- Pemigatinib is now approved for previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 fusion or other rearrangement.
Why this matters
- The drug is the first targeted therapy for rare bile duct cancer.
- The approval comes with the companion diagnostic FoundationOne CDX for patient selection.
- In the multicenter, open-label, single-arm FIGHT-202 trial (n=107), patients received pemigatinib, 13.5 mg orally, once daily for 14 consecutive days, followed by 7 days off therapy.
- The overall response rate was 36% (95% CI, 27%-45%).
- There were 3 complete responses.
- The median duration of response was 9.1 months.
- 63% of responses lasted 6 months or longer, and 18% lasted 12 months or longer.
- Adverse events with incidence ≥20% were hyperphosphatemia, alopecia, diarrhea, nail toxicity, fatigue, dysgeusia, nausea, constipation, stomatitis, dry eye, dry mouth, decreased appetite, vomiting, arthralgia, abdominal pain, hypophosphatemia, back pain, and dry skin.
- Pemigatinib carries a risk for ocular toxicity and hyperphosphatemia.
- The recommended dose is 13.5 mg orally once per day for 14 consecutive days, followed by 7 days off therapy in 21-day cycles.
- Full prescribing information.