FDA NEWS RELEASE. For Immediate Release:
Nov. 16, 2011. The U.S. Food and Drug Administration today approved Jakafi (ruxolitinib),
the first drug approved to specifically treat patients with the bone marrow
disease myelofibrosis. Myelofibrosis is a 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. This disease is marked by an enlarged spleen, anemia, decreased white
blood cells and platelets, and myelofibrosis-related symptoms.
Symptoms include fatigue, abdominal discomfort, pain under the ribs, feeling
full (satiety), muscle and bone pain, itching, and night sweats. Jakafi, a pill taken two times a day, inhibits enzymes called JAK 1 and 2
(JanuAssociated Kinase) that 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,” said Richard Pazdur, M.D.,
director of the Office of Hematology and Oncology Products in the FDA’s Center
for Drug Evaluation and Research. “The clinical trials leading to this approval
focused on problems that patients with myelofibrosis commonly encounter,
including enlarged spleens and pain.”
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 (procedure where a person receives blood-forming stem cells from
a genetically similar, but not necessarily identical, donor). All patients had
enlarged spleens (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 (sugar pill) or the best available therapy (hydroxyurea, a
chemotherapy agent, or glucocorticoids). A greater percentage of patients
receiving Jakafi experienced more than a 35 percent reduction in spleen size
when compared to patients receiving placebo or best available therapy.
Similarly, a greater proportion of patients receiving Jakafi saw more than a 50
percent reduction in their myelofibrosis-related symptoms, including abdominal
discomfort, night sweats, itching and bone or muscle pain, than was the case in
patients receiving placebo.
The most serious side effects seen in patients treated with Jakafi include
low blood platelet levels (thrombocytopenia), anemia, fatigue, diarrhea,
shortness of breath (dyspnea), headache, dizziness, and nausea.
Jakafi was reviewed under the FDA’s priority review program, an expedited
six-month review of drugs that may offer significant advances in treatment over
available therapy or that provide a treatment when no adequate therapy
exists.
The treatment is being approved ahead of the drug’s Dec. 3, 2011 review goal
date under the Prescription Drug User Fee Act and has been designated as an
orphan drug, which identifies the disease as affecting fewer than 200,000 people
in the U.S.
Jakafi is manufactured by Incyte Corp. of Wilmington, Del.
For more information:
The FDA, an agency within the U.S. Department of Health and Human
Services, protects the public health by assuring the safety, effectiveness, and
security of human and veterinary drugs, vaccines and other biological products
for human use, and medical devices. The agency also is responsible for the
safety and security of our nation’s food supply, cosmetics, dietary supplements,
products that give off electronic radiation, and for regulating tobacco
products.
For Immediate Release: Nov. 16, 2011. Media Inquiries: Erica Jefferson, 301-796-4988, erica.jefferson@fda.hhs.gov Consumer Inquiries: 888-INFO-FDA
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