FDA Approves Venclexta (Venetoclax) for a Type of Chronic Lymphocytic Leukemia

The US Food and Drug Administration (FDA) has approved a new targeted therapy for a certain type of chronic lymphocytic leukemia (CLL). The drug Venclexta (venetoclax) treats people with CLL whose leukemia cells have a chromosome abnormality called 17p deletion and who have had at least one treatment already. It targets BCL-2, a protein in CLL cells that helps them live longer than they should.

The FDA based its approval on a study of 106 people with CLL who have a 17p deletion and who had received at least one previous therapy. All the participants were given Venclexta. Results showed that 80% of them either improved or saw their cancer disappear completely.

Venclexta was approved using several FDA approaches designed to speed up the availability of drugs to treat serious diseases – breakthrough therapy designation, priority review status, and accelerated approval. It was also granted orphan drug designation, which provides financial incentives to encourage the development of drugs for rare diseases.

Venclexta is a pill taken once a day. Side effects can include low levels of certain white blood cells (neutropenia), low red blood cell counts (anemia), diarrhea, nausea, respiratory infections (such as colds), low platelet counts (thrombocytopenia), and feeling tired.

Less common but more serious side effects can include pneumonia and other serious infections, and tumor lysis syndrome (in which many leukemia cells break open and spill their contents into the body).

Venclexta is manufactured by AbbVie Inc., and marketed by AbbVie and Genentech USA Inc.