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 pemigatinib was demonstrated in the FIGHT-202 study, a multicenter, open-label, single-arm trial. In this trial, patients who received pemigatinib experienced a median progression-free survival of 6.9 months and an overall response rate (ORR) of 36%, with 2.8% achieving a complete response.

Lytgobi (futibatinib) for Bile Duct Cancer

Lytgobi (futibatinib) is another targeted therapy that has shown promise in treating bile duct cancer. Although at the time of writing, Lytgobi has not been approved by the FDA for any indication, it has been evaluated in clinical trials. In particular, the FOENIX-CCA2 trial has been assessing the efficacy of futibatinib in patients with advanced cholangiocarcinoma harboring FGFR2 gene rearrangements. Preliminary results from this trial have indicated that futibatinib has antitumor activity in patients with FGFR2 fusion-positive cholangiocarcinoma, suggesting potential as a treatment option for this patient population.

Both Pemazyre and Lytgobi are part of a class of drugs known as FGFR inhibitors. These drugs work by targeting and inhibiting the activity of FGFR, which can be abnormally active in certain types of cancer, including cholangiocarcinoma. By blocking this pathway, FGFR inhibitors can help to slow down or stop the growth of cancer cells. The presence of FGFR2 fusions or rearrangements in bile duct cancer cells is a key factor in determining the likelihood of response to these medications.

It is important to note that while these therapies have shown efficacy in clinical trials, their effectiveness can vary among individual patients. The decision to use these medications should be based on a comprehensive assessment by a healthcare provider, including the consideration of the specific molecular characteristics of the tumor, prior treatments, and the overall health status of the patient. As with all medications, the benefits of Pemazyre and Lytgobi must be weighed against the potential risks and side effects.