Efficacy

Inqovi (decitabine and cedazuridine) for Leukemia

Inqovi, a combination of decitabine and cedazuridine, is a medication approved for the treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British (FAB) subtypes: refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia (CMML). MDS are a group of disorders considered to be a form of blood cancer that can lead to leukemia. The efficacy of Inqovi was demonstrated in clinical trials where it was shown to be effective in achieving complete remission or partial remission in a significant number of patients. Additionally, Inqovi has been shown to improve blood cell counts in some patients, which is an important aspect of managing the disease.

The efficacy of Inqovi is particularly notable because it is an oral formulation that combines decitabine with cedazuridine, a cytidine deaminase inhibitor, which helps to increase the bioavailability of decitabine. This allows for a more convenient oral administration as opposed to the traditional intravenous administration of decitabine, potentially improving patient adherence to the treatment regimen and quality of life.

Scemblix (asciminib) for Leukemia

Scemblix, the brand name for asciminib, is a newer medication approved by the FDA for the treatment of chronic myeloid leukemia (CML). Specifically, it is indicated for adult patients with Philadelphia chromosome-positive CML in the chronic phase who have received two or more prior lines of tyrosine kinase inhibitor (TKI) therapy, or for those with the T315I mutation, which is resistant to most other TKIs. Asciminib works by a different mechanism than other TKIs, as it specifically targets the ABL myristoyl pocket of the BCR-ABL protein, which is the driver of CML.

The efficacy of Scemblix was established in clinical trials where it demonstrated a significant ability to achieve major molecular responses in patients who had resistance or intolerance to previous TKI therapies. For patients with the T315I mutation, Scemblix showed a particularly high efficacy rate, which is significant given the limited treatment options for this mutation. The approval of Scemblix provides an important new treatment option for patients with CML who have failed other therapies, offering hope for better disease management and outcomes.