Efficacy

Efficacy of Tibsovo (Ivosidenib) in Bile Duct Cancer

Tibsovo (ivosidenib) is an oral medication that targets specific genetic mutations known as isocitrate dehydrogenase-1 (IDH1) mutations. In the context of bile duct cancer, or cholangiocarcinoma, Tibsovo has shown efficacy for patients whose tumors harbor an IDH1 mutation. This subset of patients, although relatively small, can benefit significantly from this targeted therapy. The FDA approved Tibsovo for the treatment of adult patients with IDH1-mutated cholangiocarcinoma following the results of clinical trials that demonstrated a favorable response rate and a disease control rate in patients who had progressed on previous therapies.

In clinical studies, Tibsovo has shown to induce partial responses and stable disease in a proportion of patients with IDH1-mutated bile duct cancer. The median duration of response has been reported to be meaningful, providing a therapeutic option for patients with this specific genetic profile. It is important to note that the efficacy of Tibsovo is contingent upon the presence of an IDH1 mutation, and therefore, genetic testing is essential before initiating treatment with this agent.

Efficacy of Pemazyre (Pemigatinib) in Bile Duct Cancer

Pemazyre (pemigatinib) is another targeted therapy that has demonstrated efficacy in the treatment of bile duct cancer, specifically in patients with tumors that exhibit fibroblast growth factor receptor 2 (FGFR2) fusions or rearrangements. Pemazyre is an FGFR inhibitor that has been approved by the FDA for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or other rearrangement as detected by an FDA-approved test. The approval was based on the overall response rate and duration of response observed in clinical trials.

In the clinical trials that led to its approval, Pemazyre showed a significant overall response rate in patients with FGFR2 fusion-positive cholangiocarcinoma. The treatment has been associated with a clinically meaningful improvement in progression-free survival compared to chemotherapy. Similar to Tibsovo, the use of Pemazyre is highly dependent on the genetic characteristics of the tumor, making the identification of FGFR2 fusions or rearrangements critical before initiation of therapy. Pemazyre offers a new avenue of hope for patients with this specific genetic alteration, who have limited treatment options after first-line therapy.