On April 17, 2020, the FDA approved Pemazyre (pemigatinib; from Incyte)—an oral inhibitor of FGFR gene fusions or rearrangements 1, 2, and 3—for the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma and the biomarker FGFR2 gene fusions.
An FDA-approved biomarker test should be done to confirm the presence of the FGFR2 biomarker before the patient can receive this treatment. The new next-generation sequencing test FoundationOne CDx (from Foundation Medicine) was approved by the FDA on the same day as Pemazyre, and is the only test approved to determine if patients with cholangiocarcinoma have this biomarker.
“Although our nation’s emphasis is on the need to combat COVID-19, patients with cancer and their unique needs continue to be a top priority for the FDA,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence. “We continue to expedite oncology product development in this critical time.”
Pemazyre is the first targeted therapy approved by the FDA for patients with advanced cholangiocarcinoma, a rare gastrointestinal cancer, and this biomarker. Until now, the standard treatment for this patient population has been chemotherapy combinations. Approximately 9% to 14% of patients with cholangiocarcinoma have FGFR2 gene fusions.
Although cholangiocarcinoma is a rare cancer, “it has been on the rise over the past three decades,” said Ghassan Abou-Alfa, MD, Memorial Sloan Kettering Cancer Center, in a press release. “It is encouraging to have a new targeted treatment option for patients who historically have had limited options after first-line chemotherapy or surgery.”
“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,” said Dr. Pazdur.
Activating gene fusions, rearrangements, and translocations in the FGFR biomarker play an important role in cancer cells' growth and survival.
In the clinical trial that led to the FDA approval of Pemazyre, of the 107 patients in the study, 38 patients had a response to treatment, and the responses lasted from 6 months to more than 12 months, with an average response of 9.1 months.
The most common side effects with Pemazyre are electrolyte disorders, hair loss, diarrhea, fatigue, taste distortion, nausea, constipation, mouth inflammation, dry eye and mouth, low appetite, vomiting, joint pain, abdominal pain, back pain, and dry skin.
