Efficacy

Efficacy of Rydapt (Midostaurin) for Leukemia

Rydapt, known generically as midostaurin, is a kinase inhibitor approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive, as detected by an FDA-approved test. This medication is used in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation. The efficacy of Rydapt in this setting was demonstrated in a pivotal phase III clinical trial (RATIFY), which showed a significant improvement in overall survival for patients receiving Rydapt in combination with chemotherapy compared to chemotherapy alone. Patients treated with Rydapt had a median overall survival of 74.7 months compared to 25.6 months for those who received placebo.

Additionally, Rydapt is also indicated for the treatment of adults with certain types of mastocytosis. However, its efficacy in these conditions is beyond the scope of this summary, which focuses on leukemia. It is important to note that the benefit of Rydapt for AML patients is specifically associated with the presence of the FLT3 mutation, and its use is not indicated for all cases of AML.

Efficacy of Inqovi (Decitabine and Cedazuridine) for Leukemia

Inqovi, which combines decitabine and cedazuridine, is an oral hypomethylating agent approved by the FDA 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 subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups. Inqovi is also indicated for the treatment of adult patients with chronic myelomonocytic leukemia (CMML).

The efficacy of Inqovi was established in two open-label, randomized, crossover trials in which the combination of decitabine and cedazuridine demonstrated similar drug exposure over a 5-day cycle compared to intravenous decitabine alone. Clinical trials have shown that Inqovi is effective in achieving complete remission or complete remission with partial hematologic recovery in a proportion of patients, with some patients also achieving hematologic improvement. The convenience of an oral formulation allows for easier administration and potentially improved adherence compared to intravenous treatments.