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FDA Approves Marqibo (Vincristine Sulfate Liposome Injection) for Rare Leukemia

Orphan drug okayed under agency’s accelerated approval program

ALL is a rapidly progressing form of blood and bone-marrow cancer that is more commonly diagnosed in children than in adults. According to the National Cancer Institute, an estimated 6,050 men and women will be diagnosed with ALL and 1,440 will die from the disease in 2012.

Marqibo is approved for patients whose leukemia has relapsed two or more times, or whose leukemia has progressed following two or more regimens of antileukemia therapy. Marqibo contains vincristine, a commonly used anticancer drug, encased within a liposome (a drug-delivery vehicle composed of material similar to that of cell membranes). The drug is administered via intravenous injection once a week by a healthcare professional.

Marqibo was approved under the FDA’s accelerated approval program, which allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts additional clinical studies to confirm the drug’s clinical benefit and safe use. Marqibo also received orphan-product designation by the FDA because it is intended to treat a rare disease or condition.

The drug’s effectiveness was evaluated in a single clinical trial in adult patients whose leukemia had relapsed at least two times despite standard treatments, and who had at least one previous treatment response lasting at least 90 days. The study objective was to determine the rate of complete remission (CR) or a complete remission with incomplete blood count recovery (CRi) during treatment with Marqibo.

Of the 65 patients enrolled, 10 (15.4%) experienced either a CR or a CRi. In the 10 patients achieving a CR or a 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.

Prescribing information for Marqibo will carry a Boxed Warning alerting patients and healthcare professionals that the drug must be administered only intravenously because it is deadly if administered in other ways, such as into the spinal fluid. The Boxed Warning also states that Marqibo has different dosage recommendations than vincristine sulfate injection alone.

Marqibo is marketed by Talon Therapeutics Inc., based in South San Francisco, Calif.

For more information, visit the FDA Web site.

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