The FDA just approved a brand new kind of cancer drug.
The drug, called venetoclax, was approved to treat a subset of people with one of the most common forms of adult leukemia, a type of cancer that affects the blood and bone marrow. It’s made by drugmaker AbbVie.
Specifically, the drug targets those with chronic lymphocytic leukemia (CLL) who also have a precise genetic tweak (called a 17p deletion) and have already been through at least one other course of treatment.
Here’s what you need to know about the drug’s approval:
- Venetoclax, which will also go by the name Venclexta, is taken orally.
- In its phase 3 study, which looked at 106 people with CLL and the specific genetic tweak, about 80% of patients experienced partial or complete remission by the end of the five-week period. Doses were increased over the five weeks from 20 mg to 400 mg.
- Venetoclax is the first FDA-approved treatment to target something called “BCL2,” a protein that helps cancer cells grow. Especially in patients with CLL, the BCL2 gene is overexpressed, meaning it makes too many copies of the protein. Venetoclax goes in and inhibits that protein, which gives healthy cells room to grow and keeps cancer cells from growing out of control.
- That’s especially helpful for those with the 17p deletion genetic tweak, because the mutation makes it harder for people with CLL to suppress cancer growth.
- Michael Severino, AbbVie’s Chief Scientific Officer told Business Insider that by inhibiting the BCL2 protein, they’re able to avoid killing other cells (think: chemotherapy, which tends to target a wider range of cells).
- Severino said the goal is to get venetoclax approved beyond ust people who have CLL and the specific genetic tweak. Within CLL, there are some forms of the cancer that progress quicker than others. The genetic mutation, in particular, is associated with the more aggressive versions.