‘Majority of Patients’ With Cholangiocarcinoma Still Receive Upfront Standard of Care Despite Recent FDA Drug Approvals

Despite treatment advancements and Food and Drug Administration (FDA) approvals of drugs in the cholangiocarcinoma (a type of bile duct cancer) space in recent years, there are still many patients with this disease who are not eligible for targeted therapies as a first treatment option, highlighting a significant need for new developments in the field.

Over the past few years, there have been three Food and Drug Administration (FDA) approvals for drugs that treat cholangiocarcinoma:

• Pemazyre (pemigatinib) for previously treated advanced cholangiocarcinoma that has a FGFR2 rearrangement of fusion.
• Truseltiq (infigratinib) for previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement.
• Tibsovo (ivosidenib) for cholangiocarcinoma that harbors an IDH1 mutation.

Since the approvals are indicated for the treatment of specific characteristics of the disease, such as mutations or rearrangements, not everyone will be eligible to be treated with them, leaving a large population to still receive standard of care.

“The majority of patients will still, in the first line, see standard of care: gemcitabine (and) cisplatin. Another relatively new advancement in the space of cholangiocarcinoma was the addition of (Imfinzi; durvalumab), an immune-oncology drug added to standard of care, showing modest but still an improvement in responses,” said Jesper B. Andersen, associate professor and group leader at the University of Copenhagen and the Biotech Research and Innovation center at the Department of Health, in an interview with CURE®.

Transcription

We certainly do have patients that, unfortunately, are not covered by any of those three drugs. (Those who) do not have the fusion, first of all, that might be around 10 or 12%, at best. Quite often, we actually see close to 5% of patients that have FGFR2 to fusion. So that's one it's a little broader, with (Truseltiq) against FGFR2 alterations, because there are FGFR 1 through 3 alterations and amplification, so on, so there's a little more alterations in that space. And (then there’s the) FGFR fusion itself.

And then IDH1 obviously is a very specific alteration. It is a point mutation, which is also what we call a “hotspot.” So there's only one alteration typically in that gene. And that is what (Tibsovo) is targeting. And that is again 10 to 15% of patients.

So the targeted therapies, not quoting me on the percentage, but we might be currently covering 25, or maybe 30% at best with those three approvals. So the majority of patients will still, in the first line, see standard of care: gemcitabine (and) cisplatin. Now there another relatively new advancement in the space of cholangiocarcinoma was the addition of (Imfinzi; durvalumab), an immune-oncology drug added to standard of care, showing modest but still an improvement in responses.

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