The U. S. Food and Drug Administration (FDA) has granted Fast Track designation to the investigational drug CPX-351, a liposomal formulation of cytarabine-daunorubicin, for the treatment of elderly patients with secondary acute myeloid leukemia.

Acute myeloid leukemia (AML) is a cancer of the bone marrow (spongy portion found in the middle of bones) and blood characterized by the rapid, uncontrolled growth of immature white blood cells known as myelocytes. The disease is more common in adults than in children; the average age at diagnosis is over 65. Treatment of elderly patients with AML remains unsatisfactory, and most elderly patients die within a months of diagnosis.

CPX-351 is a liposomal formulation of cytarabine and daunorubicin in a 5:1 ratio designed to maximize anti-tumor synergy. CPX-351 accumulates within bone marrow with preferential uptake of liposomes by leukemia cells followed by intracellular release of encapsulated drug. CPX-351 has been granted orphan drug status by the U.S. FDA for the treatment of AML.

Fast Track designation from the FDA was designed to facilitate the development and expedite the review of drugs that demonstrate the potential to address an unmet medical need for a serious or life-threatening condition.

A phase III study comparing CPX-351 to the current standard of care has recently completed enrollment. Preliminary data from this clinical trial are expected to be available in the second quarter of this year, and overall survival data, which is the primary endpoint of the study, is expected early in 2016.

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