Efficacy

Pemazyre (pemigatinib) for Bile Duct Cancer

Pemazyre (pemigatinib) is a targeted therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (bile duct cancer) with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. The efficacy of Pemazyre was evaluated in a multicenter, open-label, single-arm trial known as FIGHT-202. The study demonstrated a significant objective response rate (ORR) in patients with FGFR2 fusions or rearrangements, indicating that the drug can induce tumor shrinkage in a proportion of patients.

The clinical trial showed that Pemazyre achieved an ORR of 36% with a median duration of response (DoR) of 9.1 months. This suggests that not only can Pemazyre effectively induce responses in a subset of patients with bile duct cancer, but these responses can also be durable. However, it is important to note that Pemazyre is not suitable for all patients with bile duct cancer, but rather those who have specific genetic alterations in FGFR2.

Truseltiq (infigratinib) for Bile Duct Cancer

Truseltiq (infigratinib) is another FGFR inhibitor that has been granted accelerated approval by the FDA for the treatment of previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or other rearrangement. The approval of Truseltiq was based on the results of a multicenter, open-label, single-arm clinical trial. This trial, known as CBGJ398X2204, assessed the efficacy of Truseltiq in patients with FGFR2 fusion or rearrangement-positive cholangiocarcinoma who had progressed on or after at least one prior line of systemic therapy.

In the trial, Truseltiq demonstrated an ORR of 23% with a median DoR of 5 months. While the ORR with Truseltiq is lower compared to Pemazyre, it still represents a meaningful therapeutic option for patients with limited treatment choices. The duration of response, though shorter than that seen with Pemazyre, indicates that Truseltiq can provide a temporary reduction in tumor burden for some patients. It is critical for patients to undergo appropriate biomarker testing to determine eligibility for treatment with Truseltiq, as it is specifically indicated for those with FGFR2 genetic alterations.