FDA Approves Pemazyre to Treat Advanced Cholangiocarcinoma


The Food and Drug Administration approved Pemazyre for the treatment of patients with previously treated, advanced cholangiocarcinoma.

The Food and Drug Administration (FDA) approved Pemazyre (pemigatinib), the first drug designed to treat patients with previously treated, advanced cholangiocarcinoma who have tumors that have a fusion or other rearrangement of FGRF2.

“This approval demonstrates that while we continue to focus our efforts on addressing the COVID-19 pandemic, the FDA remains committed to the important work of reviewing treatments for patients with cancer and other serious conditions,” Dr. Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a press release.

Patients with this rare form of cancer in the bile ducts are traditionally treated with a chemotherapy combination.

The FDA based its approval on results from a trial, designed to evaluate Pemazyre in 107 patients with locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement who were previously treated.

The overall response rate with Pemazyre was 36%, with 2.8% of patients having a complete response and 33% having a partial response. In the 28 patients with a response, 63% had a response that lasted six months or longer and 18% had a response of 12 months or longer.

Common side effects included hyperphosphatemia and hypophosphatemia (electrolyte disorders), alopecia, diarrhea, nail toxicity, fatigue, dysgeusia, nausea, constipation, stomatitis (sore or inflammation inside the mouth), dry eye, dry mouth, decreased appetite, vomiting, joint pain, abdominal pain, back pain and dry skin. Of note, ocular toxicity is also a risk associated with treatment with Pemazyre.

“With Pemazyre, we considered the observed efficacy results to be clinically meaningful and the overall risk to benefit assessment for patients with tumors harboring FGFR2 gene fusions and other rearrangements to be favorable, particularly when we considered that these patients have no other good options following first line treatment with chemotherapy,” Pazdur said.

Check back later to learn more about what this approval means for patients.

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