European Commission Approves CPX-351 for Two Types of AML

The European Commission (EC) has approved CPX-351 for the treatment of adult patients with newly diagnosed therapy-related acute myeloid leukemia (t-AML) or acute myeloid leukemia with myelodysplasia-related changes (AML-MRC). The treatment had received approval for the same indications by the U.S. Food and Drug Administration in August 2017.

CPX-351 is a liposomal bound coformulation of cytarabine and daunorubicin that delivers the two medications in a 5:1 ratio. Studies have shown that this formulation results in less toxicity, higher response rates, and improved overall survival, compared with traditional cytarabine and daunorubicin (7+3) treatment.

EC approval is based primarily on data from a phase III clinical trial in which 309 patients (aged 60 to 75 years) received either CPX-351 or 7+3 treatment. The median overall survival was 9.56 months with CPX-351 versus 5.95 months with 7+3, representing a 31-percent reduction in the risk of death (p=0.005). The rate of complete response or complete response with incomplete platelet or neutrophil recovery rate was 47.7 percent for CPX-351, compared with 33.3 percent for 7+3 (p=0.016).

Common adverse events (AEs) occurring during the study include febrile neutropenia, pneumonia, hypoxia, sepsis, hypertension, respiratory failure, fatigue, bacteremia, and reduced ejection fraction. Rates of AEs were similar between the CPX-351 and 7+3 arms of the study.

Source: Jazz Pharmaceuticals press release, August 27, 2018.