FDA Approves Pemigatinib as First Therapy for Advanced Cholangiocarcinoma

On April 17, 2020, the FDA approved pemigatinib (Pemazyre; Incyte) for the treatment of adults with certain types of pretreated, advanced cholangiocarcinoma. The application for this drug was granted Priority Review, Breakthrough Therapy designation, and Orphan Drug designation by the FDA.

“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,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence, in a press statement.

The FDA’s approval of pemigatinib was based on a clinical trial of 107 pretreated patients with locally advanced or metastatic cholangiocarcinoma and an FGFR2 fusion or rearrangement. These patients were given pemigatinib once daily for 14 consecutive days, followed by 7 days without the drug, in 21-day cycles until disease progression or an unreasonable level of side effects occurred.

Patients were scanned every 8 weeks, and established criteria were used to measure the overall response rate.

Ultimately, the overall response rate was found to be 36%, with 2.8% of patients having a complete response and 33% having a partial response. Among the 38 responders, 24 (63%) and 7 (18%) patients had responses lasting ≥6 months and ≥12 months, respectively.

“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,” Dr Pazdur said in the press release.

The most frequently reported (≥20%) adverse reactions were hyperphosphatemia and hypophosphatemia, alopecia, diarrhea, nail toxicity, fatigue, dysgeusia, nausea, constipation, stomatitis, dry eye, dry mouth, decreased appetite, vomiting, joint pain, abdominal pain, back pain, and dry skin.

Of note, ocular toxicity is also a potential risk tied to pemigatinib use.—Hina Porcelli

Source: US Food and Drug Administration. FDA Approves First Targeted Treatment for Patients with Cholangiocarcinoma, a Cancer of Bile Ducts. April 17, 2020. www.fda.gov/news-events/press-announcements/fda-approves-first-targeted-treatment-patients-cholangiocarcinoma-cancer-bile-ducts. Accessed April 20, 2020.