Venetoclax is an investigational small molecule in advanced stages of approval for the treatment of people with chronic lymphocytic leukemia (CLL), including those with 17p deletion, who have received at least one prior therapy.
In some cases of CLL, especially in relapsed or refractory cases, a part of chromosome 17 is lost along with a gene called p53. The “17p” deletion makes the treatment of CLL especially difficult, resulting in poor prognosis in these cases.
Orally administered, venetoclax targets Bcl-2, a protein central to the survival of CLL cells.
Clinical trials are ongoing in several other blood cancers as well, including indolent NHL, diffuse large B-cell lymphoma (DLBCL), acute myeloid leukemia (AML) and multiple myeloma (MM).
Venetoclax, a drug developed by Genentech and AbbVie, was granted priority review by the FDA in 2016. An application for its approval has also been submitted to the EU. Venetoclax is synthesized through a multi-step chemical process.