Efficacy

Venclyxto/Venclexta (Venetoclax) Efficacy in Leukemia

Venclyxto, known as Venclexta in the United States, contains the active substance venetoclax and is primarily used in the treatment of chronic lymphocytic leukemia (CLL) and some forms of acute myeloid leukemia (AML). Venetoclax is a BCL-2 inhibitor that induces apoptosis in cancer cells by blocking the action of the BCL-2 protein, which is overexpressed in CLL cells and contributes to their survival and resistance to chemotherapy. In clinical trials, venetoclax, often in combination with other agents such as rituximab or obinutuzumab, has demonstrated significant efficacy in reducing tumor burden and achieving remission in patients with CLL, including those with difficult-to-treat genetic mutations like 17p deletion.

For patients with AML, venetoclax is approved for use in combination with azacitidine, decitabine, or low-dose cytarabine for newly-diagnosed patients who are aged 75 years or older, or who have comorbidities that preclude the use of intensive induction chemotherapy. The combination of venetoclax with these hypomethylating agents or low-dose cytarabine has shown to improve overall survival and increase rates of complete remission compared to historical treatment options.

Xospata (Gilteritinib) Efficacy in Leukemia

Xospata, which contains the active ingredient gilteritinib, is a targeted therapy approved for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test. FLT3 mutations are one of the most common genetic abnormalities in AML and are associated with a poor prognosis. Gilteritinib is a FLT3/AXL inhibitor that works by inhibiting the signaling pathways that promote the survival and proliferation of leukemia cells.

In clinical trials, gilteritinib has demonstrated efficacy in inducing remissions in patients with FLT3-mutated AML who have relapsed after or are refractory to prior therapy. The drug has shown a significant improvement in overall survival compared to chemotherapy, and a higher percentage of patients treated with gilteritinib achieved complete remission with full or partial hematologic recovery. These results support the use of gilteritinib as an important treatment option for this specific group of AML patients.