What is Pemigatinib?
Pemigatinib is a targeted therapy drug that specifically inhibits fibroblast growth factor receptors (FGFRs). It is primarily used in the treatment of certain types of cancer that exhibit aberrant FGFR signaling. The drug is a tyrosine kinase inhibitor, which means it works by blocking the enzyme tyrosine kinase, a key player in cell signaling pathways involved in cancer cell growth and survival.
FDA Approval
Pemigatinib received its first FDA approval in April 2020 for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement. This approval was based on clinical trial results that demonstrated the drug's efficacy in this specific patient population.Mechanism of Action
Pemigatinib works by inhibiting FGFR1, FGFR2, and FGFR3, which are often overactive or mutated in certain cancers. By blocking these receptors, pemigatinib disrupts the cancer cells' ability to proliferate and survive. This action can lead to reduced tumor growth and, in some cases, tumor shrinkage.Who Can Benefit From Pemigatinib?
Pemigatinib is primarily used for patients with cholangiocarcinoma who have specific genetic alterations in the FGFR2 gene. Before starting treatment, patients typically undergo genetic testing to identify these alterations. The drug is also being investigated for its efficacy in other FGFR-altered cancers, including urothelial carcinoma and glioblastoma.
Clinical Trials
The approval of pemigatinib was largely based on the results from the FIGHT-202 trial, which enrolled patients with cholangiocarcinoma harboring FGFR2 fusions or rearrangements. The trial showed a significant overall response rate, leading to its accelerated approval. Ongoing clinical trials are exploring its utility in other cancers, expanding its potential applications.Administration and Dosage
Pemigatinib is administered orally, typically in a 21-day cycle: 13.5 mg once daily for 14 consecutive days followed by 7 days off therapy. This cycle is repeated until disease progression or unacceptable toxicity occurs. Dosage adjustments may be necessary based on the patient's tolerance and side effects.Side Effects
Common side effects of pemigatinib include hyperphosphatemia, alopecia, diarrhea, and fatigue. More serious adverse events can occur, such as retinal detachment and elevated liver enzymes. Patients are closely monitored for these potential side effects, and supportive treatments are provided as needed.Drug Interactions
Pemigatinib can interact with other medications, including strong CYP3A4 inhibitors and inducers, which can affect its metabolism. Patients should inform their healthcare provider of all medications and supplements they are taking to avoid potential interactions.Monitoring and Follow-Up
Patients on pemigatinib require regular follow-up to monitor for effectiveness and side effects. This includes routine blood tests to check for hyperphosphatemia and liver function, as well as periodic eye exams to detect any retinal changes.Future Directions
Research is ongoing to explore additional indications for pemigatinib, including its use in combination with other therapies. Studies are also focusing on understanding resistance mechanisms to improve long-term outcomes for patients.Conclusion
Pemigatinib represents a significant advancement in the treatment of FGFR-altered cancers, offering hope to patients with limited options. Its targeted mechanism of action and ongoing research promise to expand its role in oncology, making it a crucial tool in the fight against cancer.
