In April 2020, the U.S. Food and Drug Administration (FDA) granted accelerated approval to Pemazyre (pemigatinib) oral tablets, developed by Incyte, for the treatment of previously treated advanced cholangiocarcinoma in adult patients. This is the world's first targeted therapy for cholangiocarcinoma.
Pemigatinib (Pemazyre) Indications and Uses
Pemazyre is a kinase inhibitor indicated for the treatment of previously treated, unresectable locally advanced or metastatic cholangiocarcinoma in adults with fibroblast growth factor receptor 2 (FGFR2) fusions or other rearrangements detected in FDA-approved trials.
This indication was approved under accelerated approval based on overall response rate and time to response. Continued approval for this indication may depend on validation and description of clinical benefit in validation trials.
Pemigatinib (Pemazyre) Dosage and Administration
1. Before initiating Pemazyre treatment, confirm the presence of FGFR2 fusions or rearrangements.
2. The recommended dose is 13.5 mg orally once daily for 14 consecutive days, followed by a 7-day break within a 21-day cycle. Continue treatment until disease progression or unacceptable toxicity occurs.
3. Swallow the tablet whole, with or without food.
Adverse Reactions of Pemitzinib (Pemazyre)
The most common adverse reactions (incidence ≥20%) are hyperphosphatemia, alopecia, diarrhea, nail toxicity, fatigue, dyspepsia, nausea, constipation, stomatitis, dry eye, dry mouth, loss of appetite, vomiting, arthralgia, abdominal pain, hypophosphatemia, back pain, and dry skin.
Use of Pemitzinib (Pemazyre) in Specific Populations
Lactation: Breastfeeding is not recommended.
