marked.bio

FGFR inhibitor · small molecule

erdafitinib

12 active trials across 3 of our biomarkers, targeting FGFR1-4. 2 active approvals for a biomarker on marked.bio.

Also known as JNJ-42756493, Balversa.

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
erdafitinib
FDA, 2019
locally advanced or metastatic, susceptible FGFR3 alteration, after >=1 prior systemic therapy (not before a PD-1/PD-L1 inhibitor if eligible) Accelerated approval 2019 (FGFR3 or FGFR2, after platinum); traditional approval 19/01/2024 narrowed to FGFR3. Source
EU (EMA), 2024
unresectable or metastatic, susceptible FGFR3 alterations, after >=1 line containing a PD-1/PD-L1 inhibitor EU marketing authorisation 22/08/2024. Source
Balversa 		FGFR3 alteration (mutation or fusion) urothelial Approved 2019 Approved 2024

THOR (BLC3001 cohort 1) vs docetaxel or vinflunine, 266 randomised (1:1)
PFS 5.6 months vs 2.7 months, HR 0.58 (0.44-0.78)Response 35% vs 9%OS 12.1 months vs 7.8 months, HR 0.64 (0.47-0.88)

Trial details and side effects
Patients
FGFR3-altered metastatic urothelial cancer after a PD-(L)1 inhibitor (NCT03390504)
Most common side effects
high phosphate, nail disorders, diarrhoea, stomatitis, raised alkaline phosphatase, low haemoglobin, raised ALT/AST, dry mouth, hand-foot syndrome, taste changes, fatigue
Notes
FDA approval summary (2024); side effects are those in more than 20% of patients.
Source
FDA approval summary

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
FGFR3 alteration (mutation or fusion) 11 16 Phase 3
FGFR2 fusion or rearrangement 10 15 Phase 3
MSI-H / dMMR 1 1 Phase 2

Sponsors

Industry sponsors leading active biomarker-selected trials of erdafitinib.

  1. Johnson & Johnson 6

Trials

12 of 17 trials shown

  1. A Study to Evaluate TAR-210 Versus Intravesical Chemotherapy Treatment in Participants With High Risk Non-Muscle-Invasive Bladder Cancer

    Recruiting Phase 3 Johnson & Johnson Started 2025Primary completion 2028-04 NCT06919965

    erdafitinib FDA EUMitomycin Cgemcitabine

    Have a susceptible fibroblast growth factor receptor (FGFR) mutation or fusion either by urine testing or tumor tissue testing (from TURBT tissue) as determin…

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementNon-Muscle Invasive Bladder Neoplasms

  2. A Study to Evaluate TAR-210 Versus Single Agent Intravesical Cancer Treatment in Participants With Bladder Cancer

    Recruiting Phase 3 Johnson & Johnson Started 2024Primary completion 2028-06 NCT06319820

    erdafitinib FDA EUgemcitabineMMC

    Have a susceptible fibroblast growth factor receptor (FGFR) mutation or fusion either by urine testing or tumor tissue testing (from TURBT tissue), as determi…

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementNon-Muscle Invasive Bladder Neoplasms

  3. A Study of Erdafitinib Compared With Vinflunine or Docetaxel or Pembrolizumab in Participants With Advanced Urothelial Cancer and Selected Fibroblast Growth Factor Receptor (FGFR) Gene Aberrations

    Active, not recruiting Phase 3 Johnson & Johnson Started 2018Primary completion 2024-04 NCT03390504

    erdafitinib FDA EUVinfluninedocetaxelpembrolizumab

    …ticipants With Advanced Urothelial Cancer and Selected Fibroblast Growth Factor Receptor (FGFR) Gene Aberrations

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementUrothelial Cancer

  4. Erdafitinib Monotherapy or in Combination With Cetrelimab in Muscle-invasive Bladder Cancer Patients With Fibroblast Growth Factor Receptor (FGFR ) Gene Alterations

    Recruiting Phase 2 Spanish Oncology Genito-Urinary Group Started 2023Primary completion 2026-07 NCT06511648

    erdafitinib FDA EU

    Presence of a selected FGFR alteration on analysis of tumour biopsy

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementMuscle-invasive Bladder Cancer

  5. A Study of Oral Erdafitinib in People With Recurrent Non-Invasive Bladder Cancer

    Active, not recruiting Phase 2 Memorial Sloan Kettering Cancer Center Started 2022Primary completion 2027-08 NCT04917809

    erdafitinib FDA EU

    Documentation on MSK-IMPACT of an oncogenic FGFR3 mutation (R248C, S249C, G370C, Y373C, etc.) or FGFR3 gene fusion with compelling clinical or biolo…

    FGFR3 alteration (mutation or fusion)Bladder Cancer, Recurrent Bladder Cancer, FGFR3 Gene Mutation

  6. Study of Erdafitinib Intravesical Delivery System for Localized Bladder Cancer

    Active, not recruiting Phase 1/2 Johnson & Johnson Started 2022Primary completion 2028-03 NCT05316155

    erdafitinib FDA EU

    For selected Cohorts: Activating tumor pan-fibroblast growth factor receptor (FGFR) mutation or fusion, as determined by local or central testing, approved by the sponsor prior to th…

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementNon-Muscle Invasive Bladder Neoplasms

  7. Testing JNJ-42756493 (Erdafitinib) as Potentially Targeting Treatment in Cancers With FGFR Mutations or Fusions (MATCH - Subprotocol K2)

    Active, not recruiting Phase 2 National Cancer Institute (NCI) Started 2018Primary completion 2022-06 NCT06351371

    erdafitinib FDA EU

    Patients must have FGFR mutation or fusion as determined via the MATCH Master Protocol

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementMalignant Solid Neoplasm

  8. Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)

    Active, not recruiting Phase 2 National Cancer Institute (NCI) Started 2018Primary completion 2023-09 NCT03210714

    erdafitinib FDA EU

    … or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementAdvanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma

  9. An Efficacy and Safety Study of Erdafitinib (JNJ-42756493) in Participants With Urothelial Cancer

    Active, not recruiting Phase 2 Johnson & Johnson Started 2015Primary completion 2022-09 NCT02365597

    erdafitinib FDA EUmidazolammetformin

    …NJ-42756493 in Subjects With Metastatic or Surgically Unresectable Urothelial Cancer With FGFR Genomic Alterations

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementUrothelial Cancer

  10. Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial)

    Active, not recruiting Phase 2 National Cancer Institute (NCI) Started 2015Primary completion 2027-12 NCT02465060

    adavosertibafatinibbinimetinibcapivasertibcopanlisibcrizotinibdabrafenibDasatinibdefactiniberdafitinibfexagratinibipatasertib

    …candidate for Z1M based on local Clinical Laboratory Improvement Act (CLIA) assessment of mismatch repair deficiency (MMRd) by immunohistochemistry (IHC) or microsatellite instability (MSI) status by polyme…

    MSI-H / dMMRAdvanced Lymphoma, Advanced Malignant Solid Neoplasm, Bladder Carcinoma, Breast Carcinoma

  11. A Study of Erdafitinib Intravesical Delivery System in Japanese Participants With Bladder Cancer

    Active, not recruiting Phase 1 Johnson & Johnson Started 2023Primary completion 2025-03 NCT05567185

    erdafitinib FDA EU

    At least 1 of the study protocol defined activating fibroblast growth factor receptors (FGFR) mutation or fusion, as determined by local or central testing using either tumor tissue or urine s…

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementUrinary Bladder Neoplasms, Receptors, Fibroblast Growth Factor

  12. Testing Combination Erdafitinib and Enfortumab Vedotin in Metastatic Bladder Cancer After Treatment With Chemotherapy and Immunotherapy

    Active, not recruiting Phase 1 National Cancer Institute (NCI) Started 2022Primary completion 2026-12 NCT04963153

    enfortumab vedotinerdafitinib FDA EU

    …ith Enfortumab Vedotin Following Prior Therapies for Metastatic Urothelial Carcinoma With FGFR2/3 Genetic Alterations

    FGFR3 alteration (mutation or fusion)FGFR2 fusion or rearrangementLocally Advanced Bladder Urothelial Carcinoma, Locally Advanced Renal Pelvis Urothelial Carcinoma, Locally Advanced Ureter Urothelial Carcinoma, Locally Advanced Urethral Urothelial Carcinoma