comprehensive medical services.
Copyright © 2011 HealthDay. All rights reserved.