Pemazyre

Cholangiocarcinoma — previously treated, unresectable, locally advanced or metastatic, with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test

13.5 mg orally once daily on Days 1-14 of 21-day cycles
Take with or without food
Dose reductions: 9 mg → 4.5 mg (same 14 days on/7 days off schedule)
Strong CYP3A inhibitors: Reduce to 4.5 mg daily

Tablets: 4.5 mg, 9 mg, 13.5 mg

None listed.

• Hyperphosphatemia: 60%. Related to FGFR inhibition. Monitor serum phosphate levels. Restrict dietary phosphate if levels exceed 5.5 mg/dL. Consider phosphate binders for levels ≥7 mg/dL.
• Ocular Toxicity: 6% serous retinal detachment. Monitor for visual symptoms. Perform OCT at baseline and periodically. Withhold for serous retinal detachment.
• Soft Tissue Mineralization: Due to hyperphosphatemia. Manage phosphate levels aggressively.
• Embryo-Fetal Toxicity

Hyperphosphatemia (60%), alopecia (49%), diarrhea (47%), fatigue (42%), nail toxicity (43%), nausea (40%), dysgeusia (40%), constipation (35%), stomatitis (34%), dry eye (34%), dry mouth (34%), decreased appetite (33%), vomiting (27%), arthralgia (25%), abdominal pain (23%), dry skin (22%), back pain (20%)

Consult the complete prescribing information for a comprehensive list of adverse reactions and their frequencies.

Consult the complete prescribing information for drug interactions, including effects on CYP enzymes, transporters, and concomitant medications that may require dose adjustments or monitoring.

Consult the full prescribing information for pregnancy-related considerations.

Refer to prescribing information for lactation guidance.

Pediatric safety and efficacy information is detailed in the full label.

Dose modifications for organ impairment are specified in the complete prescribing information.

Pemigatinib is a selective, potent, oral inhibitor of FGFR1, FGFR2, and FGFR3 with lower activity against FGFR4. In cholangiocarcinoma, FGFR2 fusions or rearrangements generate constitutively active FGFR2 fusion proteins that drive tumor proliferation. Pemigatinib inhibits these aberrant FGFR2 fusion proteins, blocking downstream RAS-MAPK and PI3K-AKT signaling and inducing tumor cell death.

Tmax: 1-2 hours. Half-life: approximately 15.4 hours. Protein binding: 97.4%. Metabolized primarily by CYP3A4. Fecal excretion (82%), urinary excretion (12%).

Clinical efficacy and safety data supporting the approval are available in the full prescribing information and from the clinical trials listed below.

• FIGHT-202 — Pemigatinib in FGFR2 fusion/rearrangement-positive cholangiocarcinoma. Phase II, n=146.
  🔬 NCT02924376 ↗ 📄 Primary Publication ↗

Pemazyre has FDA-approved indications across the following cancer types covered on PipelineEvidence: