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

Early Access Program in oncology: Retrospective study at a Portuguese hospital (ECOP 2024) - "During the study period were submitted 163 EAP requests for abiraterone, amivantamab, bevacizumab, durvalumab, encorafenib, enfortumab, everolimus, erdafitinib, lenvatinib, lurbinectedin, niraparib, nivolumab, olaparib, pembrolizumab, pertuzumab, ramucirumab, sacituzumab govitecan, selpercatinib, trifluridine/tipiracil, trametinib+dabrafenib, trastuzumab-deruxtecan and tucatinib. Conclusion Most cases correspond to metastatic disease, EAPs facilitate timely access to innovative therapies for patients with high unmet medical needs. The majority of situations were financed, which confirms the importance of EAPs in an era where oncology is constantly innovating."

Retrospective data • Gastrointestinal Disorder • Oncology

Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial) (clinicaltrials.gov) - P2 | N=1376 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | N=2316 ➔ 1376 | Trial completion date: Jun 2025 ➔ May 2026 | Trial primary completion date: Jun 2025 ➔ Mar 2025

Biomarker • Enrollment change • Trial completion date • Trial primary completion date • Brain Cancer • CNS Tumor • Embryonal Tumor • Ependymoma • Ewing Sarcoma • Germ Cell Tumors • Glioma • Hematological Malignancies • Hepatoblastoma • Langerhans Cell Histiocytosis • Lymphoma • Malignant Glioma • Medulloblastoma • Nephrology • Neuroblastoma • Non-Hodgkin’s Lymphoma • Oncology • Osteosarcoma • Pediatrics • Rhabdoid Tumor • Rhabdomyosarcoma • Sarcoma • Soft Tissue Sarcoma • Solid Tumor • Wilms Tumor • BRAF

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

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

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 Disorders • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • Targeted Protein Degradation • BRD4 • FGFR • MYC

A Selective MAP3K1 Inhibitor Facilitates Discovery of NPM1 as a Member of the Network. (PubMed, Molecules) - "The quinoxaline core is found in several biologically active compounds, with Erdafitinib being the first FDA-approved quinoxaline derivative that targets a kinase and exhibits anti-cancer properties...Here, we present the synthesis of a regioisomer (51-106) and its characterization as a selective MAP3K1 inhibitor with improved metabolic stability and oral bioavailability. We used the small molecule MAP3K1 inhibitor in a proteomics study that identified NPM1 as a member of the MAP3K1 network."

Journal • Oncology • MAP3K1 • NPM1

European Association of Urology Guidelines on Muscle-invasive and Metastatic Bladder Cancer: Summary of the 2025 Guidelines. (PubMed, Eur Urol) - "This overview of the 2025 EAU guidelines offers valuable insights into risk factors, diagnosis, classification, treatment, and follow-up of MIBC patients and is designed for effective integration into clinical practice."

Journal • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urology • Urothelial Cancer • FGFR3 • HER-2

Advancing Ocular Safety Profile Assessment: A Novel Grading Scale for Ocular Adverse Reactions Associated with Bemarituzumab. (PubMed, Ophthalmol Ther) - "Fibroblast growth factor receptors (FGFRs), expressed across various parts of the eye, can become unintended targets of FGFR inhibitors such as erdafitinib, infigratinib, and pemigatinib, leading to ocular adverse events (AEs) affecting the ocular surface and retina. This grading scale is being used across the clinical development program for bemarituzumab to precisely characterize the ocular safety profile, enabling cross-specialty collaboration between oncologists and eye care providers to implement appropriate management strategies. This commentary article highlights the efforts led by Amgen in collaboration with regulatory, medical, and academic fields to develop tools that facilitate early recognition of adverse reactions and appropriate interventions for patient care."

Journal • Oncology • Ophthalmology

erdafitinib (Balversa) is accepted for use within NHSScotland. (Scottish Medicines Consortium) - "Indication under review: as monotherapy for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible FGFR3 genetic alterations who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting....This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower."

Reimbursement • Urothelial Cancer

Economic evaluation of Erdafitinib as a second-line treatment for advanced metastatic urothelial carcinoma: real-world data from the USA and prospective analysis from China. (PubMed, Future Oncol) - "From the perspective of U.S. payers, Erdafitinib as a second-line treatment for mUC is not cost-effective. From the perspective of China, the cost-effectiveness of Erdafitinib is highly sensitive to its price, which could provide a reference for healthcare reimbursement negotiations."

HEOR • Journal • Real-world evidence • Oncology • Solid Tumor • Urothelial Cancer

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

Using Cancer-Associated Fibroblasts as a Shear-Wave Elastography Imaging Biomarker to Predict Anti-PD-1 Efficacy of Triple-Negative Breast Cancer. (PubMed, Int J Mol Sci) - "Based on the classification of the level of WH CAFs, while tumors with a high level of WH CAFs were found to exhibit a poor response to anti programmed cell death protein 1 (PD-1) monotherapy, they were responsive to the combination of anti-PD-1 and erdafitinib, a selective fibroblast growth factor receptor (FGFR) inhibitor. Overall, these findings establish a reference for a novel non-invasive method for predicting ICI efficacy to guide the selection of TNBC patients for precision treatment in clinical settings."

Biomarker • IO biomarker • Journal • Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • CAFs • CD8 • FGFR • VTN

Bone voyage: FGFR1, stemness, and the Wnt trail to prostate cancer metastasis (AACR 2025) - P2 | "An on-going clinical study at our department at MD Anderson is currently evaluating erdafitinib, an FGFR inhibitor, for treating men with CRPC bone metastases (ClinicalTrials.gov Identifier: NCT04754425)...Overall, our findings suggest that FGFR1 drives PCa progression through Wnt signaling-induced stemness, conferring cancer cells the plasticity to adapt to the bone microenvironment. Future studies aim to further characterize the FGFR1-induced stemness and dedifferentiation mechanisms underlying PCa bone dissemination and evaluate FGFR-targeted therapies."

Castration-Resistant Prostate Cancer • Genito-urinary Cancer • Oncology • Prostate Cancer • Solid Tumor • BMP4 • CDH2 • CORO1C • FERMT2 • FGFR1 • FZD7 • GJA1 • MEF2C • SFRP1

Inferring genomic properties from H&E whole-slide images of >60,000 solid tumors (AACR 2025) - "First, BRAF mutational status, inferred with AUROC 0.85 (precision 0.95, recall 0.63) in papillary thyroid cancer has level two evidence for dabrafenib and trametinib in metastatic differentiated thyroid cancer. Second, FGFR3 mutational status, identified with AUROC 0.92 (precision 0.95, recall 0.42) in bladder cancer indicates erdafitinib, surpassing the prior state of the art of AUROC 0.73... Taken together, our work uncovers phenome-genome relations for granular subtypes lumped together in prior studies. Such approaches advance our knowledge of how somatic mutations affect tissue morphology and have the potential to improve access to precision oncology."

Bladder Cancer • Colorectal Adenocarcinoma • Colorectal Cancer • Endocrine Cancer • Endometrial Cancer • Genito-urinary Cancer • Lung Adenocarcinoma • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • Thyroid Gland Carcinoma • Thyroid Gland Papillary Carcinoma • BRAF • FGFR3 • MSI • STK11

FGFR1 promotes endocrine resistance and regulates DNA sensing via STING modulation in hormone receptor-positive breast cancer [WITHDRAWN] (AACR 2025) - "Strikingly, FGFR1 expression negatively correlates with Type 1 IFN responses in these cells, while endocrine-resistant cells exhibit upregulated STING expression at both mRNA and protein levels.Pharmacological inhibition of FGFR1 using erdafitinib or shRNA-mediated silencing enhances the expression of interferon-stimulated genes (ISGs) by activating the STING-TBK1-IRF3 signaling axis...In vivo, the combination of FGFR1 inhibition endocrine therapy led to tumor regression in a luminal patient-derived xenograft (PDX) model, driven by an enhanced tumor-intrinsic Type 1 IFN response.These results suggest that FGFR1 acts as an innate immune checkpoint, enabling tumor cells to suppress STING mediated Type 1 IFN response and evade antitumor immunity in hormone receptor-positive breast cancer. Targeting FGFR1 may, therefore, represent a promising strategy to overcome endocrine resistance and restore immune surveillance in this context."

Breast Cancer • Estrogen Receptor Positive Breast Cancer • Hormone Receptor Breast Cancer • Hormone Receptor Positive Breast Cancer • Oncology • Solid Tumor • ER • FGFR1 • STING

Psychometric Analysis of the EORTC QLQ-NMIBC24 Using Data from a Global Phase 2 Randomized Trial of Erdafitinib[1] in Participants with High-Risk Non-Muscle-Invasive Bladder Cancer (NMIBC) (ISPOR 2025) - "This is the first investigation of the psychometric properties and MSD thresholds for the QLQ-NMIBC24 in a global sample. Results provide additional support for the reliability and validity of inferences that can be made using QLQ-NMIBC24 domain scores in adults living with NMIBC.[1] Erdafitinib (JNJ-42756493) was discovered in collaboration with Astex Pharmaceuticals"

Clinical • P2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Novel Molecular Biomarkers to Guide Treatment Decision-making in Metastatic Urothelial Cancer-A Patient Cohort Analysis. (PubMed, Eur Urol Oncol) - "The molecular makeup of tumors, including molecular subtype, tumor microenvironment composition, and gene mutations, fusions, and amplifications, has previously been correlated with a response to immune checkpoint inhibitors, erdafitinib, or enfortumab vedotin (EV) monotherapy, and may withhold potential candidate biomarkers. Stratification of patients into subgroups based on these molecular features is possible. Our data challenge the concept of a one-treatment-fits-all paradigm and support the rationale for prospective clinical trials with biomarker-guided treatment selection of mUC patients."

Biomarker • IO biomarker • Journal • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • FGFR2 • NECTIN4

SOGUG-NEOWIN: A phase 2, open-label, multicenter, multinational interventional trial evaluating the efficacy and safety of erdafitinib (ERDA) monotherapy and the combination of ERDA and cetrelimab (CET) as neoadjuvant treatment in cisplatin-ineligible patients with muscle-invasive bladder cancer (MIBC) harboring FGFR gene alterations. (ASCO 2025) - "Clinical Trial Registration Number: 2024-512573-27-01 The abstract will be released to the public on May 22, 2025 at 4:00 PM CDT"

Clinical • Monotherapy • P2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • FGFR

Development and evaluation of a non-invasive qPCR assay for sensitive detection of FGFR2/3 alterations in urine samples as a companion diagnostic for erdafitinib (AACR 2025) - "The Predicine FGFR2/3 qPCR Kit offers a rapid, robust, and non-invasive solution for detecting FGFR alterations in urine samples. By providing high sensitivity, specificity, and efficiency, it represents a promising alternative to invasive tissue-based diagnostics for identifying patients eligible for FGFR-targeted therapies such as erdafitinib. These findings highlight its potential utility as a companion diagnostic, with further clinical validation planned to support its integration into routine molecular diagnostics."

Companion diagnostic • Non-invasive • Oncology • Solid Tumor • Urothelial Cancer • BAIAP2 • BAIAP2L1 • BICC1 • CASP7 • FGFR2 • FGFR3 • TACC3

ASN-7350, a highly selective FGFR2/3 dual inhibitor, for FGFR2/FGFR3 driven solid tumors (AACR 2025) - "Alterations in fibroblast growth factor receptors (FGFRs) are recognized as oncogenic drivers in numerous human cancers. Notably, ASN-8639 was more efficacious than reference FGFR2 inhibitor in the endometrial tumor model with erdafitinib-resistant FGFR2-N549K mutation at the same dose. These findings support the further clinical development of ASN-8639 as a potential best-in-class FGFR2/3 dual inhibitor for the treatment of bladder, endometrial, and other solid tumors with FGFR2/3 aberrations."

Biliary Cancer • Cholangiocarcinoma • Endometrial Cancer • Oncology • Solid Tumor • Urothelial Cancer • FGFR1 • FGFR2 • FGFR3 • FGFR4 • TACC3

GSC000829, a novel selective FGFR2/3 inhibitor, displays superior antitumor activity in preclinical FGFR-driven neoplasms models (AACR 2025) - "It also demonstrates enhanced antitumor activity at lower dosage in FGFR3-driven CDX models compared to the pan-FGFR inhibitor Erdafitinib, as well as other FGFR3-selective inhibitors such as TYRA300 and LOXO-435. GSC000829 exhibits favorable physicochemical and pharmacokinetic properties along with an excellent preclinical toxicity profile with no obvious phosphate increase at quite high exposure level in rats. Taken together, these findings bolster the rationale for advancing GSC000829 into clinical trials as a potential best-in-class FGFR isoform-selective inhibitors, offering an approach for treating advanced solid tumors harboring FGFR2/3 aberrations."

Preclinical • Oncology • Solid Tumor • FGFR1 • FGFR2 • FGFR3 • FGFR4

FGFR3 inhibition is a highly effective therapy in MTAP-deficient, FGFR3 altered urothelial carcinoma (AACR 2025) - "In 2019, erdafitinib was the first FGFR inhibitor approved by the FDA for treatment of FGFR3-altered metastatic urothelial cancer with an objective response rate of 40%...Moreover, our data demonstrate that PRMT5 inhibition sensitizes MTAP-proficient tumors to FGFR inhibitors, suggesting that inhibition of PRMT5 activity by MTA contributes to tumor response to FGFR inhibitors in MTAP-deficient tumors. Taken together, our current findings suggest that: 1) MTAP deficiency could serve as a rational biomarker to predict response of FGFR inhibitors and; 2) FGFR inhibitors may be highly effective therapies for patients with MTAP-deficient, FGFR3 altered urothelial cancer."

Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • FGFR3 • MTAP

Erdafitinib diminishes LPS-mediated neuroinflammatory responses through NLRP3 in wild-type mice (Front Pharmacol) - "In C57BL6/N mice, erdafitinib pretreatment significantly suppressed LPS-stimulated microglial/astroglial activation and proinflammatory cytokine expression. Importantly, erdafitinib pretreatment significantly downregulated LPS-induced NLRP3 inflammasome activation and astroglial neuroinflammation-associated molecules in C57BL6/N mice."

Preclinical • Inflammation

Navigating a Pilot Program for CMS Principal Illness Navigation (PIN) Reimbursement (ONS 2025) - "The final rule included new codes and payments for Principal Illness Navigation (PIN) services, HCPCS codes G0023, G0024, G0140, and G0146, which use auxiliary personnel such as patient navigators to provide care coordination in the treatment of a serious, high-risk illness... In the first month following the implementation of this PIN billing pilot (July 2024)), we successfully submitted ten claims to CMS, marking a significant step toward utilizing the navigation services reimbursement framework effectively. Each claim was carefully tracked to ensure compliance and facilitate necessary follow-ups with the navigation team. The implications of PIN billing for nursing practice are profound, as successful reimbursement for navigation services can lead to enhanced care coordination, ultimately improving patient engagement and adherence to treatment plans."

Reimbursement • US reimbursement • Oncology

Utilization of Principal Illness Navigation Codes (ONS 2025) - "Two billing codes (G0023 and G0024) for PIN services have been accepted by the Centers for Medicare & Medicaid Services (CMS.) CMS requires that incident to services providers, including auxiliary staff, adhere to state-specific licensing criteria...Discussion The use of Principal Illness Navigation Codes remains a work in progress. The program team encountered the following challenges during implementation: staffing, concern of patient cost sharing, documentation requirements, meeting the minimum 60-minute lead time requirement using code G0023, providing organizational return on investment, cost avoidance estimation, and the manual billing process."

Oncology