Efficacy

Efficacy of Tibsovo (Ivosidenib) in Treating Leukemia

Tibsovo (ivosidenib) is an oral medication specifically approved for the treatment of acute myeloid leukemia (AML) with a mutation in the isocitrate dehydrogenase-1 (IDH1) gene in adult patients. This targeted therapy has shown efficacy in patients with relapsed or refractory AML, as well as those who are newly diagnosed and are aged 75 years or older or who have comorbidities that preclude the use of intensive induction chemotherapy. Clinical trials have demonstrated that Tibsovo can induce complete remission (CR) or complete remission with partial hematologic recovery (CRh) in a significant proportion of patients, with some achieving a reduction in the risk of disease progression or death compared to conventional care.

The efficacy of Tibsovo was evaluated in a pivotal phase 1 study and a subsequent phase 3 trial. In the phase 1 study, the CR+CRh rate was observed to be around 30%, with a median duration of response lasting over 8 months. In the phase 3 trial, the median overall survival for patients treated with Tibsovo was notably greater than for those who received placebo. These outcomes suggest that Tibsovo can be an effective monotherapy for AML patients with an IDH1 mutation, especially for those who have limited treatment options due to age or comorbidities.

Efficacy of Xospata (Gilteritinib) in Treating Leukemia

Xospata (gilteritinib) is an oral medication approved for the treatment of adult patients who have relapsed or refractory AML with an FLT3 mutation as detected by an FDA-approved test. Gilteritinib is a type of drug known as a kinase inhibitor, which works by blocking several enzymes that promote cell growth. The approval of Xospata was based on the rate of complete remission or complete remission with partial hematologic recovery and the duration of these remissions. In clinical trials, Xospata has been shown to lead to higher response rates and longer survival compared to chemotherapy in patients with FLT3 mutation-positive relapsed or refractory AML.

The ADMIRAL trial, a pivotal phase 3 study, demonstrated that patients receiving Xospata had a significantly higher rate of CR+CRh compared to those who received salvage chemotherapy. Additionally, the median overall survival was significantly longer for patients treated with Xospata. These results underscore the importance of Xospata as a treatment option for patients with FLT3-mutated AML, particularly when the disease has not responded to or has returned after other treatments.