Talon Gets Approval of FDA to Treat Rare Leukemia
The Food and Drug Administration has approved Talon Therapeutics Marqibo treatment to treat adults with a rare form of blood cancer and bone marrow cancer, Philadelphia chromosome negative acute lymphoblastic leukemia.
FDA prescribes this drug only for people whose disease has progressed despite the use of at least two anti-leukemia regimens.
According to the National Cancer Institute, an estimated 6,050 men and women will be diagnosed with rare type of leukemia called Philadelphia chromosome negative (Ph-) acute lymphoblastic leukemia (ALL) and 1,440 will die from the disease this year. ALL is most often diagnosed in children.
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The drug was evaluated in a clinical trial of adults whose disease had relapsed at least twice, despite standard anti-leukemia treatments.
The study objective was to determine the response rate to Marqibo, as either a complete remission (CR) or a complete remission with incomplete blood count recovery (CRi). Of 65 patients enrolled, 10 patients, or 15.4 percent, responded with either a CR or CRi. In the 10 patients achieving CR or CRi, the median duration of documented remission was 28 days. The median time to the first event of relapse, death, or next therapy was 56 days.
The safety of Marqibo was evaluated in two single-arm trials of 83 patients who received the clinical treatment regimen. Serious adverse events such as low white blood cell counts with fever, low blood pressure, respiratory distress and cardiac arrest occurred in 76 percent of the patients studied.
The most common side effects reported were constipation, nausea, low blood cell count, fever, nerve damage, fatigue, diarrhea, loss of appetite, and insomnia.
The drug's labeling will include a boxed warning that the drug must only been administered intravenously, and that it could be lethal if administered in another way.
"We are delighted that Marqibo will be available to a patient population with an underserved hematologic malignancy," stated Steven R. Deitcher MD, President, Chief Executive Officer and Board Member of Talon Therapeutics. "This represents a transformational event for Talon and fulfillment of our most important corporate goal, to date."