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Pemigatinib

Pemigatinib is an innovative drug being studied in clinical trials for the treatment of various types of cancer. This article explores the use of pemigatinib in different clinical trials, focusing on its potential benefits for patients with cancers characterized by specific genetic alterations, particularly in the FGFR (Fibroblast Growth Factor Receptor) genes.

Table of Contents

What is Pemigatinib?

Pemigatinib is a targeted cancer therapy drug. It is also known by the brand name Pemazyre[1] or by its research code INCB054828[2]. This medication is designed to treat specific types of cancers that have certain genetic changes in their cells[3].

How Does Pemigatinib Work?

Pemigatinib works by targeting and blocking proteins called fibroblast growth factor receptors (FGFRs), specifically FGFR1, FGFR2, and FGFR3. These proteins are involved in cancer cell growth and survival. By blocking these proteins, pemigatinib can help slow down or stop the growth of cancer cells[3].

What Cancers Can Pemigatinib Treat?

Pemigatinib is being studied for the treatment of various types of cancers, including:

  • Cholangiocarcinoma (bile duct cancer): Pemigatinib is approved in Europe for treating a specific type of cholangiocarcinoma with FGFR2 alterations[4].
  • Urothelial cancer (bladder cancer and cancers of the urinary system)[2]
  • Myeloid/lymphoid neoplasms (certain blood cancers)[5]
  • Other solid tumors with specific FGFR alterations, such as breast cancer, lung cancer, and soft tissue sarcomas[6]

It’s important to note that for many of these cancers, pemigatinib is still being studied and may not be approved for regular use yet.

How is Pemigatinib Administered?

Pemigatinib is taken orally (by mouth) as a tablet. The most common dosing schedule is:

  • 13.5 mg once daily for 14 days, followed by 7 days off (21-day cycle)[1]

Some studies are also looking at continuous daily dosing. Your doctor will determine the best dosing schedule for you based on your specific situation and how you respond to the treatment[2].

Effectiveness of Pemigatinib

The effectiveness of pemigatinib is measured in several ways in clinical trials:

  • Objective Response Rate (ORR): This measures how many patients have a significant reduction in tumor size after treatment[7].
  • Progression-Free Survival (PFS): This measures how long patients live without their cancer getting worse[7].
  • Overall Survival (OS): This measures how long patients live after starting treatment[7].
  • Duration of Response (DOR): This measures how long the cancer remains controlled after it starts responding to treatment[7].

The exact effectiveness can vary depending on the type of cancer and its specific genetic alterations. Your doctor can provide more specific information based on your individual case.

Side Effects and Safety

Like all medications, pemigatinib can cause side effects. Common side effects may include:

  • High phosphate levels in the blood (hyperphosphatemia)
  • Eye problems, including changes to the cornea and retina
  • Fatigue
  • Nausea and vomiting
  • Diarrhea or constipation
  • Changes in taste
  • Mouth sores

Your doctor will monitor you closely for these and other side effects. They may adjust your dose or recommend treatments to manage side effects if they occur[8].

Ongoing Research

Pemigatinib is still being studied in many clinical trials. These studies are looking at its effectiveness in different types of cancers, different stages of disease, and in combination with other treatments. Some areas of ongoing research include:

  • Using pemigatinib after surgery in cholangiocarcinoma to prevent cancer recurrence[1]
  • Combining pemigatinib with immunotherapy drugs[6]
  • Using pemigatinib in early-stage bladder cancer[9]
  • Studying pemigatinib in a wide range of solid tumors with specific genetic alterations[3]

These ongoing studies will help researchers better understand how to use pemigatinib most effectively and safely in different situations.

Aspect Details
Drug Name Pemigatinib (also known as INCB054828)
Mechanism of Action Inhibitor of FGFR1, 2, and 3
Cancer Types Studied Urothelial carcinoma, cholangiocarcinoma, myeloid/lymphoid neoplasms, various solid tumors with FGFR alterations
Common Dosing 13.5 mg orally once daily, 2 weeks on / 1 week off in 21-day cycles
Key Outcomes Measured Objective response rate, progression-free survival, overall survival, duration of response, safety
Notable Side Effects Hyperphosphatemia, eye problems, treatment-emergent adverse events
Patient Selection Often based on presence of specific FGFR gene alterations

FAQ

  • What is pemigatinib and how does it work? Pemigatinib is a targeted therapy drug that inhibits FGFR1, 2, and 3. It works by blocking the activity of these receptors, which are often altered in certain types of cancer, potentially slowing or stopping tumor growth.
  • What types of cancers are being studied with pemigatinib? Clinical trials are investigating pemigatinib for various cancers including urothelial carcinoma (bladder cancer), cholangiocarcinoma (bile duct cancer), myeloid/lymphoid neoplasms, and other solid tumors with FGFR alterations.
  • How is pemigatinib administered in clinical trials? Pemigatinib is typically given orally, once daily. The most common dosing schedule is 13.5 mg taken for 2 weeks, followed by 1 week off, in 21-day cycles. Some trials also explore continuous dosing.
  • What are the main outcomes being measured in pemigatinib trials? Key outcomes include objective response rate (tumor shrinkage), progression-free survival, overall survival, duration of response, and safety profiles. Some trials also assess quality of life measures.
  • Are there any common side effects of pemigatinib? Common side effects may include hyperphosphatemia (high phosphate levels in blood), eye problems, and other treatment-emergent adverse events. Regular monitoring and management of side effects are part of the clinical trial protocols.

Ongoing Clinical Trials on Pemigatinib

  • Study on the Effectiveness of Tepotinib and Drug Combination for Patients with Advanced Cancer

    Recruiting

    1 1 1
    Investigated drugs:
    Norway

  • Study of Drug Combination for Advanced Cancer Patients with Known Molecular Profile – FINPROVE Study

    Recruiting

    1 1 1
    Investigated drugs:
    Finland

  • Study on the Effectiveness of Entrectinib and Other Drug Combinations for Patients with Advanced Solid Tumors, Multiple Myeloma, or Non-Hodgkin Lymphoma

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

  • Study of drug combination in patients with advanced cancer based on genomic profiling – ProTarget Danish nationwide trial

    Recruiting

    1 1 1
    Investigated drugs:
    Denmark

  • Study of Pemigatinib and Retifanlimab for Patients with Advanced Dedifferentiated Liposarcoma

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Denmark Norway Sweden

  • Study of Pemigatinib for Patients with Recurrent or Metastatic Solid Tumors with FGFR Gene Alteration

    Recruiting

    1 1 1
    Investigated drugs:
    France

  • Molecularly Tailored Therapy Versus Standard Care in Patients With Advanced Pancreatic Cancer Using Olaparib, Erlotinib, Crizotinib, Trametinib, Pemigatinib, Selpercatinib, Axitinib, Vismodegib, Pembrolizumab, Larotrectinib, Dabrafenib, Capecitabine, Oxaliplatin, Fluorouracil, Irinotecan, Gemcitabine, Paclitaxel Albumin-Bound, and Calcium Folinate

    Not yet recruiting

    1 1 1
    Investigated drugs:
    Denmark

  • Study of Pemigatinib for Patients with Advanced Intrahepatic Cholangiocarcinoma with FGFR2 Changes After Local Therapy

    Not recruiting

    1 1 1
    Investigated drugs:
    Germany

  • Study Comparing Pemigatinib and Gemcitabine Plus Cisplatin for First-Line Treatment in Patients with Unresectable or Metastatic Cholangiocarcinoma with FGFR2 Rearrangement

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium France Germany Italy The Netherlands +2

  • Study of Retifanlimab Alone and in Combination with Drug Therapy for Adults with Advanced or Metastatic Endometrial Cancer Who Have Not Responded to Platinum Chemotherapy

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium France Greece Italy

Glossary

  • FGFR: Fibroblast Growth Factor Receptor, a type of protein found on cells that plays a role in cell growth and division. Alterations in FGFR genes can contribute to cancer development.
  • Urothelial Carcinoma: A type of cancer that typically occurs in the urinary system, most commonly in the bladder.
  • Cholangiocarcinoma: A type of cancer that forms in the bile ducts, which are tubes that connect the liver to the small intestine.
  • Objective Response Rate (ORR): The proportion of patients whose tumor shrinks or disappears after treatment.
  • Progression-Free Survival (PFS): The length of time during and after treatment that a patient lives with cancer without it worsening.
  • Overall Survival (OS): The length of time from the start of treatment that patients are still alive.
  • RECIST: Response Evaluation Criteria in Solid Tumors, a set of rules used to measure how well a cancer patient responds to treatment.
  • Hyperphosphatemia: A condition where there is an abnormally high level of phosphate in the blood.
  • Transurethral Resection of Bladder Tumor (TURBT): A surgical procedure to diagnose bladder cancer and remove cancerous tissue from the bladder.
  • Myeloid/Lymphoid Neoplasms: A group of blood cancers that affect the production and function of blood cells.

References

  1. https://clinicaltrials.eu/trial/study-of-pemigatinib-for-patients-with-advanced-intrahepatic-cholangiocarcinoma-with-fgfr2-changes-after-local-therapy/
  2. https://clinicaltrials.gov/study/NCT02872714
  3. https://clinicaltrials.gov/study/NCT06022289
  4. https://clinicaltrials.gov/study/NCT06653777
  5. https://clinicaltrials.gov/study/NCT03011372
  6. https://clinicaltrials.gov/study/NCT06551896
  7. https://clinicaltrials.gov/study/NCT04003623
  8. https://clinicaltrials.gov/study/NCT04294277
  9. https://clinicaltrials.gov/study/NCT03914794