Efficacy

Efficacy of Tibsovo (Ivosidenib) in Treating Bile Duct Cancer

Tibsovo (ivosidenib) is a targeted therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma (bile duct cancer) that harbors an isocitrate dehydrogenase 1 (IDH1) mutation. The efficacy of Tibsovo in bile duct cancer was demonstrated in a multicenter, single-arm, open-label clinical trial. The trial's primary endpoint was overall response rate (ORR), which reflects the proportion of patients who experienced a significant reduction in tumor size. The results showed that Tibsovo achieved an ORR of 36% in patients with IDH1-mutated cholangiocarcinoma, with 2.4% of patients having a complete response and 33.6% having a partial response.

Furthermore, the duration of response (DOR) was another critical measure in the trial. Among the responders, 63% had a DOR of six months or longer, indicating that the responses were durable for a significant period. These results suggest that Tibsovo can be an effective treatment option for patients with this specific genetic mutation in bile duct cancer, offering a promising alternative for those who have exhausted other treatment options.

Efficacy of Lytgobi (Futibatinib) in Treating Bile Duct Cancer

Lytgobi (futibatinib) is a novel, oral, irreversible inhibitor of the fibroblast growth factor receptor (FGFR) family, which has been implicated in the development and progression of various cancers, including bile duct cancer. While not yet approved by the FDA specifically for bile duct cancer, futibatinib has shown promising results in clinical trials. In a phase 2 study involving patients with FGFR2 gene fusions or rearrangements who had previously received systemic therapy, futibatinib demonstrated an ORR of 37.7%. This included a complete response rate of 3.2% and a partial response rate of 34.5%, indicating a significant tumor shrinkage in a substantial proportion of patients.

The study also evaluated the DOR, progression-free survival (PFS), and overall survival (OS) as secondary endpoints. The median DOR was not reached at the time of data cutoff, suggesting that the responses could be long-lasting. The median PFS was 9.0 months, and the median OS was 21.7 months, which are encouraging figures in the context of bile duct cancer, a disease known for its poor prognosis. These results support the potential use of futibatinib for patients with FGFR2-driven bile duct cancer, particularly for those who have limited treatment options due to the refractory nature of their disease.