The AP (11/17) reports, "The first drug to treat a rare disorder that causes red blood cells to build up inside bone marrow was cleared Wednesday by the Food and Drug Administration." The medication, ruxolitinib (Jakafi), is taken "twice-a-day... to treat myelofibrosis."
The New York Times (11/17, Pollack) reports in its "prescriptions" blog that two clinical trials showed ruxolitinib "to shrink spleens and reduce symptoms, according to the data on the label." The first trial showed 41.9% of patients receiving ruxolitinib "compared to 0.7 percent getting a placebo - experienced a reduction of at least 35 percent in the size of their spleen after 24 weeks." The second trial showed 28.5% "of patients getting the drug had a 35 percent or greater shrinkage in spleen size, compared to zero patients getting the best available therapy," which was "usually a chemotherapy drug or a steroid."
MedPage Today (11/17, Gever) reports ruxolitinib works by inhibiting two kinases, JAK-1 and JAK-2. The FDA said "serious adverse effects seen in the ruxolitinib groups included thrombocytopenia, anemia, fatigue, shortness of breath, headache, dizziness, nausea, and confusion." A recent study showed "that serious adverse events may occur when ruxolitinib is stopped. Withdrawal symptoms included acute relapse of myelofibrosis symptoms, rapid and painful enlargement of the spleen, and acute hemodynamic decompensation, which in some patients led to a septic shock-like syndrome."
Medscape (11/17, Chustecka) reports ruxolitinib "was approved ahead of schedule under the FDA's priority review program for products that offer a significant advance over available therapies or provide a treatment where no adequate therapy exists." Also covering the story are the Wall Street Journal (11/17, Warner, Subscription Publication) and Reuters (11/17).
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