Editor’s note: this article was updated to address an error.

The US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to erdafitinib for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC), according to a press release.1

Erdafitinib — an oral pan-fibroblast growth factor receptor tyrosine kinase inhibitor (FGFR-TKI) — is the first targeted therapy for urothelial cancer.

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The FDA based its approval on the results of the ongoing phase 2 BLC2001 study (ClinicalTrials.gov Identifier: NCT02365597), for which researchers assigned 78 and 33 patients to receive continuous or intermittent dosing of erdafitinib, respectively. Eligible patients had mUC with specific FGFR2/FGFR3 mutations or translocations, had failed at least 1 line of chemotherapy, or received less than 1 year of neoadjuvant chemotherapy.

The overall response rate was 42% among 59 patients with mUC, and the disease control rate was 74% for patients who received continuous dosing compared with 73% for intermittent dosing.2

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The most commonly observed treatment-related adverse events (AEs) included hyperphosphatemia, diarrhea, and FGFR inhibitor class effects (eg, nail, skin, and eye AEs), but AEs were manageable and did not lead to any treatment-related deaths.


  1. Janssen announces US FDA breakthrough therapy designation for erdafitinib in the treatment of metastatic urothelial cancer [news release]. Raritan, NJ: PRNewswire; March 15, 2018. https://prnmedia.prnewswire.com/news-releases/janssen-announces-us-fda-breakthrough-therapy-designation-for-erdafitinib-in-the-treatment-of-metastatic-urothelial-cancer-300614532.html. Accessed March 15, 2018.
  2. Loriot Y, Necchi A, Park SH, et al. Erdafitinib (ERDA; JNJ-42756493), a pan-fibroblast growth factor receptor (FGFR) inhibitor, in patients (pts) with metastatic or unresectable urothelial carcinoma (mUC) and FGFR alterations (FGFRa): Phase 2 continuous versus intermittent dosing. Oral presentation at: 2018 Genitourinary Cancers Symposium; February 8-10, 2018; San Francisco, CA.