Venclexta

Venclexta (venetoclax) received FDA approval on October 16, 2020, as a Priority review sNDA (Application Number 208573, S-020 and S-021). It is indicated in combination with azacitidine, or decitabine, or low-dose cytarabine for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy. The drug was granted Breakthrough Therapy Designation on January 25, 2016.

Efficacy findings include a PFS p-value of <0.0001. In CR patients, the MRD negativity rate in peripheral blood was 87% for Venclexta + Obinutuzumab, compared to 62% for Obinutuzumab + Chlorambucil. The MRD negativity rate in bone marrow for CR patients (Obinutuzumab + Chlorambucil) was 45%, with a p-value of 0.0048. The CR+CRh response rate was 47%, with a median duration of CR+CRh of 11.1 months. The Transfusion Independence (TI) rate for RBCs and platelets was 33%.

Safety data indicates common adverse reactions in CLL/SLL patients including neutropenia, thrombocytopenia, anemia, diarrhea, nausea, upper respiratory tract infection, cough, musculoskeletal pain, fatigue, and edema. Any Serious Adverse Event (SAE) occurred in 73% of patients. Specific SAEs included febrile neutropenia (10%), pneumonia (26%), sepsis (8%), and hemorrhage (5%).