FDA Approves First and Only Drug For Myelofibrosis

Incyte Corporation announced last month that the U.S. Food and Drug Administration has approved their drug, Jakafi, for the treatment of patients with intermediate or high-risk myelofibrosis (MF), a debilitating bone marrow disease.

MF is a progressive, potentially life-threatening blood cancer, and patients who suffer from MF live an impaired quality of life.  MF has a poor prognosis and limited treatment options.

More specifically, myelofibrosis is a disease where the bone marrow is replaced by scar tissue, resulting in blood cell production in organs like the liver and spleen. MF may be detected by enlarged spleens, anemia, decreased white blood cells, and platelets. Other symptoms include fatigue, abdominal discomfort, pain under the ribs, satiety, muscle or bone pain, itching, and night sweats.

Jakafi is the first (and only) product approved by the FDA for MF, and the first in a new class of drugs, known as JAK (Janus Associated Kinase). The safety and effectiveness of Jakafi was evaluated in two clinical trials with 528 patients. All the patients had enlarged spleens and were in need of treatment as a result of the disease-related symptoms.

The most serious side effects seen in patients treated with Jakafi include shortness of breath, anemia, fatigue, diarrhea, nausea, dizziness, headache, and low blood platelet levels.

“Today’s FDA approval of Jakafi has the potential to transform the way we treat myelofibrosis,” said Srdan Verstovsek, M.D., Ph.D., Associate Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center. He mentioned significant reductions in spleen size and improvements in symptoms early on in the Phase III clinical trial, especially compared to the placebo.

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