Pemigatinib gains approval for metastatic cholangiocarcinoma subgroup

  • US Food and Drug Administration

  • curated by Jim Kling
  • Univadis Clinical Summaries
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Takeaway

  • 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

Key points

  • 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.