^
    2d
    Management of Nail Toxicities From Fibroblast Growth Factor Receptor Inhibitors. (PubMed, J Drugs Dermatol)
    The incidence of FGFRi-associated nail toxicities varies by agent and can affect quality of life and treatment adherence. The pathogenesis remains unclear, and no predictive biomarkers exist. Further research into optimized management and preventative strategies is needed. Early recognition and proactive multidisciplinary management are essential to minimizing complications and maintaining oncologic treatment continuity. &nbsp.
    Journal
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    FGFR (Fibroblast Growth Factor Receptor)
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    Balversa (erdafitinib) • Lytgobi (futibatinib) • Pemazyre (pemigatinib) • derazantinib (ARQ 087)
    2d
    Myeloid/Lymphoid Neoplasms with FGFR1 Rearrangement and Pemigatinib. (PubMed, Blood)
    Outside allogeneic stem cell transplantation (ASCT), survival with conventional therapy is dismal, representing an unmet clinical need. We summarize here the data that led to approval of pemigatinib, a FGFR1 inhibitor, showing unprecedented efficacy in M/LN-FGFR1.
    Journal
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    FGFR1 (Fibroblast growth factor receptor 1)
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    Pemazyre (pemigatinib)
    7d
    PEMIGIST: PH 2 Pemigatinib in SDH-deficient GIST (clinicaltrials.gov)
    P2, N=24, Not yet recruiting, Dana-Farber Cancer Institute
    New P2 trial
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    Pemazyre (pemigatinib)
    22d
    FGFR2-Rearranged Biliary Tract Cancer: Biology, Resistance Mechanisms, and Emerging Therapeutic Strategies. (PubMed, Cancers (Basel))
    Selective FGFR tyrosine kinase inhibitors, including the reversible inhibitor pemigatinib and the irreversible inhibitor futibatinib, have demonstrated clinically meaningful response rates and durable disease control in patients with previously treated FGFR2-altered iCCA, leading to regulatory approvals and the incorporation of FGFR inhibition into contemporary treatment paradigms. In addition, we highlight the growing role of circulating tumor DNA as a noninvasive tool for longitudinal molecular monitoring and treatment guidance. Together, these insights underscore the central role of FGFR2-directed therapy in precision oncology for biliary tract cancer and provide a framework for optimizing and extending targeted treatment in this molecularly defined disease subset.
    Review • Journal
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    FGFR2 (Fibroblast growth factor receptor 2)
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    FGFR2 mutation • FGFR2 fusion • FGFR2 rearrangement
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    Lytgobi (futibatinib) • Pemazyre (pemigatinib)
    22d
    Phase I results of a multicenter, open-label, dose de-escalation and expansion study of gemcitabine and cisplatin with ivosidenib or pemigatinib for advanced cholangiocarcinoma. (PubMed, Invest New Drugs)
    Adding ivosidenib to gemcitabine and cisplatin in this study demonstrated a challenging safety profile in advanced CCA. Further research and dose optimization are warranted to confirm these findings and optimize the integration of targeted therapies into first-line regimens.
    P1 data • Journal • IO biomarker
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    FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)
    |
    IDH1 mutation • FGFR2 mutation • FGFR2 fusion
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    cisplatin • gemcitabine • Pemazyre (pemigatinib) • Tibsovo (ivosidenib)
    1m
    Acquired FGFR2 mutation leads resistance to pemigatinib in a patient with FGFR2-driven Borrmann type IV gastric cancer. (PubMed, Anticancer Drugs)
    Subsequent administration of the irreversible FGFR1-4 inhibitor futibatinib was associated with a declining trend in tumor biomarkers, indicating preliminary antitumor activity against the resistant clone. This case underscores the clinical activity of FGFR inhibition in FGFR2-altered SGC and exemplifies the emergence of kinase domain mutations as a principal resistance pathway. It further suggests that irreversible FGFR inhibitors may represent a rational therapeutic strategy upon progression on prior FGFR-directed therapy, warranting further clinical investigation in this molecularly defined patient subset.
    Journal
    |
    FGFR2 (Fibroblast growth factor receptor 2)
    |
    FGFR2 mutation • FGFR2 rearrangement
    |
    Lytgobi (futibatinib) • Pemazyre (pemigatinib)
    2ms
    FGFR Aberrations in Solid Tumors: Mechanistic Insights and Clinical Translation of Targeted Therapies. (PubMed, Cancers (Basel))
    Recent development of selective FGFR inhibitors-such as pemigatinib, erdafitinib, and futibatinib-has translated mechanistic insights into measurable clinical benefits in genomically defined patient populations. This review also highlights emerging therapeutic modalities, such as antibody-drug conjugates and nanotechnology-based delivery systems, which may improve target specificity and prolong therapeutic durability. By integrating molecular, translational, and clinical evidence, this review aims to establish a comprehensive framework for precision oncology strategies targeting FGFR-driven malignancies.
    Review • Journal
    |
    FGFR (Fibroblast Growth Factor Receptor)
    |
    Balversa (erdafitinib) • Lytgobi (futibatinib) • Pemazyre (pemigatinib)
    2ms
    FGF1-FGFR2 axis regulated by nuclear receptor RORγ represents an effective strategy in intrahepatic cholangiocarcinoma. (PubMed, Cell Death Discov)
    Genetic silencing and pharmacological inhibition of RORγ (GSK805/XY101) suppressed proliferation, induced apoptosis in vitro, and significantly reduced xenograft tumor growth in vivo. Notably, RORγ antagonists synergized with pemigatinib to overcome resistance in pemigatinib-refractory models. Collectively, these findings identify the RORγ-FGF1-FGFR2 axis as a critical oncogenic driver in iCCA and highlight RORγ inhibition as a promising therapeutic strategy to suppress tumor progression and enhance sensitivity to FGFR inhibitors.
    Journal
    |
    FGFR2 (Fibroblast growth factor receptor 2) • FGF1 (Fibroblast Growth Factor 1)
    |
    Pemazyre (pemigatinib)
    3ms
    Pemigatinib After Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia (clinicaltrials.gov)
    P1, N=32, Recruiting, OHSU Knight Cancer Institute | Trial completion date: Jun 2026 --> Dec 2026 | Trial primary completion date: Dec 2025 --> Dec 2026
    Trial completion date • Trial primary completion date
    |
    TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • EZH2 (Enhancer of zeste 2 polycomb repressive complex 2 subunit) • RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • KMT2A (Lysine Methyltransferase 2A) • SRSF2 (Serine and arginine rich splicing factor 2) • BCOR (BCL6 Corepressor) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • STAG2 (Stromal Antigen 2) • MECOM (MDS1 And EVI1 Complex Locus) • NUP214 (Nucleoporin 214) • GATA2 (GATA Binding Protein 2) • MLLT3 (MLLT3 Super Elongation Complex Subunit) • CDKN1A (Cyclin-dependent kinase inhibitor 1A) • DEK (DEK Proto-Oncogene) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
    |
    TP53 mutation • FLT3 mutation • RUNX1 mutation • ASXL1 mutation • EZH2 mutation • SRSF2 mutation • Chr del(5q)
    |
    cytarabine • Pemazyre (pemigatinib) • daunorubicin • Starasid (cytarabine ocfosfate)
    3ms
    Saturation mutagenesis identifies activating and resistance-inducing FGFR kinase domain mutations. (PubMed, Nat Genet)
    Pooled positive selection screens identified 474 activating and 738 mutations mediating resistance to the FGFR inhibitors pemigatinib and futibatinib, together revealing 301 druggable FGFR mutations analogous to a strong PS3/BS3 evidence level. The functional screens identified 97% of acquired resistance mutations in clinical trials. Our comprehensive catalog of every druggable mutation in the FGFR kinase domains is readily available for clinical decision support.
    Journal
    |
    FGFR2 (Fibroblast growth factor receptor 2) • FGFR3 (Fibroblast growth factor receptor 3) • FGFR1 (Fibroblast growth factor receptor 1) • FGFR (Fibroblast Growth Factor Receptor) • FGFR4 (Fibroblast growth factor receptor 4)
    |
    FGFR2 mutation • FGFR mutation
    |
    Lytgobi (futibatinib) • Pemazyre (pemigatinib)
    3ms
    Tinengotinib for adults with advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 trial. (PubMed, Lancet Gastroenterol Hepatol)
    These findings suggest that tinengotinib might have activity in patients with cholangiocarcinoma with FGFR2 fusions that progressed following FGFR inhibitor therapy. Anti-tumour activity was also observed in patients with other FGFR alterations. The data from this phase 2 study supported the initiation of a phase 3 registration trial.
    P2 data • Journal
    |
    FGFR2 (Fibroblast growth factor receptor 2)
    |
    FGFR2 mutation • FGFR2 fusion
    |
    Lytgobi (futibatinib) • Pemazyre (pemigatinib) • tinengotinib (TT-00420)
    4ms
    Expanding Horizons in Cholangiocarcinoma: Emerging Targets Beyond FGFR2 and IDH1. (PubMed, Int J Mol Sci)
    Advanced CCA remains largely inoperable, and combination gemcitabine plus cisplatin (GemCis) chemotherapy remains the standard treatment for patients affected by this disease...The FDA has approved the targeted therapies ivosidenib, pemigatinib, infigratinib, and futibatinib, as well as the immunotherapy durvalumab, for patients with CCA in recent years...While several promising advancements have been made, further research is required to improve outcomes for patients with CCA. This review provides an up-to-date, comprehensive overview of currently approved targeted therapies in CCA, as well as those under investigation.
    Review • Journal
    |
    HER-2 (Human epidermal growth factor receptor 2) • KRAS (KRAS proto-oncogene GTPase) • BRAF (B-raf proto-oncogene) • ALK (Anaplastic lymphoma kinase) • FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)
    |
    cisplatin • Imfinzi (durvalumab) • gemcitabine • Truseltiq (infigratinib) • Lytgobi (futibatinib) • Pemazyre (pemigatinib) • Tibsovo (ivosidenib)