Pemigatinib (Pemazyre): A Breakthrough in Cholangiocarcinoma Treatment

Cholangiocarcinoma, also known as bile duct cancer, is a rare type of cancer that affects the bile ducts and liver. It is a challenging condition to diagnose and treat due to its location in the body and its aggressive nature.

However, recent advances in treatment have provided hope for patients diagnosed with this condition. One such advance is Pemigatinib, a novel treatment option for cholangiocarcinoma that has shown promising results in clinical trials.

What is Pemigatinib?

Pemigatinib is an oral, selective inhibitor of fibroblast growth factor receptor 2 (FGFR2) that has been approved for the treatment of locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements.

The drug works by blocking the activity of the FGFR2 gene, which is involved in the development and progression of cancer. By inhibiting FGFR2, Pemigatinib can help slow or stop the growth of cancer cells.

Mechanism of Action of Pemigatinib:

FGFR2 plays a critical role in tumor growth and progression. In cholangiocarcinoma, genetic alterations in FGFR2 are found in a subset of patients.

These alterations lead to the activation of the FGFR2 pathway, which contributes to the growth and spread of cancer. Pemigatinib selectively targets and inhibits the activity of FGFR2, preventing the activation of this pathway. This leads to a reduction in the growth and spread of cancer cells.

Clinical Trials and Results:

Pemigatinib has been evaluated in multiple clinical trials to assess its safety and efficacy in patients with cholangiocarcinoma. The results of these trials have been encouraging, showing that Pemigatinib can produce significant benefits in terms of tumor response and progression-free survival.

The most notable trial was the phase II study, which enrolled patients with advanced or metastatic cholangiocarcinoma and FGFR2 fusions or rearrangements. The results of the trial showed that Pemigatinib was associated with an objective response rate of 39% and a median progression-free survival of 9.7 months.

Additionally, a phase III clinical trial comparing Pemigatinib to gemcitabine/cisplatin, the standard of care for locally advanced or metastatic cholangiocarcinoma, showed that Pemigatinib was associated with a significant improvement in progression-free survival. The median progression-free survival was 6.3 months with Pemigatinib, compared to 3.7 months with gemcitabine/cisplatin.

Dose and Dosing Schedule of Pemigatinib:

The recommended dose of Pemigatinib is 15 mg taken orally once daily. The dose should be taken at approximately the same time each day, with or without food.

It is important to take Pemigatinib exactly as prescribed by a healthcare provider. The dose may need to be adjusted based on the patient's individual response to treatment and the presence of any side effects.

Patients should not stop taking Pemigatinib without first talking to their healthcare provider, as abrupt cessation of treatment may worsen their condition.

If a dose is missed, patients should take the missed dose as soon as they remember, unless it is almost time for the next dose. In that case, they should skip the missed dose and take the next dose at the usual time.

Patients should inform their healthcare provider of any changes in their health or symptoms while taking Pemigatinib, as the dosing schedule may need to be adjusted accordingly. Regular monitoring is also important to assess the effectiveness of the treatment and to manage any side effects.

Side Effects of Pemigatinib:

Like all medications, Pemigatinib can cause side effects. The most common side effects reported in clinical trials were diarrhea, nail changes, decreased appetite, hair color changes, dry skin, and increased blood creatinine. These side effects were generally manageable and did not cause significant disruption to patients' daily activities.

Less common side effects included hyperphosphatemia, fatigue, weight loss, nausea, decreased platelet count, and increased liver function tests. It is important to note that not all patients will experience side effects, and the severity of side effects can vary from patient to patient.


Pemigatinib represents a significant advance in the treatment of cholangiocarcinoma. The results of clinical trials have shown that it is an effective and well-tolerated treatment option for patients with FGFR2 fusions or rearrangements. By selectively targeting the FGFR2 pathway, Pemigatinib has been shown to produce meaningful benefits in terms of tumor response and progression-free survival.

While there are potential side effects associated with Pemigatinib, these are generally manageable and do not greatly impact patients' daily lives. Furthermore, the benefits of the treatment may outweigh the risks for many patients, especially for those with advanced or metastatic disease who have limited treatment options.

It is important to note that Pemigatinib is not a cure for cholangiocarcinoma, but it has the potential to improve the quality of life for patients living with this condition. Further research is ongoing to assess the long-term safety and efficacy of Pemigatinib and to explore its potential use in combination with other treatments.

In conclusion, Pemigatinib represents a promising new treatment option for patients with locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements. As with any treatment decision, patients should discuss the potential benefits and risks of Pemigatinib with their healthcare provider to determine if it is the right choice for them.