Efficacy

Copiktra (Duvelisib) Efficacy in Leukemia

Copiktra (duvelisib) is a kinase inhibitor specifically indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior therapies. In clinical trials, duvelisib has demonstrated efficacy in patients with CLL/SLL. The approval of duvelisib for these indications was based on a randomized, multicenter, open-label, phase III study known as DUO. In this study, duvelisib monotherapy showed a significant improvement in progression-free survival (PFS) compared to the control therapy, with a median PFS of 13.3 months versus 9.9 months in the comparator arm.

For the treatment of relapsed or refractory follicular lymphoma (FL), another type of leukemia, after at least two prior systemic therapies, duvelisib has shown activity as well. However, the efficacy in FL is based on overall response rate, and there is no data available that demonstrate an improvement in patient survival or symptoms. It is important to note that the use of Copiktra comes with a boxed warning for serious toxicities such as infections, diarrhea or colitis, cutaneous reactions, and pneumonitis.

Xospata (Gilteritinib) Efficacy in Leukemia

Xospata (gilteritinib) is approved for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test. Gilteritinib has shown efficacy in clinical trials specifically for this subgroup of AML patients. The approval of gilteritinib was based on the results of a multicenter, open-label, single-arm trial. In this study, gilteritinib demonstrated a combined complete remission (CR) and complete remission with partial hematological recovery (CRh) rate, which was a key measure of efficacy. The CR/CRh rate was found to be significantly higher in patients treated with gilteritinib compared to historical controls.

Furthermore, gilteritinib has been associated with an extension in overall survival for patients with FLT3 mutation-positive relapsed or refractory AML. The ADMIRAL trial, a subsequent confirmatory Phase III study, supported the continued approval of gilteritinib, demonstrating a survival benefit over salvage chemotherapy. The median overall survival was significantly longer for patients receiving gilteritinib as compared to those who received standard salvage chemotherapy. These findings underscore gilteritinib as an important therapeutic option for this challenging patient population.