Efficacy

Copiktra (Duvelisib) Efficacy in Leukemia

Copiktra (duvelisib) is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and is specifically a dual inhibitor of PI3K-delta and PI3K-gamma isoforms. It has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies. The efficacy of Copiktra in CLL/SLL was primarily shown in a clinical trial where the overall response rate (ORR) was observed to be significant in patients who had previously undergone multiple lines of therapy, indicating a beneficial effect in this difficult-to-treat population.

In addition to CLL/SLL, Copiktra has also shown efficacy in relapsed or refractory follicular lymphoma (FL), another type of leukemia, after at least two prior systemic therapies. The approval for this indication was based on a single-arm trial where duvelisib demonstrated an ORR of 42%, with a median duration of response of 9.9 months. While FL is not the primary indication, the results suggest that Copiktra may provide a therapeutic option for patients with this form of leukemia who have limited treatment choices.

Tibsovo (Ivosidenib) Efficacy in Leukemia

Tibsovo (ivosidenib) is an oral, targeted inhibitor of the isocitrate dehydrogenase-1 (IDH1) enzyme, which is mutated in a variety of malignancies, including certain types of leukemia. Tibsovo is FDA-approved for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation as detected by an FDA-approved test. The efficacy of Tibsovo was evaluated in a clinical trial involving patients with relapsed or refractory AML, and the results showed a significant ORR with 41.6% of patients achieving a complete remission (CR) or a complete remission with partial hematologic recovery (CRh).

Furthermore, Tibsovo has been approved for the treatment of newly-diagnosed AML in adult patients who are aged 75 years or older, or who have comorbidities that preclude the use of intensive induction chemotherapy, and have an IDH1 mutation. In a clinical trial for this patient population, Tibsovo demonstrated a CR+CRh rate of 42.9%, highlighting its potential as a first-line treatment option for AML patients with specific genetic mutations who cannot tolerate more aggressive chemotherapy regimens.