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  • Writer's pictureDr Edin Hamzić

💊 Shortly About Venetoclax

Updated: Jan 14

💊 What is Venetoclax?

  • ⚠️Venetoclax is a Bcl-2 inhibitor for treating chronic lymphocytic leukemia (CLL) and acute myeloid leukemia (AML).

  • ⚠️Venetoclax was approved by the US FDA in 2018 and 2021 by the European Medicine Agency (EMA) for the treatment of acute myeloid leukemia (AML) [citation].

  • A UK-led, international clinical trial VICTOR (Venetoclax or intensive chemotherapy for treatment of favorable risk AML), is investigating the efficacy of Venetoclax versus standard intense chemotherapy in over 60-year-olds with NPM1 mutated AML.

✍️ What are other names for Venetoclax?

  • Commercial names under which venetoclax is distributed are Venclexta and Venclyxto.

🧬 When is Venetoclax used?

  • ⚠️ As already mentioned, Venetoclax is used for the treatment of chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), or acute myeloid leukemia (AML).

  • Venetoclax is indicated for newly diagnosed patients, especially for older (>75) or ineligible for intensive CTX in combination with hypomethylating agents (HMA) or low-dose cytarabine (LDAC) [citation].

  • ⚠️ Venetoclax is used in AML patients with mutated NPM1.

  • Further research should be directed toward patients who do not fit this type of therapy.

  • Furthermore, Venetoclax, combined with FLT3 inhibitors, in patients with mutations on NPM1 mutated AML that are co-mutated with FLT3 mutations [citation].

🏭 Who produces Venetoclax?

  • Venclexta is manufactured by AbbVie Inc., and it is marketed by both Abbvie and Genentech USA, which is a member of the Roche Group [citation].

💊 How does Venetoclax work? What is a mode of action for Venetoclax?

  • ⚠️ As mentioned above, venetoclax is a Bcl-2 inhibitor that works by blocking the action of Bcl-2 that helps cancer cells survive [citation].

  • Venetoclax (VEN) plus azacitidine has become the first-line therapy for elderly patients with acute myeloid leukemia [citation].

  • ⚠️ The main side effects of venetoclax plus hypomethylating agents (HMAs) are hematological, with about 40% of cases showing:

    • Cytopenias (cytopenia is a condition characterized by a lower than normal number of blood cells) and

    • about 30% grade 3 or higher infectious complications [citation].

  • Proteins in the B cell of chronic lymphocytic leukemia (CLL)/lymphoma 2 (Bcl-2) family are important regulators of the apoptotic (programmed cell death) process.

  • The selective inhibition of Bcl-2 by venetoclax enables therapeutic induction of apoptosis without the negative effect of thrombocytopenia. Venetoclax helps restore the process of apoptosis by binding directly to the Bcl-2 protein, displacing pro-apoptotic proteins, leading to mitochondrial outer membrane permeabilization and the activation of caspase enzymes [citation].

ℹ️ How is Venetoclax applied?

  • ℹ️ The FDA label indicates that venetoclax should be taken with food, 400 mg daily [citation]. Venetoclax is a cytochrome P450 3A4 (CYP3A4) substrate. Thus, its dose should be reduced by at least 75% when combined with posaconazole, a strong CYP3A4 inhibitor commonly used for antifungal prophylaxis in AML [citation].

  • ℹ️ In combination with Venetoclax with “5+2” induction chemotherapy (CAVEAT trial) followed by four consolidation cycles was found to be safe in fit older patients with AML [citation].


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