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Erdafitinib Approved for Locally Advanced or Metastatic Urothelial Cancer

Clear cell urothelial carcinoma.

The FDA has granted approval to erdafitinib (Balversa™, Janssen Biotech) for adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations, as determined by an FDA-approved companion diagnostic test, whose disease has progressed on or after at least one line of prior systemic therapy. Patients are not recommended to receive treatment with erdafitinib if they are eligible for and have not received prior programmed cell death protein 1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor therapy. The previous accelerated approval, which was granted for patients with mUC with susceptible FGFR3 or FGFR2 alterations after prior platinum-containing chemotherapy, has been amended due to this approval.  

Why it matters: "Erdafitinib adds a unique therapy to the armamentarium of therapies in metastatic urothelial cancer, an area of unmet clinical need," said Ulka Vaishampayan, MD, MBBS, an Oncology Data Advisor Editorial Board Member and a Professor of Internal Medicine and Director of the Phase 1 Program at the University of Michigan Rogel Cancer Center.

Ulka Vaishampayan, MD, MBBS

What they studied: Efficacy was evaluated in Cohort 1 of the BLC3001 study (NCT03390504), a phase 3, open-label trial, in which 266 patients with mUC harboring selected FGFR3 alterations who had received one to two prior systemic treatments, including a PD-1 or PD-L1 inhibitor, were enrolled. Participating patients were randomized 1:1 to receive either erdafitinib or investigator's choice of chemotherapy—docetaxel or vinflunine. Patients were stratified by region, performance status, and presence of visceral or bone metastases. In 75% of patients, FGFR3 alterations were identified by the therascreen FGFR RGQ RT-PCR Kit (Qiagen) using tumor tissue samples in a central laboratory; the remaining alterations were identified locally by next-generation sequencing assays.

The primary end point was overall survival. Secondary end points included investigator-assessed progression-free survival and objective response rate.

What they found: Treatment with erdafitinib produced statistically significant improvements in overall survival, progression-free survival, and objective response rate compared with chemotherapy.

  • The median overall survival was 12.1 months in patients receiving erdafitinib compared with 7.8 months in patients receiving chemotherapy
  • The median progression-free survival was 5.6 months in those receiving erdafitinib compared with 2.7 months in those receiving chemotherapy
  • The confirmed objective response rate was 35.3% in those receiving erdafitinib compared with 8.5% in those receiving chemotherapy

"Erdafitinib showed consistency of results, and benefit was proved over taxane-based chemotherapy with progression-free survival and overall survival benefit," commented Dr. Vaishampayan.

Adverse events: The most common adverse events experienced in >20% of patients receiving erdafitinib, including laboratory abnormalities, were increased phosphate, nail disorders, diarrhea, stomatitis, increased alkaline phosphatase, decreased hemoglobin, increased alanine aminotransferase, increased aspartate aminotransferase, decreased sodium, increased creatinine, dry mouth, decreased phosphate, palmar-plantar erythrodysesthesia syndrome, dysgeusia, fatigue, dry skin, constipation, decreased appetite, increased calcium, alopecia, dry eye, increased potassium, and decreased weight.

Conclusion: "The study results confirm the efficacy and tolerability of FGFR3-targeted therapy in urothelial cancer. The sequencing recommended is to use erdafitinib after immune checkpoint therapy in advanced urothelial cancer patients that have FGFR3 mutations," concluded Dr. Vaishampayan. "This approval represents a step in the right direction towards solidifying the use of precision medicine in urothelial carcinoma."


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