FGFR1 antagonists(Fibroblast growth factor receptor 1 antagonists), FGFR2 antagonists(Fibroblast growth factor receptor 2 antagonists), FGFR3 antagonists(Fibroblast growth factor receptor 3 antagonists)

Therapeutic Areas

NeoplasmsUrogenital DiseasesImmune System Diseases+ [4]

Active Indication

Locally Advanced Urothelial CarcinomaMetastatic urothelial carcinomaUnresectable Urothelial Carcinoma+ [21]

Inactive Indication

Adenocarcinoma of prostateCastration-Resistant Prostatic CancerMetastatic Prostate Carcinoma

Originator Organization

Astex Pharmaceuticals, Inc.

Active Organization

Janssen Research & Development LLC

Xian-Janssen Pharmaceutical Ltd.

Shanghai Johnson & Johnson Pharmaceutical Ltd.

+ [9]

Inactive Organization

Johnson & Johnson Pharmaceutical Research & Development LLC

Drug Highest PhaseApproved

First Approval Date

United States (12 Apr 2019),

FGFR positive Transitional Cell Carcinoma

RegulationAccelerated Approval (United States), Orphan Drug (South Korea), Breakthrough Therapy (United States)

Structure/Sequence

Molecular FormulaC25H30N6O2

InChIKeyOLAHOMJCDNXHFI-UHFFFAOYSA-N

CAS Registry1346242-81-6

Clinical Trials associated with Erdafitinib

NCT06919965

/ Not yet recruitingPhase 3

A Phase 3, Randomized, Open-label, Multicenter Study Evaluating the Efficacy and Safety of TAR-210 Erdafitinib Intravesical Delivery System Versus Investigator's Choice of Intravesical Chemotherapy in Participants With High-risk Non-muscle-invasive Bladder Cancer With Susceptible FGFR Alterations Who Had Received Intravesical Bacillus Calmette-Guérin (BCG)

The main purpose of this study is to compare the disease-free survival (the length of time after randomization that a participant survives without any signs or symptoms of the cancer returning, or progressing) between Bacillus Calmette-Guérin (BCG) treated participants receiving treatment with TAR-210 versus investigator's choice of intravesical chemotherapy for treatment of high-risk non-muscle-invasive bladder cancer (HR-NMIBC).

Start Date25 Aug 2025

Sponsor / Collaborator

Janssen Research & Development LLC

CTR20242467

/ RecruitingPhase 3

一项在携带易感型FGFR变异的中危非肌层浸润性膀胱癌（IR-NMIBC）受试者中评估TAR-210（Erdafitinib膀胱内给药系统）相比膀胱内单药化疗灌注的疗效和安全性的III期、随机化研究

[Translation] A Phase III, randomized study evaluating the efficacy and safety of TAR-210 (erdafitinib intravesical delivery system) compared with intravesical single-agent chemotherapy in subjects with intermediate-risk non-muscle invasive bladder cancer (IR-NMIBC) harboring susceptible FGFR alterations

本研究的主要目的是比较接受TAR-210的受试者与接受研究者选择的膀胱化疗灌注的受试者的DFS。

[Translation]

The primary objective of this study was to compare DFS in subjects who received TAR-210 with those who received investigator's choice of intravesical chemotherapy.

Start Date25 Jul 2024

Sponsor / Collaborator

Johnson & Johnson (China) Investment Ltd.

[+1]

CTIS2023-507684-19-00

/ RecruitingPhase 3

Phase 3, Randomized Study Evaluating the Efficacy and Safety of TAR-210 Erdafitinib Intravesical Delivery System Versus Single Agent Intravesical Chemotherapy in Participants With Intermediate-risk Non-muscle Invasive Bladder Cancer (IR-NMIBC) and Susceptible FGFR Alterations - 42756493BLC3004

Start Date24 Jul 2024

Sponsor / Collaborator-

100 Clinical Results associated with Erdafitinib

100 Translational Medicine associated with Erdafitinib

100 Patents (Medical) associated with Erdafitinib

448

Literatures (Medical) associated with Erdafitinib

01 May 2025·EUROPEAN UROLOGY

European Association of Urology Guidelines on Muscle-invasive and Metastatic Bladder Cancer: Summary of the 2025 Guidelines

Review

Author: Bruins, Harman Max ; Smith, Emma J ; Sæbjørnsen, Sæbjørn ; Martini, Alberto ; Mariappan, Param ; Cathomas, Richard ; van der Heijden, Antoine G ; Dimitropoulos, Konstantinos ; Neuzillet, Yann ; Panebianco, Valeria ; Efstathiou, Jason A ; Thalmann, George N ; Fietkau, Rainer ; Lorch, Anja ; Mertens, Laura S ; Kailavasan, Mithun ; Compérat, Eva ; Meijer, Richard P ; Carrion, Albert ; Milowsky, Matthew I ; Rink, Michael

BACKGROUND AND OBJECTIVE:

This publication represents a summary of the updated 2025 European Association of Urology (EAU) guidelines for muscle-invasive and metastatic bladder cancer (MMIBC). The aim is to provide practical recommendations on the clinical management of MMIBC with a focus on diagnosis, treatment, and follow-up.

METHODS:

For the 2025 guidelines, new and relevant evidence was identified, collated, and appraised via a structured assessment of the literature. Databases searched included Medline, EMBASE, and the Cochrane Libraries. Recommendations within the guidelines were developed by the panel to prioritise clinically important care decisions. The strength of each recommendation was determined according to a balance between desirable and undesirable consequences of alternative management strategies, the quality of the evidence (including the certainty of estimates), and the nature and variability of patient values and preferences.

KEY FINDINGS AND LIMITATIONS:

The key recommendations emphasise the importance of thorough diagnosis, treatment, and follow-up for patients with MMIBC. The guidelines stress the importance of a multidisciplinary approach to the treatment of MMIBC patients and the importance of shared decision-making with patients. The key changes in the 2025 muscle-invasive bladder cancer (MIBC) guidelines include the following: a new recommendation for the use of susceptible FGFR3 alterations to select patients with unresectable or metastatic urothelial carcinoma for treatment with erdafitinib; significant adaption and update of the recommendations for pre- and postoperative radiotherapy and sexual organ-preserving techniques in women; new recommendation related to radical cystectomy and extent of lymph node dissection based on the results of the SWOG trial; recommendation related to hospital volume; new recommendations for salvage cystectomy after trimodality therapy and for the management of all patients who are candidates for trimodality bladder-preserving treatment in a multidisciplinary team setting using a shared decision-making process; significant adaption and update to the recommendation for adjuvant nivolumab in selected patients with pT3/4 and/or pN+ disease not eligible for, or who declined, adjuvant cisplatin-based chemotherapy; and addition of a new recommendation for metastatic disease regarding the antibody-drug conjugate trastuzumab deruxtecan in case of HER2 overexpression; in addition, removal of the recommendations on sacituzumab govitecan as the manufacturer has withdrawn the US Food and Drug Administration approval for this product; update of the follow-up of MIBC; and full update of the management algorithms of MIBC.

CONCLUSIONS AND CLINICAL IMPLICATIONS:

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.

01 Apr 2025·CANCER TREATMENT REVIEWS

Post-progression treatment options after CDK4/6 inhibitors in hormone receptor-positive, HER2-negative metastatic breast cancer

Review

Author: Sahin, Taha Koray ; Aksoy, Sercan ; Guven, Deniz Can ; Rizzo, Alessandro

The combination of cyclin-dependent kinase 4/6 inhibitors (CDK4/6i) and endocrine therapy (ET) is the standard first-line treatment for hormone receptor-positive (HR + ) and HER2-negative metastatic breast cancer (mBC). Despite their efficacy, resistance inevitably develops, necessitating alternative therapeutic strategies post-progression. This review explores current and emerging treatment options following progression on CDK4/6i, focusing on endocrine therapies, targeted therapies, combination approaches, and the continued use of CDK4/6i. Endocrine therapies, including fulvestrant and novel oral selective estrogen receptor degraders (SERDs) like elacestrant, show promise, especially in patients with ESR1 mutations. Targeted therapies such as PI3K/AKT/mTOR inhibitors, exemplified by alpelisib and capivasertib, offer potential by addressing downstream signaling pathways involved in resistance. Additionally, FGFR inhibitors like erdafitinib are under investigation for their role in overcoming specific resistance mechanisms. Combination strategies involving CDK4/6 inhibitors with immune checkpoint inhibitors or other targeted agents are also being explored, with early trials suggesting possible synergistic effects, although further validation is required. Continuation of CDK4/6 inhibitors beyond progression has shown potential benefits in selected patients, but the data are heterogeneous, and further studies are needed to clarify their role. While chemotherapy remains a standard option for patients who progress on these treatments, the goal is to delay its use through the effective utilization of endocrine and targeted therapies. Understanding resistance mechanisms and tailoring treatment to individual patient profiles is crucial for optimizing outcomes. Ongoing clinical trials are expected to provide deeper insights, guiding the development of more effective post-progression therapeutic strategies. This evolving landscape highlights the need for continuous research and individualized patient care to improve survival and quality of life in HR + mBC patients.

01 Apr 2025·ANNALS OF ONCOLOGY

The unjustified fear of erdafitinib induced central serous retinopathy

Letter

Author: Peeters, Freya ; Vulsteke, Christof ; Strijbos, Michiel ; Schauwvlieghe, Pieter-Paul

News (Medical) associated with Erdafitinib

21 Apr 2025

·www.prnewswire.com

Johnson & Johnson unveils highly anticipated and potential practice-changing data in bladder cancer treatment at AUA

TAR-200 monotherapy shows highest complete response with sustained benefits in 12-month data from Phase 2b SunRISe-1 study (Cohort 2) Compelling first results from Cohort 4 of Phase 2b SunRISe-1 study show potential of TAR-200 monotherapy in patients with papillary-only, high-risk non-muscle invasive bladder cancer RARITAN, N.J., April 21, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) announced today that new data from its leading oncology pipeline will be presented at the American Urological Association (AUA) 2025 Annual Meeting, taking place April 26-29 in Las Vegas. Among the highlights are the 12-month duration of response (DOR) data from the Phase 2b Cohort 2 SunRISe-1 study, evaluating TAR-200—an intravesical gemcitabine releasing system—for patients with Bacillus Calmette-Guérin (BCG)—unresponsive, high-risk non-muscle-invasive bladder cancer (HR-NMIBC) with carcinoma in situ (CIS) with or without papillary disease. These findings will be featured in the Practice-changing, Paradigm-shifting Clinical Trials in Urology plenary session on Saturday, April 26. Bladder cancer ranks among the top ten most common cancers worldwide, affecting nearly a million people each year.1 Despite advancements, standard treatment has remained largely unchanged for over 40 years, leaving patients with limited treatment options if initial BCG therapy does not work.2 TAR-200 delivers sustained medication directly into the bladder and, in a pre-clinical setting, has been shown to allow for depth of penetration across bladder tissue layers.3 "Patients with bladder cancer need more effective treatment options that are both tolerable and easily incorporated into everyday practice, especially for those with HR-NMIBC, a highly recurrent disease that often necessitates difficult, life-altering decisions like bladder removal," said Yusri Elsayed, M.D., M.H.Sc., Ph.D., Global Therapeutic Area Head, Oncology, Johnson & Johnson Innovative Medicine. "TAR-200 provides a new approach, with clinical data showing an impressive complete response rate, meaning the cancer was undetectable following treatment. The highly anticipated 12-month duration of response findings from our Cohort 2, SunRISe-1 study further support the potential for patients to remain cancer-free for a clinically meaningful period." A second plenary presentation will feature first results from Cohort 4 of the Phase 2b SunRISe-1 study evaluating TAR-200 monotherapy in patients with BCG–unresponsive, papillary-only HR-NMIBC. In this patient population, bladder removal remains a standard treatment, but many patients are elderly, have significant comorbidities, or are unwilling to undergo radical surgery, making treatment challenging.4 "Patients deserve more than the currently available treatment options. TAR-200 is a groundbreaking therapy for early-stage bladder cancer, designed to deliver a sustained local release of medication directly into the bladder—right where it is needed," said Biljana Naumovic, U.S. President, Oncology, Solid Tumor, Johnson & Johnson Innovative Medicine. "This innovation provides a bladder-sparing treatment option that can meaningfully improve outcomes while integrating seamlessly into any urology practice." TAR-200 is inserted directly into the bladder by a healthcare professional in a brief outpatient, in-office procedure, without the need for anesthesia. Designed to remain in the bladder, it does not interfere with daily activities and provides sustained release of medication throughout the day. To date, TAR-200 has been placed more than 10,000 times as part of the SunRISe clinical program. AUA 2025 Presentation Highlights: One-year duration of response data from the Phase 2b SunRISe-1 study evaluating TAR-200 monotherapy in patients with BCG–unresponsive, HR-NMIBC plus carcinoma in situ with or without papillary disease (P2 Plenary Presentation). First results from Cohort 4 of the Phase 2b SunRISe-1 study evaluating TAR-200 monotherapy in patients with BCG–unresponsive papillary-only HR-NMIBC (P2 Plenary Presentation). Trial-in-progress mini-oral presentation from the Phase 3 MoonRISe-1 study evaluating TAR-210, an erdafitinib intravesical drug-releasing system, versus intravesical chemotherapy in patients with fibroblast growth factor receptors (FGFR)-altered intermediate-risk NMIBC (Clinical Trials in Progress Presentation). Trial-in-progress presentation from the Phase 3 SunRISe-5 study evaluating TAR-200 compared to intravesical chemotherapy after treatment with BCG in patients with recurrent HR-NMIBC (Clinical Trials in Progress Presentation). Real-world time-to-next-treatment and time-to-castration-resistance among patients with metastatic castration-sensitive prostate cancer using androgen-receptor pathway inhibitors with and without homologous recombination repair alterations (Oral Presentation #25-3830). A complete list of Johnson & Johnson's sponsored abstracts is available on JNJ.com. About TAR-200 TAR-200 is an investigational intravesical gemcitabine releasing system. In January 2025, Johnson & Johnson announced the initiation of a new drug application with the FDA for TAR-200 under the real-time oncology review (RTOR) program. In December 2023, the FDA granted Breakthrough Therapy Designation (BTD) to TAR-200 for the treatment of adult patients with BCG—unresponsive HR-NMIBC with CIS who are ineligible for or have elected not to undergo radical cystectomy. The safety and efficacy of TAR-200 are being evaluated in Phase 2 and Phase 3 studies in patients with MIBC in SunRISe-4, and NMIBC in SunRISe-1, SunRISe-3 and SunRISe-5. About TAR-210 TAR-210 is an investigational intravesical erdafitinib releasing system. The safety and efficacy of TAR-210 is being evaluated in a Phase 1 study (NCT05316155) in patients with muscle-invasive bladder cancer (MIBC) and NMIBC. About High-Risk Non-Muscle-Invasive Bladder Cancer High-risk non-muscle-invasive bladder cancer is a type of non-invasive bladder cancer that is more likely to recur or spread beyond the lining of the bladder, called the urothelium, and progress to invasive bladder cancer compared to low-risk NMIBC.5,6 HR-NMIBC makes up 15-44 percent of patients with NMIBC and is characterized by a high-grade, large tumor size, presence of multiple tumors, with or without CIS.7 Radical cystectomy is currently recommended for NMIBC patients who fail BCG therapy, with over 90 percent cancer-specific survival if performed before muscle-invasive progression.8,9 Given that NMIBC typically affects older patients, many may be unwilling or unfit to undergo radical cystectomy.10 The high rates of recurrence and progression can pose significant morbidity and distress for these patients.3,6 About Prostate Cancer Approximately 300,000 people are diagnosed with prostate cancer each year in the U.S.11 Up to 40 percent of patients will be classified as high-risk.12 Despite advancements in treatment, disease recurrence remains substantial; up to 50 percent of patients within ten years of surgery experience recurrence and carry a significant risk of disease progression and death.13 About Johnson & Johnson At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity. Learn more at or at . Follow us at @JNJInnovMed. Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC and Janssen Scientific Affairs, LLC are Johnson & Johnson companies. Cautions Concerning Forward-Looking Statements This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of TAR-200, TAR-210 or BALVERSA® (erdafitinib). The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC, Janssen Scientific Affairs, LLC and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at , or on request from Johnson & Johnson. None of Janssen Research & Development, LLC, Janssen Biotech, Inc., Janssen Global Services, LLC, Janssen Scientific Affairs, LLC nor Johnson & Johnson undertake to update any forward-looking statement as a result of new information or future events or developments. 1 2 Dobruch J, Oszczudłowski M. Bladder Cancer: Current Challenges and Future Directions. Medicina (Kaunas). 2021;57(8):749. Published 2021 Jul 24. doi:10.3390/medicina57080749 3 Pradère B., et al. PENELOPE: Tissue penetration of gemcitabine phosphate metabolites following TAR-200 administration versus standard intravesical instillation in minipigs. EAU 2025. March 23, 2025. 4 Lebacle C, Loriot Y, Irani J. BCG-unresponsive high-grade non-muscle invasive bladder cancer: what does the practicing urologist need to know?. World J Urol. 2021;39(11):4037-4046. doi:10.1007/s00345-021-03666-w 5 Grab-Heyne K, Henne C, Mariappan P, et al. Intermediate and high-risk non–muscle-invasive bladder cancer: an overview of epidemiology, burden, and unmet needs. Front Oncol. 2023;13:1170124. 6 Lieblich A, Henne C, Mariappan P, Geiges G, Pöhlmann J, Pollock RF. The management of non–muscle-invasive bladder cancer: a comparison of European and UK guidelines. J Clin Urol. 2018;11(2):144-148. 7 Babjuk M, Burger M, Capoun O, et al. European Association of Urology Guidelines on Non-muscle-invasive Bladder Cancer (Ta, T1, and Carcinoma in Situ). Eur Urol. 2022;81(1):75-94. doi:10.1016/j.eururo.2021.08.010 8 Brooks NA, O'Donnell MA. Treatment options in non–muscle-invasive bladder cancer after BCG failure. Indian J Urol. 2015;31(4):312-319. doi:10.4103/0970-1591.166475 9 Guancial EA, Roussel B, Bergsma DP, et al. Bladder cancer in the elderly patient: challenges and solutions. Clin Interv Aging. 2015;10:939-949. 10 Chamie K, Litwin MS, Bassett JC, et al. Recurrence of high-risk bladder cancer: A population-based analysis. Cancer. 2013;119(17):3219-3227. 11 Key statistics for prostate cancer. American Cancer Society. Accessed September 2024. 12 Cooperberg MR, Cowan J, Broering JM, et al. High-risk prostate cancer in the United States, 1990-2007. World J Urol. 2008;26(3):211-218. doi: 10.1007/s00345-008-0250-7. 13 Napodano G, Ferro M, Sanseverino R. High-risk prostate cancer: A very challenging disease in the field of uro-oncology. Diagnostics (Basel). 2021;11(3):400. doi: 10.3390/diagnostics11030400. SOURCE Johnson & Johnson WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Phase 3Breakthrough Therapy

14 Feb 2025

·endpts.com

Lilly’s early bladder cancer data hint at safety edge over J&J’s Balversa

Eli Lilly has shared first-in-human results for its selective FGFR3 inhibitor in a subset of bladder cancer patients, marking the start of a program that could eventually challenge Johnson & Johnson’s Balversa. Balversa is marketed for metastatic FGFR3-altered bladder cancer in the US, but the drug’s broad spectrum inhibition of FGFR means it comes with a host of serious side effects. The medicine is “extremely difficult to stay on” with patients often tapering doses, Lilly’s head of oncology Jacob Van Naarden told Endpoints News in an interview ahead of the confab. By contrast, Lilly’s LOXO-435 selectively targets the FGFR3 isoform, homing in on the somatic alteration that drives the cancer to avoid off-target effects. Lilly’s Phase 1 data from 70 patients with various solid tumors given a 200 mg or higher dose of the drug showed its most common side effect was diarrhea (76%). The side effect was mostly low-grade and manageable, according to data presented Friday at the ASCO genitourinary cancer symposium in San Francisco. Retinopathy and onycholysis were rare and treatment discontinuation rates for all doses 200 mg or higher were in the single-digit percentages, according to Lilly. Balversa patients experience serious retinal toxicities and hyperphosphatemia as well as fatigue and onycholysis. LOXO-435’s safety profile seems “similar to or slightly worse” than that for Tyra Biosciences’ selective FGFR3 inhibitor TYRA-300, Jefferies analysts said Monday when Lilly’s abstract was published on the confab website. In Tyra’s Phase 1/2 trial, its drug was associated with a lower rate of diarrhea (20%) and hyperphosphatemia (13% versus 28% for LOXO-435) in patients with FGFR3-altered metastatic urothelial carcinoma. But LOXO-435 saw fewer cases of increases in certain liver enzymes (22%) versus TYRA-300 (47%). As for efficacy, LOXO-435 is “on par with what we expect for this class” and “probably in the same ballpark as pan-FGFR inhibitors,” Lilly’s clinical lead for the program Arjun Balar told Endpoints. In Lilly’s study, LOXO-435 achieved a 41% confirmed overall response rate when given at doses of at least 200 mg to 39 patients with metastatic FGFR3-altered bladder cancer, according to Friday’s data. Tyra’s candidate hit a higher ORR of around 55% in its study, the Jefferies analysts highlighted. But “it’s very difficult to make any sort of comparisons [across trials] within Phase 1 patient populations,” Balar said. Tyra’s data came from just 11 subjects with a median of two prior lines of therapy versus three for the patients enrolled in Lilly’s trial. Lilly will now focus on refining an optimal dose of its drug and enrolling various expansion cohorts, including one evaluating a triplet regimen of LOXO-435, Pfizer/Seagen’s Padcev and Merck’s Keytruda in relevant patients with first-line metastatic bladder cancer. Other cohorts will include patients previously treated with Balversa and those naive to Balversa, Balar said. The company’s “ultimate goal” is to capture the market of around 15,000 patients worldwide with FGFR3-altered metastatic urothelial carcinoma, he added.

Clinical ResultPhase 1Drug Approval

02 Jan 2025

·www.globenewswire.com

Bionano Announces Publication from Johns Hopkins School of Medicine Showing that OGM Outperformed Multiple Cytogenetic Assays in a Study of Bone and Soft Tissue Tumor Analysis

In the largest study to date of bone and soft tissue tumors, OGM detected 100% of the variants found by multiple standard techniques, including karyotyping, fluorescent in-situ hybridization (FISH) & gene fusion assays OGM was also more sensitive, including detection of diagnostic or pathogenic variants missed by karyotype in 74% (14/19) of cases that failed or were negative by karyotyping When OGM results and next-generation sequencing (NGS) results were combined, diagnostic and pathogenic structural variants (SVs), copy number variants (CNVs), and/or single nucleotide variants (SNVs) were found in ~98% of cases, a substantially greater rate than when karyotyping, FISH and NGS are used Dec. 23, 2024 -- Bionano Genomics, Inc. (Nasdaq: BNGO) today announced a publication in Modern Pathology by a group of researchers at the Johns Hopkins University School of Medicine, showing that optical genome mapping (OGM) can outperform traditional techniques in analysis of bone and soft tissue tumors. Several prior publications have shown the utility of OGM compared to traditional cytogenetics in studies of hematologic malignancies, however, data on the application of OGM in solid tumors has been relatively sparse. This study provides compelling support for extending the utility of OGM in cancer beyond hematologic malignancies to solid tumors. OGM detected all variants revealed by conventional cytogenetics: OGM showed 100% concordance, identifying all pathogenic variants detected by standard of care cytogenetic methods. The specificity of OGM was assessed to be 100%, i.e. OGM correctly identified the same pathogenic SVs and CNVs detected by standard of care/routine cytogenetics (karyotyping and FISH). OGM detected pathogenic variants missed by karyotyping: In 74% of cases with normal or failed karyotype, OGM detected diagnostic or pathogenic SVs that were missed by karyotyping. Further, in 6 cases that failed to yield any karyotyping results due to culture failure, OGM detected pathogenic SVs in all of them. Variants found by OGM but missed by standard of care included the EWSR1::ETV1 fusion, which is a key molecular hallmark of clear cell sarcoma and helps to differentiate it from other soft tissue sarcomas and melanomas. OGM resolved complex cancer genomes: Study authors found that OGM data could re-characterized and better defined complex structural rearrangements including chromoanagenesis in 27% of cases and complex 3-6-way translocations in 15% of cases when compared to traditional cytogenetic methods. OGM combined with NGS found pathogenic variants in 98% of cases, a substantially greater rate than when karyotyping, FISH and NGS are used: The integrated approach of the combination of OGM and NGS resulted in the detection of pathogenic SVs and sequence variants in ~98% of cases. OGM was 100% concordant with NGS for aneuploidy detection. OGM findings have the potential to qualify subjects for targeted therapies that otherwise would not have been possible: The authors state that several of the OGM findings could result in the potential for these cases to qualify for either targeted treatments or clinical trials. For example, cases with potential to be treated by CDK4/6 inhibitors (palbociclib, ribociclib, abemaciclib), TRK inhibitors (larotrectinib, entrectinib), pan-FGFR inhibitors (erdafitinib or futibatinib) were highlighted. Erik Holmlin, president and chief executive officer of Bionano commented, “Approximately 50% of bone and soft tissue tumor samples fail to reveal actionable information for proper classification of disease, prognosis and therapeutic management because they either fail to culture or because traditional techniques in cytogenetics lack adequate sensitivity and specificity to reliably detect relevant variants. We have seen increasing evidence for OGM as a valuable alternative to cytogenetic methods in blood cancers, and we are thrilled to see researchers at Johns Hopkins publishing this compelling case for extending OGM’s utility to bone and soft tissue tumors.” The full research publication is available at: https://doi.org/10.1016/j.modpat.2024.100684 Bionano is a provider of genome analysis solutions that can enable researchers and clinicians to reveal answers to challenging questions in biology and medicine. The Company’s mission is to transform the way the world sees the genome through optical genome mapping (OGM) solutions, diagnostic services and software. The Company offers OGM solutions for applications across basic, translational and clinical research. The Company also offers an industry-leading, platform-agnostic genome analysis software solution, and nucleic acid extraction and purification solutions using proprietary isotachophoresis (ITP) technology. Through its Lineagen, Inc. d/b/a Bionano Laboratories business, the Company also offers OGM-based diagnostic testing services. The content above comes from the network. if any infringement, please contact us to modify.

Oligonucleotide

100 Deals associated with Erdafitinib

External Link

KEGG	Wiki	ATC	Drug Bank
D10927	Erdafitinib	L01XE	DB12147

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Locally Advanced Urothelial Carcinoma	China	Shanghai Johnson & Johnson Pharmaceutical Ltd.	08 Jan 2025
Metastatic urothelial carcinoma	European Union	Janssen-Cilag International NV	22 Aug 2024
Metastatic urothelial carcinoma	Iceland	Janssen-Cilag International NV	22 Aug 2024
Metastatic urothelial carcinoma	Liechtenstein	Janssen-Cilag International NV	22 Aug 2024
Metastatic urothelial carcinoma	Norway	Janssen-Cilag International NV	22 Aug 2024
Unresectable Urothelial Carcinoma	European Union	Janssen-Cilag International NV	22 Aug 2024
Unresectable Urothelial Carcinoma	Iceland	Janssen-Cilag International NV	22 Aug 2024
Unresectable Urothelial Carcinoma	Liechtenstein	Janssen-Cilag International NV	22 Aug 2024
Unresectable Urothelial Carcinoma	Norway	Janssen-Cilag International NV	22 Aug 2024
FGFR2 positive Transitional Cell Carcinoma	South Korea	Janssen Korea Ltd.	24 Nov 2022
FGFR3 positive Transitional Cell Carcinoma	South Korea	Janssen Korea Ltd.	24 Nov 2022
Bladder Cancer	Canada	Janssen Biotech, Inc.	09 Dec 2019
Transitional Cell Carcinoma	Canada	Janssen, Inc.	25 Oct 2019
FGFR positive Transitional Cell Carcinoma	United States	Janssen Biotech, Inc.	12 Apr 2019

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Advanced Urothelial Carcinoma	NDA/BLA	China	Janssen Cilag SpA	15 Dec 2023
Advanced Urothelial Carcinoma	NDA/BLA	China	Shanghai Johnson & Johnson Pharmaceutical Ltd.	15 Dec 2023
Advanced Urothelial Carcinoma	NDA/BLA	China	Janssen Biotech, Inc.	15 Dec 2023
Non-Muscle Invasive Bladder Neoplasms	Phase 3	United States	Janssen Research & Development LLC	01 Apr 2024
Non-Muscle Invasive Bladder Neoplasms	Phase 3	China	Janssen Research & Development LLC	01 Apr 2024
Non-Muscle Invasive Bladder Neoplasms	Phase 3	Japan	Janssen Research & Development LLC	01 Apr 2024
Non-Muscle Invasive Bladder Neoplasms	Phase 3	Argentina	Janssen Research & Development LLC	01 Apr 2024
Non-Muscle Invasive Bladder Neoplasms	Phase 3	Austria	Janssen Research & Development LLC	01 Apr 2024
Non-Muscle Invasive Bladder Neoplasms	Phase 3	Belgium	Janssen Research & Development LLC	01 Apr 2024
Non-Muscle Invasive Bladder Neoplasms	Phase 3	Brazil	Janssen Research & Development LLC	01 Apr 2024

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


AACR2025	Not Applicable	FGFR2 Mutant Neoplasms FGFR2/3 alterations	60	erdafitinib (Predicine FGFR2/3 qPCR Kit)	zewquduixn(lmqefrbdzw) = lxpdvwntwc dwsnybifpn (xenjqppncy ) View more	Positive	27 Apr 2025
NCT04083976 (RAGNAR, CTgov)	Phase 2	Advanced Malignant Solid Neoplasm	316	erdafitinib (Broad Panel Cohort)	hrbmdavaca = sklsmzrvor tzpmujkagf (lgfyiobvlo, byfahjqgew - mzdqnimxyh) View more	-	24 Feb 2025
NCT04083976 (RAGNAR, CTgov)	Phase 2	Advanced Malignant Solid Neoplasm	316	erdafitinib (Pediatric Cohort)	hrbmdavaca = psgpatkhpi tzpmujkagf (lgfyiobvlo, oobsqiifrc - piuecrjbao) View more	-	24 Feb 2025
NCT04963153 (ETCTN 10483, ASCO_GU2025) Manual	Phase 1	Metastatic urothelial carcinoma FGFR3 Mutation \| FGFR2 Mutation	9	Erdafitinib + Enfortumab Vedotin	nttcyuyayz(lathjdmkej) = hyperphosphatemia (88%), mucositis (88%), high AST (88%), hypercalcemia (75%), palmar plantar erythrodysesthesia (75%), peripheral neuropathy (75%), alopecia (63%), diarrhea (63%), hypoalbuminemia (63%) and hypomagnesemia (63%). hmsakwcbzy (bgzchslodi ) View more	Positive	13 Feb 2025
NCT03210714 (APEC1621B, CTgov)	Phase 2	Refractory Soft Tissue Sarcoma \| Refractory Central Nervous System Lymphoma \| Recurrent Medulloblastoma ... View more	20	Computed Tomography+Erdafitinib	qzwsvrruxj = htkdlzlxog ruuemfuhcu (pbygrgylqx, irnvoktpho - hujtkvnvbc) View more	-	19 Sep 2024
NCT05316155 (ESMO2024)	Phase 1/2	FGFR positive Cholangiocarcinoma	-	TAR-210	iprmcqzidt(uyokatprrt) = cxkhwoqpfq ysqltysswh (ofhmyyvnlt ) View more	-	15 Sep 2024
NCT02699606 (Pubmed \| BMC Cancer) Manual	Phase 2	Advanced Malignant Solid Neoplasm FGFR genomic aberrations	35	Erdafitinib 8 mg once daily	tqufvkwfds(bzrbsbmysu) = xpilivtqex fupaopafek (naxwlusbtu, 20.7 - 63.6) View more	Positive	13 Aug 2024
NCT02699606 (Pubmed \| BMC Cancer) Manual	Phase 2	Advanced Malignant Solid Neoplasm FGFR genomic aberrations	35	Erdafitinib 10 mg (7 days on/7 days off)	tqufvkwfds(bzrbsbmysu) = cgoqfwwjha fupaopafek (naxwlusbtu ) View more	Positive	13 Aug 2024
NCT04083976 (RAGNAR, ASCO2024) Manual	Phase 2	Non-Small Cell Lung Cancer CDKN2A \| FGFR mutations \| FGFR fusions ... View more	23	Erdafitinib	bmewjdzkhm(kxvmrxiibh) = negzczvrja hxxhtjzrzq (efmkajogwt, 10 - 48) View more	Positive	24 May 2024
NCT04083976 (RAGNAR, ASCO2024) Manual	Phase 2	Breast Cancer FGFR Mutation (Activating)	16	Erdafitinib 8 mg daily	nmxwwhboey(avfewahhao) = wcebregtho mpubttfzlf (ycuffidkzz, 11 - 59) View more	Positive	24 May 2024
NCT04083976 (RAGNAR, ASCO2024) Manual	Phase 2	Advanced Malignant Solid Neoplasm FGFR1 \| FGFR2 \| FGFR3 ... View more	53	Erdafitinib 8 mg daily	gzonzjsygz(xkhnewqkus) = ueywbpkfyw aywkkixxjw (oorvqsjzmo ) View more	Negative	24 May 2024
UNITE study (ASCO2024)	Not Applicable	Metastatic urothelial carcinoma FGFR2/3-altered \| MTAP loss	633	Erdafitinib	ooitxuqgtt(ypepmrpcsq) = naayyyftgm yntpfrczgq (fxahyxqzoy ) View more	Positive	24 May 2024

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