Pemazyre: Dosage, side effects, cost, uses, and more

Here are answers to some frequently asked questions about Pemazyre.

How does Pemazyre work?

Pemazyre is a targeted therapy. It’s designed to slow or stop the growth of cancer cells. It works by inhibiting (blocking) specific proteins in cancer cells from functioning.

Pemazyre targets fibroblast growth factor receptor proteins that are abnormal due to changes in the genes that encode these proteins. These abnormal proteins help cancer cells grow and survive. When Pemazyre blocks these proteins, the cancer cells may stop growing. This can stop the cancer from progressing (worsening) and may cause tumors to become smaller.

If you have questions about how Pemazyre works, talk with your doctor or pharmacist.

Does Pemazyre cause long-term side effects?

Pemazyre isn’t known to cause long-term side effects. If you develop certain bothersome or severe side effects, such as retinal pigment epithelial detachment, your doctor may have you pause or stop treatment. Side effects should go away soon after the drug is stopped.

Pemazyre was approved by the Food and Drug Administration (FDA) in 2020. As with any drug that’s relatively new, it’s possible that long-term side effects or risks could be identified in the future.

If you have questions about Pemazyre’s possible side effects or how long they might last, talk with your doctor or pharmacist.

Will Pemazyre cure bile duct cancer?

It’s possible, but the drug hasn’t been shown to cure cancer. In a clinical trial for bile duct cancer,* taking Pemazyre was shown to cause a partial response in some participants. This means the tumors of some participants shrunk, as seen on imaging scans. A few participants in these trials experienced a complete response with Pemazyre treatment. This means their tumor disappeared or became too small to detect on the scans.

Having a complete response doesn’t necessarily mean that the cancer is cured or won’t come back in the future. And your results may vary from what was seen in clinical trials.

Talk with your doctor if you have questions about what to expect with Pemazyre treatment.

* For this use, Pemazyre has received accelerated approval from the FDA. This means the drug has been approved for this purpose based on limited data from early clinical trials. A decision regarding full approval will be made once additional clinical trials have been completed.