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Orphan Drug Designation has been granted by the FDA for AMV564, a novel CD33/CD3 T-cell engagement therapy, for the treatment of acute myeloid leukemia (AML).

FDA; acute myeloid l... - Click to enlarge in new windowFDA; acute myeloid leukemia. FDA; acute myeloid leukemia

A phase I clinical study of AMV564 in relapsed or refractory AML is being conducted. A phase I clinical study in patients with MDS is being planned for early 2018. The utility of AMV564 in solid tumors is also being explored.


In preclinical studies, this novel CD33/CD3 bispecific antibody demonstrated potent activity against AML patient samples that was independent of CD33 expression level, disease stage, and cytogenetic risk. The antibody eliminated nearly all blasts from bone marrow and spleen in a stringent AML patient-derived xenograft murine model. In addition, a therapeutic window for AMV564 in cynomolgus monkeys has been established, with rapid and sustained elimination of CD33-expressing cells during AMV564 dosing and rapid hematopoietic recovery following dosing.


Orphan Drug Designation is granted to products that treat rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits and incentives.