marked.bio

FGFR inhibitor · small molecule

pemigatinib

9 active trials across 5 of our biomarkers, targeting FGFR1-3. 2 active approvals for a biomarker on marked.bio.

Also known as INCB054828, Pemazyre.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

TherapyBiomarkerTumourUnited StatesEuropean Union
pemigatinib
FDA, 2020
previously treated, unresectable locally advanced or metastatic, FGFR2 fusion or other rearrangement FDA accelerated approval 17/04/2020; companion test FoundationOne CDx. Source
EU (EMA), 2021
locally advanced or metastatic, FGFR2 fusion or rearrangement, after >=1 prior line of systemic therapy EU conditional authorisation 26/03/2021. Source
Pemazyre 		FGFR2 fusion or rearrangement cholangiocarcinoma Approved 2020 accelerated Approved 2021 conditional

In the FDA approval summary

FIGHT-202 single-arm, 107 patients
Response 36% (27-45)Lasting 9.1 months

Trial details and side effects
Patients
previously treated FGFR2 fusion/rearrangement cholangiocarcinoma (NCT02924376)
Most common side effects
high phosphate, alopecia, diarrhoea, nail toxicity, fatigue, taste changes, nausea, constipation, stomatitis, dry eye, dry mouth, decreased appetite
Notes
FDA approval summary (2020); side effects are those in 20% or more of patients. Eye toxicity and high phosphate need monitoring.
Source
FDA approval summary

In the EU summary (EPAR)

FIGHT-202 single-arm, 108 patients
Response 37%Lasting 8 months on average

Trial details and side effects
Patients
previously treated FGFR2 fusion/rearrangement cholangiocarcinoma (NCT02924376)
Most common side effects
high phosphate, alopecia, diarrhoea, nail toxicity, fatigue, taste changes, nausea, constipation, stomatitis, dry eye, dry mouth, decreased appetite
Notes
EU figures from the EPAR summary; trial details and side effects from the US approval.
Source
EU summary (EPAR)

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
FGFR2 fusion or rearrangement 7 32 Phase 2
FGFR3 alteration (mutation or fusion) 5 25 Phase 2
FLT3 mutation (ITD/TKD) 1 1 Phase 1
IDH1/2 mutation 0 1 –
MSI-H / dMMR 0 1 –

Sponsors

Industry sponsors leading active biomarker-selected trials of pemigatinib.

  1. Incyte 1

Trials

9 of 36 trials shown

  1. Alternating Chemo-immunotherapy and FGFR Inhibitor for FGFR-positive Biliary or Cholangiocarcinoma

    Recruiting Phase 2 Ohio State University Comprehensive Cancer Center Started 2026Primary completion 2027-12 NCT07780838

    cisplatindurvalumabgemcitabinepemigatinib FDA EU

    Patients must have tumors classified as having FGFR2 gene fusion/rearrangements or other gain-of function alterations involved in FGFR as detected by any …

    FGFR2 fusion or rearrangementLocally Advanced Ampulla of Vater Carcinoma, Locally Advanced Biliary Tract Carcinoma, Locally Advanced Extrahepatic Cholangiocarcinoma, Locally Advanced Gallbladder Carcinoma

  2. A Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Advanced/Metastatic or Surgically Unresectable Cholangiocarcinoma With FGFR2 Fusion or Rearrangements After at Least 1 Prior Line of Systemic Therapy

    Not yet recruiting Phase 2 Incyte Started 2026Primary completion 2031-02 NCT07862998

    pemigatinib FDA EU

    Documented FGFR2 fusion or rearrangement

    FGFR2 fusion or rearrangementUnresectable Cholangiocarcinoma, Advanced/Metastatic Cholangiocarcinoma

  3. Efficacy of Pemigatinib in Patients With Solid Tumors Characterized by an Alteration of the Gene FGFR in Tumor Cells

    Recruiting Phase 2 UNICANCER Started 2025Primary completion 2027-06 NCT06653777

    pemigatinib

    …nt with locally recurrent unresectable and/or advanced or metastatic disease harbouring a FGFR1,2,3 fusion/rearrangement or mutation

    FGFR3 alteration (mutation or fusion)Solid Tumor, Miscellaneous

  4. A Phase II Nationwide, Fully Decentralized, Telemedicine Study of Pemigatinib in Adult Patients With Advanced or Metastatic Pancreatic Cancer With FGFR Genetic Alterations

    Recruiting Phase 2 Sameek Roychowdhury Started 2025Primary completion 2026-12 NCT06906562

    pemigatinib

    … central Clinical Laboratory Improvement Act (CLIA)-certified laboratory determination of FGFR gene fusions/translocations or activating mutations

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementAdvanced Pancreatic Carcinoma, Metastatic Pancreatic Carcinoma, Stage II Pancreatic Cancer AJCC v8, Stage III Pancreatic Cancer AJCC v8

  5. Pemigatinib and Immune Checkpoint Inhibitor Treated FGFR1/2/3 Alteration Advanced Solid Tumor

    Not yet recruiting Phase 2 Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University Started 2024Primary completion 2026-07 NCT06551896

    pemigatinib

    Pemigatinib and Immune Checkpoint Inhibitor Treated FGFR1/2/3 Alteration Advanced Solid Tumor

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementStatus may be outdated, last updated 2024-08-13Urothelial Carcinoma, Breast Neoplasms, Lung Cancer, Gastric Cancer

  6. Pemigatinib Combined With Durvalumab for Previously Treated Biliary Tract Carcinoma

    Not yet recruiting Phase 2 Eastern Hepatobiliary Surgery Hospital Started 2024Primary completion 2026-08 NCT06530823

    pemigatinib FDA EUdurvalumab

    …d, metastatic, or unresectable cholangiocarcinoma or gallbladder cancer confirmed to have FGFR2 fusion or rearrangement;

    FGFR2 fusion or rearrangementStatus may be outdated, last updated 2024-07-31Biliary Tract Carcinoma, Cholangiocarcinoma

  7. Pemigatinib in the Advanced Gastrointestinal Cancer With FGFR 1-3 Alterations

    Recruiting Phase 2 Tianjin Medical University Cancer Institute and Hospital Started 2022Primary completion 2024-12 NCT05651672

    pemigatinib

    With histologically confirmed FGFR1-3 alterations, including but not limited to amplification, mutation, fusion/rearrangement

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementStatus may be outdated, last updated 2022-12-15Gastrointestinal Cancer

  8. Pemigatinib + Afatinib in Advanced Refractory Solid Tumors

    Recruiting Phase 1 Massachusetts General Hospital Started 2024Primary completion 2027-12 NCT06302621

    afatinibpemigatinib

    FGFR1-3 fusion, rearrangement, activating mutation, or FGFR2 extracellular domain in-frame deletions on …

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementAdvanced Solid Tumor, Unresectable Solid Tumor, Metastatic Solid Tumor, Cholangiocarcinoma

  9. Pemigatinib After Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia

    Recruiting Phase 1 OHSU Knight Cancer Institute Started 2021Primary completion 2026-12 NCT04659616

    cytarabinedaunorubicinpemigatinib

    Patients with known favorable risk AML (2022 European Leukemia Net \[ELN\] Guidelines) or FLT3 mutations should not be pre-screened.

    FLT3 mutation (ITD/TKD)Acute Myeloid Leukemia