FDA Grants CPX-351 Breakthrough Therapy Designation for AML

The FDA granted breakthrough therapy designation for CPX-351 for the treatment of adult patients with therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes.

The designation was based on the results from a phase III study of older patients with previously untreated high-risk AML or AML with myelodysplasia-related changes. The median overall survival (primary endpoint) was 9.6 months for those treated with CPX-351, compared with 6 months for those treated with the “7+3” regimen, representing a 31 percent reduction in the risk of death with CPX-351 (hazard ratio = 0.69; p=0.005).

At 12 and 24 months, 41.5 percent and 31.1 percent, respectively, of patients treated with CPX-351 were alive, compared with 27.6 percent and 12.3 percent, respectively, of those treated with 7+3. Sixty-day all-cause mortality was 13.7 percent with CPX-351 and 21.2 percent with 7+3.

AEs were similar among both treatment groups; grade 3-5 hematologic AEs included infections, febrile neutropenia, and bleeding events.

Source: Celator Pharmaceuticals press release, May 19, 2016.