Balversa (erdafitinib) / J&J, Otsuka - LARVOL DELTA

Diagnosis and Management of Upper Tract Urothelial Carcinoma: A Review. (PubMed, Cancers (Basel)) - "Enfortumab vedotin (EV) plus pembrolizumab showed superior efficacy over chemotherapy in the EV-302 trial, with improved response rate, progression-free survival, and overall survival across subgroups, including UTUC. For patients ineligible for EV, the CheckMate-901 study supported first-line chemoimmunotherapy with gemcitabine, cisplatin, and nivolumab. Further systemic therapy strategies include maintenance avelumab post-chemotherapy (JAVELIN Bladder 100), targeted therapies such as erdafitinib (THOR trial), and trastuzumab deruxtecan (DESTINY-PanTumor02) in FGFR2/3-altered and HER2-positive disease, respectively. Historically, the therapeutic landscape of UTUC has been extrapolated from bladder cancer; however, ongoing research specific to UTUC is deriving more precise regimens involving the use of immune checkpoint inhibitors, antibody-drug conjugates, and biomarker-driven therapies."

IO biomarker • Journal • Review • Bladder Cancer • Genito-urinary Cancer • Oncology • Renal Disease • Solid Tumor • Urothelial Cancer • FGFR2 • HER-2

FGFR1/3 Signaling as an Achilles' Heel of Phenotypic Diversity in Urothelial Carcinoma. (PubMed, Eur Urol Oncol) - "In vitro, erdafitinib reduced proliferation in FGFR wild-type UC cell lines in a similar manner to FGFR3-mutated cell lines. Our findings highlight FGFR1 and FGFR3 as pivotal signaling pathways with distinct, molecular subtype-specific activation mechanisms. The results suggest that FGFR inhibitors may have therapeutic applications beyond UC tumors with FGFR2/3 alterations."

Journal • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • FGFR1 • FGFR2 • FGFR3

Digital PCR analysis of FGFR3 aberrations: fast, multiplex, and reliable (ECP 2025) - "Background & Objectives: Genetic aberrations, including point mutations and fusion oncogenes of fibroblast growth factor receptor 3 (FGFR3) are known drivers of tumor initiation, progression and resistance, and established biomarkers for targeted therapies such as erdafitinib in urothelial carcinoma... Digital PCR on Absolute Q instrument offers a rapid and highly accurate technique for FGFR3 genetic aberrations detection for cancer research. The method is particularly suitable for analyzing liquid biopsy material including urine and blood as well as formalin-fixed, paraffin-embedded tissue samples, as it demonstrates high analytical sensitivity and specificity in identifying mutational variants with low allele frequencies."

Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • BAIAP2 • BAIAP2L1 • FGFR3 • TACC3

Incidence of FGFR mutations in lung carcinomas (ECP 2025) - "4/6 cases with mutations (SNVs) are classified by NCCN guidelines as emerging biomarker linked to sensitivity to Erdafitinib (FGFR3: D641G, R248C, G380R, and D641N) and for 2/6 (V550M and V550L) there are no therapeutic approval... FGFR1/2/3 gene alterations are not so infrequent (5,4%). Careful interpretation is needed as concomitant mutations are frequent, some of them in targetable genes. These cases should be discussed with clinicians especially when two targetable mutations are present knowing that some FGFR mutations are considered as emerging biomarkers."

Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • EGFR • FGFR • FGFR1 • FGFR3

Impact of Dose Reductions on the Efficacy of Erdafitinib (Erda) in Patients (Pts) with Advanced or Metastatic Urothelial Carcinoma (mUC): A Post-hoc Analysis of the Phase 3 THOR Study Cohort-1 Evaluating Erda versus Chemotherapy (Chemo) (ESMO 2025) - No abstract available

Clinical • Metastases • P3 data • Retrospective data • Oncology • Solid Tumor • Urothelial Cancer

Case Report Response to Treatment With Erdafitinib in a Patient With FGFR2 Mutation in Adenocarcinoma Lung Cancer (IASLC-WCLC 2025) - "After failing a third line with Docetaxel, next-generation sequencing (NGS) studies were conducted, revealing: FGFR2 rearrangement intron 17, FGFR2-MYH16 rearrangement. The adverse effects related to the therapy were not the reason for discontinuation; however, treatment had to be stopped due to disease progression. This report aims to provide greater insight into the response to novel treatments in patients with FGFR mutations in non-small cell lung cancer."

Case report • Clinical • Lung Adenocarcinoma • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • Urothelial Cancer • ALK • EGFR • FGFR2 • FGFR3 • KRAS • ROS1 • TP53

Analysis of FGF19 in Lung Squamous Cell Cancer Tissue and Cell Models (IASLC-WCLC 2025) - "Testing of SCC cell lines for their sensitivity against a selective FGFR4 inhibitor (BLU9931) and a pan-FGFR inhibitor (Erdafitinib) was done in 2D monolayer and in 3D spheroid culture. Conclusions : Taken together, our results suggest that FGF19 amplification is not as common in SCC of Central European patients as the 37% previously found in Chinese SCC patients. High FGF19 mRNA and protein expression in SCC cell lines is independent of FGF19 gene amplification but may not be a good predictor of FGFR inhibitor sensitivity."

Non Small Cell Lung Cancer • Oncology • FGF19 • FGFR4

IThe AIFA Board of Directors approves the reimbursability of 11 medicines… (Italian Medicines Agency) - "...New chemical molecules will be eligible for reimbursement: Balversa (erdafitinib), an anticancer medicine indicated for unresectable or metastatic urothelial carcinoma....The extensions of therapeutic indications concern six medicines already reimbursed for other indications...the anticancer medicines Tagrisso (osimertinib) and Xalkori (crizotinib)."

Reimbursement • Urothelial Cancer

Erdafitinib inhibits the tumorigenicity of MDA-MB-231 triple-negative breast cancer cells by inducing TRIM25/ubiquitin-dependent degradation of FGFR4. (PubMed, Breast Cancer Res) - "Notably, co-knockdown of FGFR1 and FGFR4 induced cytotoxicity in MDA-MB-231 cells, highlighting the therapeutic relevance of FGFR1/4 degradation by Erdafitinib in TNBC. These findings provide novel insights into the mechanisms underlying the anti-cancer efficacy of Erdafitinib, supporting its potential as a promising therapeutic agent for TNBC."

Journal • Breast Cancer • Oncology • Solid Tumor • Targeted Protein Degradation • Triple Negative Breast Cancer • FGFR1 • FGFR4

Erdafitinib inhibits the tumorigenicity of MDA-MB-231 triple-negative breast cancer cells by inducing TRIM25/ubiquitin-dependent degradation of FGFR4 (Breast Cancer Res) - "The results demonstrated that Erdafitinib suppressed TNBC tumorigenicity by promoting FGFR1/4 degradation, generating reactive oxygen species (ROS), inducing DNA damage, and ultimately triggering cell death. Mechanistic analyses revealed that Erdafitinib facilitated FGFR1/4 degradation through ubiquitination, enhanced interaction between TRIM25 and FGFR1/4, and subsequent lysosomal degradation. Furthermore, RNA-seq data from the TCGA and GEO databases, along with paired tumor tissues from TNBC patients, indicated that FGFR4 was significantly upregulated in TNBC. Notably, co-knockdown of FGFR1 and FGFR4 induced cytotoxicity in MDA-MB-231 cells, highlighting the therapeutic relevance of FGFR1/4 degradation by Erdafitinib in TNBC."

Preclinical • Triple Negative Breast Cancer

Pharmacovigilance of drug-induced cataract using the FDA Adverse Event Reporting System. (PubMed, Sci Rep) - "Disproportionality analysis identified 24 drugs significantly associated with cataract risk, with the highest risks linked to glucocorticoids (e.g., fluticasone furoate, triamcinolone), insulin analogs (e.g., insulin glargine, insulin human), and other agents like nitisinone and ranibizumab. Difluprednate showed the strongest association (Bayesian Confidence Propagation Neural Network [BCPNN] = 7.83), followed by prednisolone (BCPNN = 6.84) and erdafitinib (BCPNN = 5.44)...The majority of cases were reported in females (57.9%), with a noticeable annual increase in cases. This study provides a comprehensive pharmacovigilance evaluation, offering insights into high-risk medications, their onset patterns, and demographic trends, contributing to improved clinical decision-making and cataract prevention strategies."

Adverse events • Journal • Cataract • Oncology • Ophthalmology

Selective detection of tyrosine kinase inhibitor erdafitinib using nitrogen-doped carbon dots synthesized at room temperature. (PubMed, Anal Methods) - "Recovery rates in spiked human serum and urine ranged from 97.8% to 104.4%, validating the method's practical applicability. The relative standard deviations (RSDs) remained below 3.72%, confirming the precision and reproducibility of the NCD-based sensing platform."

Journal • Oncology • Solid Tumor • Urothelial Cancer

Data mining and safety analysis of FGFR tyrosine kinase inhibitors based on the FAERS database. (PubMed, Sci Rep) - "Fibroblast growth factor receptor tyrosine kinase inhibitors (FGFR-TKIs), including erdafitinib, pemigatinib, and futibatinib, are a promising class of therapies for FGFR-driven cancers. Subgroup analysis indicated an increased risk of death in futibatinib-treated patients aged < 65 years. These findings offer critical insights beyond clinical trials, informing oncologists of emerging safety concerns associated with FGFR-TKIs in real-world settings."

Journal • Gastroenterology • Gastrointestinal Disorder • Oncology • FGFR

Phase Ib Trial of Fulvestrant, Palbociclib and Erdafitinib, a pan-FGFR Tyrosine Kinase Inhibitor, in HR+/HER2- Metastatic Breast Cancer. (PubMed, Clin Cancer Res) - P1 | "The trial endpoint was met establishing the MTD of erdafitinib at 6 mg. While the triplet regimen may pose tolerability challenges, alterative doublets with selective FGFR1 inhibitors in patients with FGFR1 dependent tumors, possibly administered in sequence, are worthy of further investigation."

Journal • P1 data • Breast Cancer • Hematological Disorders • HER2 Breast Cancer • HER2 Negative Breast Cancer • HER2 Positive Breast Cancer • Metabolic Disorders • Mucositis • Nephrology • Neutropenia • Oncology • Renal Disease • Solid Tumor • Stomatitis • FGFR • HER-2

Targeting Drug Resistance in Hepatocellular Carcinoma: Uncovering Metabolic Vulnerabilities for Therapeutic Re-Sensitization (EACR 2025) - "Dose-response experiments in the presence of key growth factors were performed to determine sensitivities to five kinase inhibitors: Lenvatinib, Sorafenib, Regorafenib, Roblitinib, and Erdafitinib, the latter two being fibroblast growth factor receptor inhibitors currently in clinical trials. This study aims to provide a framework for targeting metabolic vulnerabilities in HCC, offering potential new therapies to enhance treatment efficacy and to break or delay drug resistance. By integrating physiologically relevant culture systems and metabolic reprogramming strategies, future findings could contribute to the development of more effective interventions for overcoming therapeutic resistance in HCC."

Hepatitis B • Hepatitis C • Hepatocellular Cancer • Hepatology • Infectious Disease • Liver Cancer • Metabolic Disorders • Metabolic Dysfunction-Associated Steatotic Liver Disease • Oncology • Solid Tumor • FGFR

FGF/FGFR System in Medulloblastoma: New Approaches for Tumor Treatment. (EACR 2025) - "The inhibition of FGF/FGFR was carried out using a clinical grade FGFR inhibitor (Erdafitinib) and a FGF trap small molecule (UPR1430)... In conclusion, our preliminary data suggest that the FGF/FGFR system plays a role and might represent a promising target for the treatment of MB-Gr3."

Brain Cancer • CNS Disorders • Medulloblastoma • Oncology • Pediatrics • Solid Tumor • Ventriculomegaly • FGFR • MYC

LC-MF-4, a Novel FGFR3 Degrader for Therapeutic Intervention in FGFR3-Altered Cancers. (PubMed, J Med Chem) - "Erdafitinib was approved for the treatment of UC but is limited by the progression of on-target gatekeeper resistance mutations...In FGFR3-TACC3 fusion-positive cells, LC-MF-4 exerted its effects by suppressing the expression of genes involved in mitochondrial biogenesis and ATP synthesis. This study demonstrated robust antitumor activity of LC-MF-4 in the Ba/F3-FGFR3-TACC3 xenograft model, highlighting its potential for the treatment of FGFR3-altered cancers."

Journal • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • FGFR1 • FGFR2 • FGFR3 • TACC3

Leveraging Small Voxel with Optimal Acquisition Time for [18F]mFBG Total-Body PET/CT imaging in Pediatric Patients with Neuroblastoma: A Preliminary Study (SNMMI 2025) - "Of the 33 patients with 37 [18F]mFBG PET/CT scans, 17 patients with 20 scans had positive [18F]mFBG PET/CT findings. Sufficient subjective image quality was achieved with at least 2-min acquisition of 192×192 matrix and 4-min acquisition of 512×512 matrix (with all scores ≥ 3). SNR increased with longer acquisition times for the same voxel size, while decreased as voxel size shrunk."

Clinical • Neuroblastoma • Oncology • Pediatrics • Solid Tumor

Plain language summary of the THOR Cohort 1 study comparing treatment with erdafitinib or chemotherapy in advanced or metastatic urothelial cancer. (PubMed, Future Oncol) - No abstract available

Journal • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

Erdafitinib diminishes LPS-mediated neuroinflammatory responses through NLRP3 in wild-type mice. (PubMed, Front Pharmacol) - "Importantly, erdafitinib pretreatment significantly downregulated LPS-induced NLRP3 inflammasome activation and astroglial neuroinflammation-associated molecules in C57BL6/N mice. Collectively, our experiments demonstrate that erdafitinib pretreatment diminishes LPS-induced neuroinflammation by suppressing NLRP3 inflammasome activation in vitro and in vivo and suggest that erdafitinib is a potential therapeutic agent for neuroinflammation-related diseases."

Journal • Preclinical • Genito-urinary Cancer • Inflammation • Oncology • Solid Tumor • Urothelial Cancer • NLRP3

FGF SIGNALING BLOCKADE HAMPERS THE GROWTH OF C-MYC-DRIVEN AGGRESSIVE B-CELL NON-HODGKIN LYMPHOMA (EHA 2025) - "This was confirmed by co-treatment with the proteosome inhibitor MG132 that prevented c-MYC degradation. Altogether, these findings demonstrate that the FGF/FGFR system sustains the growth and survival of DLBCL and BL cells and open new therapeutic hints for the treatment of c-MYC-driven aggressive B-NHL. Indeed, these data may provide a clinical rationale to start phase 1/2 clinical trials with FDA-approved FGFR TK inhibitors (e.g. Erdafitinib, Pemigatinib, Futinatinib) as single agents or in combination with standard R-CHOP regimens."

B Cell Lymphoma • B Cell Non-Hodgkin Lymphoma • Burkitt Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • ANXA5 • FGF • MYC

C-MYC ONCOPROTEIN IS STABILIZED BY FGFR ACTIVATION IN C-MYC-DRIVEN B-CELL NON-HODGKIN LYMPHOMA (EHA 2025) - "pFGFR and c-Myc levels were progressively reduced by increasing the doses of NSC12 and Erdafitinib and, importantly, the reduction of c-Myc strictly followed the inhibition of FGFR phosphorylation, suggesting a strong correlation between FGFR activation and c-Myc protein expression...Accordingly, co-treatment with the proteosome inhibitor MG132 was able to prevent the degradation of c-Myc induced by FGF/FGFR blockade... These data highlight the presence of a FGF/FGFR/c-Myc axis in c-Myc-driven aggressive B-NHL and indicate that the FGF/FGFR system plays an important role in the biology of these hematologic malignancies by acting as a pivotal regulator of c-Myc protein stability. These findings may provide the rationale for the use of FGF/FGFR inhibitors in c-Myc-driven aggressive B-NHL."

B Cell Lymphoma • B Cell Non-Hodgkin Lymphoma • Burkitt Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • Targeted Protein Degradation • BRD4 • FGFR • MYC

Barriers and challenges for precision medicine and evidence-based oncology in genitourinary tumors in Latin America: insights from the LACOG 1024 survey CIELO. (ASCO 2025) - "Regarding access to therapies, 48.1% of the participants had access to adjuvant pembrolizumab for renal cell carcinoma, 42.7% to olaparib monotherapy for PC, 41.2% to enzalutamide for non-metastatic castration-sensitive PC, 38.2% to adjuvant nivolumab for urothelial carcinoma, and 31.3% to pembrolizumab for non-muscle invasive bladder cancer. Conversely, treatments such as first-line enfortumab vedotin with pembrolizumab (18.3%), erdafitinib (18.3%), Lu 177 vipivotide tetraxetan (9.9%), relugolix (6.9%), and belzutifan (1.5%) faced significant access barriers... To the best of our knowledge, this is the largest survey conducted on this topic to date in LATAM. The results underscore substantial barriers to the adoption of precision medicine, driven predominantly by limited insurance coverage and financial constraints. Enhancing access to advanced diagnostics and therapies is crucial for bringing regional practices in line with global standards and for improving patient..."

Bladder Cancer • Genito-urinary Cancer • Oncology • Prostate Cancer • Renal Cell Carcinoma • Solid Tumor • Urothelial Cancer

Molecular profiling in targeted therapy of gliomas in a community setting. (ASCO 2025) - "Accordingly, 11 patients received targeted therapy: 3 patients olaparib (RAD51C, BRCA2, H3.3 G34), 2 pembrolizumab (TMB high), 1 ivosidenib (IDH1), 1 selumetinib (NF1), 1 alpelisib (PIK3CA), 1 erdafitinib (FGFR3), 1 trametinib (NF1) and 1 dabrafenib/trametinib (BRAF) and then everolimus (TSC2). Molecular profiling nowadays is not only mandatory in glioma classification but can also provide additional therapies in patients with limited options."

IO biomarker • Tumor mutational burden • Anaplastic Astrocytoma • Astrocytoma • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • High Grade Glioma • Oligodendroglioma • Oncology • Solid Tumor • ATRX • BRAF • BRCA2 • EGFR • FGFR3 • IDH1 • MSI • NF1 • PD-L1 • PIK3CA • PTEN • RAD51C • TERT • TMB • TP53 • TSC2

SPRING: Study of Precision Treatment for Rare Tumours in China Guided by PDO and NGS (clinicaltrials.gov) - P2 | N=200 | Not yet recruiting | Sponsor: Peking University Shenzhen Hospital

Biomarker • New P2 trial • Oncology