Erdafitinib

In early April of 2019, the US FDA approved Janssen Pharmaceutical Companies' brand name Balversa (erdafitinib) as the first-ever fibroblast growth factor receptor (FGFR) kinase inhibitor indicated for patients with locally advanced or metastatic urothelial carcinoma, with susceptible FGFR3 or FGFR2 genetic alterations, that has progressed during or following platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. At the same time, the FDA also approved the therascreen FGFR RGQ RT-PCR Kit (Qiagen) for utilization as a companion diagnostic with erdafitinib for selecting patients for the indicated therapy . Erdafitinib is the first personalized treatment targeting susceptible FGFR genetic alterations for patients with metastatic bladder cancer, which demonstrates the development of more personalized and precise medicines tailoring to a patient's specific genetic mutation. Considering urothelial cancer is statistically the fourth most common kind of cancer in the world, the introduction of erdafitinib offers a much-needed new option in the ever-expanding therapeutic tool kit to treat such a prevalent medical condition. Nevertheless, although erdafitinib was granted Breakthrough Therapy designation and Accelerated Approval from the FDA, such designations mean further ongoing clinical trials are necessary to confirm the clinical benefit of erdafitinib going forward.

Erdafitinib is a pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor that is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma that has susceptible FGFR3 or FGFR2 genetic alterations and has progressed during or following at least one line of prior platinum-containing chemotherapy including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. The selection of patients for the treatment of locally advanced or metastatic urothelial carcinoma with erdafitinib should be based on the presence of susceptible FGFR genetic alterations in tumor specimens as detected by an FDA-approved companion diagnostic like the FDA-approved therascreen FGFR RGQ RT-PCR Kit as developed by QIAGEN. This above indication is approved under accelerated approval by the FDA based on tumor response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

• Locally Advanced or Metastatic Urothelial Carcinoma (UC)