Erdafitinib

Erdafitinib is a kinase inhibitor that binds to and inhibits enzymatic activity of FGFR1, FGFR2, FGFR3 and FGFR4 based on in vitro data. Erdafitinib inhibited FGFR phosphorylation and signaling and decreased cell viability in cell lines expressing FGFR genetic alterations, including point mutations, amplifications, and fusions.

• BALVERSA is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible FGFR3 genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy. Select patients for therapy based on an FDA-approved companion diagnostic for BALVERSA [see Dosage and Administration (2.1) and Clinical Studies (14.1) ] .

• None. None.contraindicated

Confirm the presence of FGFR3 genetic alterations in tumor specimens prior to initiation of treatment with BALVERSA. ( 2.1 ) Recommended initial dosage: 8 mg orally once daily with a dose increase to 9 mg daily if criteria are met. ( 2.2 ) Swallow whole with or without food. ( 2.2 ) 2.1 Patient Selection Select patients for the treatment of locally advanced or metastatic urothelial carcinoma with BALVERSA based on the presence of susceptible FGFR3 genetic alterations in tumor specimens as detected by an FDA-approved companion diagnostic [see Clinical Studies (14.1) ] . Information on FDA-approved tests for the detection of FGFR3 genetic alterations in urothelial cancer is available at: http://www.fda.gov/CompanionDiagnostics. 2.2 Recommended Dosage and Schedule The recommended starting dose of BALVERSA is 8 mg (two 4 mg tablets) orally once daily, with a dose increase to 9 mg (three 3 mg tablets) once daily based on tolerability, including hyperphosphatemia, at 14 to 21 days [see Dosage and Administration (2.3) ]. Swallow tablets whole with or without food. If vomiting occurs any time after taking BALVERSA, the next dose should be taken the next day. Treatment should continue until disease progression or unacceptable toxicity occurs. If a dose of BALVERSA is missed, it can be taken as soon as possible on the same day. Resume the regular daily dose schedule for BALVERSA the next day. Extra tablets should not be taken to make up for the missed dose. Dose Increase based on Serum Phosphate Levels Assess serum phosphate levels 14 to 21 days after initiating treatment.

Ocular disorders: BALVERSA can cause central serous retinopathy/retinal pigment epithelial detachment (CSR/RPED). Perform monthly ophthalmological examinations during the first four months of treatment, every 3 months afterwards, and at any time for visual symptoms. Withhold BALVERSA when CSR/RPED occurs and permanently discontinue if it does not resolve within 4 weeks or if Grade 4 in severity. ( 2.3 , 5.1 ) Hyperphosphatemia: Increases in phosphate levels are a pharmacodynamic effect of BALVERSA. Monitor for hyperphosphatemia and manage with dose modifications when required. ( 2.3 , 5.2 ) Embryo-fetal toxicity: Can cause fetal harm. Advise patients of the potential risk to the fetus and to use effective contraception ( 5.3 , 8.1 , 8.3) 5.1 Ocular Disorders BALVERSA can cause ocular disorders, including central serous retinopathy/retinal pigment epithelial detachment (CSR/RPED) resulting in visual field defect. In the pooled safety population [see Adverse Reactions (6) ] , CSR/RPED occurred in 22% of patients treated with BALVERSA, with a median time to first onset of 46 days. In 104 patients with CSR, 40% required dose interruptions and 56% required dose reductions; 2.9% of BALVERSA-treated patients required permanent discontinuation for CSR. Of the 24 patients who restarted BALVERSA after dose interruption with or without dose reduction, 67% had recurrence and/or worsening of CSR after restarting. CSR was ongoing in 41% of the 104 patients at the time of last evaluation. Dry eye symptoms occurred in 26% of BALVERSA-treated patients.

The following serious adverse reactions are also described elsewhere in the labeling: Ocular Disorders [see Warnings and Precautions (5.1) ] . Hyperphosphatemia [see Warnings and Precautions (5.2) ] . The most common (>20%) adverse reactions, including laboratory abnormalities, were increased phosphate, nail disorders, stomatitis, diarrhea, increased creatinine, increased alkaline phosphatase, increased alanine aminotransferase, decreased hemoglobin, decreased sodium, increased aspartate aminotransferase, fatigue, dry mouth, dry skin, decreased phosphate, decreased appetite, dysgeusia, constipation, increased calcium, dry eye, palmar-plantar erythrodysesthesia syndrome, increased potassium, alopecia, and central serous retinopathy. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Janssen Products, LP. at 1-800-526-7736 (1-800-JANSSEN and www.BALVERSA.com) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The pooled safety population described in the WARNINGS AND PRECAUTIONS reflects exposure to BALVERSA as a single agent at the recommended dose (8 to 9 mg orally daily) in 479 patients with advanced urothelial cancer and FGFR alterations in 42756493BLC3001 (NCT03390504), 42756493BLC2001 (NCT02365597), 42756493BLC2002 (NCT 03473743), and 42756493EDI1001 (NCT01703481).

Lactation: Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on the mechanism of action and findings in animal reproduction studies, BALVERSA can cause fetal harm when administered to a pregnant woman [see Clinical Pharmacology (12.1) ] . There are no available data on BALVERSA use in pregnant women to inform a drug-associated risk. Oral administration of erdafitinib to pregnant rats during organogenesis caused malformations and embryo-fetal death at maternal exposures that were less than the human exposures at the maximum recommended human dose based on AUC (see Data ) . Advise pregnant women and females of reproductive potential of the potential risk to the fetus. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2–4% and 15–20%, respectively. Data Animal Data In an embryo-fetal toxicity study, erdafitinib was orally administered to pregnant rats during the period of organogenesis.

Metabolism

Following administration of BALVERSA 8 mg once daily, the mean (coefficient of variation [CV%]) erdafitinib steady-state maximum plasma concentration (C max ), area under the curve (AUC tau ), and minimum plasma concentration (C min ) were 1,399 ng/mL (51%), 29,268 ng∙h/mL (60%), and 936 ng/mL (65%), respectively. Following single and repeat once daily dosing of BALVERSA, erdafitinib exposure (C max and AUC) increased proportionally across the dose range of 0.5 to 12 mg (0.06 to 1.3 times the maximum approved recommended dose).

• Apr 12, 2019NDANDA212018Janssen Biotech

1. 1Death26222%
2. 2Off Label Use958.0%
3. 3Diarrhoea887.4%
4. 4Stomatitis675.7%
5. 5Nail Disorder514.3%
6. 6Onycholysis504.2%
7. 7Fatigue494.1%
8. 8Hyperphosphataemia453.8%
9. 9Dry Mouth433.6%
10. 10Disease Progression423.6%
11. 11Dry Eye393.3%
12. 12Mucosal Inflammation383.2%
13. 13Nail Discolouration373.1%
14. 14Decreased Appetite353.0%
15. 15Drug Ineffective353.0%

The 10 most recently updated of 43 ClinicalTrials.gov registrations naming Erdafitinib as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• 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 · Interventional · 629 enrolled · Janssen Research & Development, LLC
  NCT03390504updated 2026-06-08
• A Study to Evaluate TAR-210 Versus Intravesical Chemotherapy Treatment in Participants With High Risk Non-Muscle-Invasive Bladder Cancer
  Recruiting · Phase 3 · Interventional · 220 enrolled · Janssen Research & Development, LLC
  NCT06919965updated 2026-06-05
• An Efficacy and Safety Study of Erdafitinib (JNJ-42756493) in Participants With Urothelial Cancer
  Active not recruiting · Phase 2 · Interventional · 239 enrolled · Janssen Research & Development, LLC
  NCT02365597updated 2026-06-05
• Study of Erdafitinib Intravesical Delivery System for Localized Bladder Cancer
  Recruiting · Phase 1 · Phase 2 · Interventional · 235 enrolled · Janssen Research & Development, LLC
  NCT05316155updated 2026-06-05
• A Study of Erdafitinib Intravesical Delivery System in Japanese Participants With Bladder Cancer
  Active not recruiting · Phase 1 · Interventional · 5 enrolled · Janssen Pharmaceutical K.K.
  NCT05567185updated 2026-06-05
• A Study to Evaluate TAR-210 Versus Single Agent Intravesical Cancer Treatment in Participants With Bladder Cancer
  Recruiting · Phase 3 · Interventional · 641 enrolled · Janssen Research & Development, LLC
  NCT06319820updated 2026-06-05
• 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 · Interventional · 6,452 enrolled · National Cancer Institute (NCI)
  NCT02465060updated 2026-06-03
• Testing the Anti-cancer Drug Erdafitinib for Brain Cancers That Have Returned or Progressed Following Treatment
  Recruiting · Phase 2 · Interventional · 30 enrolled · National Cancer Institute (NCI)
  NCT05859334updated 2026-06-02
• 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)
  Active not recruiting · Phase 2 · Interventional · 1,377 enrolled · National Cancer Institute (NCI)
  NCT03155620updated 2026-06-01
• 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 · Interventional · 20 enrolled · National Cancer Institute (NCI)
  NCT03210714updated 2026-06-01

CPIC-curated drug–gene pairs for Erdafitinib. Levels describe the strength of curated evidence and guideline status — never a recommendation to test or to adjust therapy.

• CYP2C9CPIC B/C (provisional)FDA label: Actionable PGx

Frequently asked questions

How does Erdafitinib work?

Erdafitinib is a kinase inhibitor that binds to and inhibits enzymatic activity of FGFR1, FGFR2, FGFR3 and FGFR4 based on in vitro data. Erdafitinib inhibited FGFR phosphorylation and signaling and decreased cell viability in cell lines expressing FGFR genetic alterations, including point mutations, amplifications, and fusions.

What is Erdafitinib used for?

According to FDA labeling, Erdafitinib carries indications including: BALVERSA is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible FGFR3 genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy. Select patients for therapy based on an FDA-approved companion diagnostic for BALVERSA [see Dosage and Administration (2.1) and Clinical Studies (14.1) ] .. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Erdafitinib?

Erdafitinib is classified as Fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitors, Kinase Inhibitor, Cytochrome P450 3A4 Inducers, Cytochrome P450 3A4 Inhibitors, Fibroblast Growth Factor Receptor Inhibitors, Organic Cation Transporter 2 Inhibitors, P-Glycoprotein Inhibitors, Tyrosine Kinase Inhibitors, Cellular Proliferation Alteration.

What are the brand names for Erdafitinib?

Erdafitinib is marketed under brand names including Balversa.

What are the contraindications for Erdafitinib?

Erdafitinib labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.