World first in bladder cancer with FDA approval of erdafitinib

Following the release of trial data, the FDA has approved erdafitinib for the treatment of adult patients with fibroblast growth factor receptor 3 (FGFR3) altered metastatic urothelial cancer that has progressed on platinum-containing chemotherapy.

Late-stage bladder cancer can present a poor prognosis with few treatment options. Arlene Siefker-Radtke, MD, of the University of Texas MD Anderson Cancer Center, Houston, TX, describes the present situation in an exclusive interview with VJOncology:

“Patients currently have very little [in terms of] treatment options for a stage IV bladder cancer. Platinum-based chemotherapy has been the standard and frontline approach, but a lot of patients are elderly, fragile, with poor kidney function, and can’t take such an aggressive regimen.

Additionally, there’s still a large number of patients who don’t benefit from immunotherapy. There is a clear unmet need for patients with bladder cancer to find a treatment that’s tolerable in poor kidney function, and has clinical activity.” 

FGFR3, the target of erdafitinib, plays key roles in the promotion of oncogenesis in a subset of patients with urothelial carcinoma. The inhibition of FGFR3 has been implicated in reduced proliferation of certain cancers. Dr Siefker-Radtke commented that “In early low-grade superficial tumors, the frequency is as high as 60%. So, one thought is they play a role in the development of bladder cancer, and may be targetable, even in patients with metastatic, incurable cancers for which there’s critically very limited treatment options.”

A study published in The New England Journal of Medicine (NCT02365597), in which Arlene was involved, investigated the use of erdafitinib for progressive bladder cancer. Dr Siefker-Radtke said:

“This is the first FGFR3 inhibitor approved for cancer, or approved in general. It’s also the first oral agent in bladder cancer and, some might argue, the first personalized therapy in bladder cancer based on the presence of an FGFR3 mutation.”

The trial investigated in 99 patients whether it best possible to give an FGFR3 therapy in a continuous or intermittent fashion, as wells as it’s efficacy and safety.

Dr Siefker-Radtke explains the findings of the trial:

“The trial did meet its primary endpoint, with an objective response rate of 40%. It also had an attractive overall survival of about 13.8 months, which compares quite favorably to the survival that had been reported with single-agent taxane. And even favorable when compared to immune-checkpoint inhibition, where the median survival may be in the order of 10 months. So erdafitinib appeared tolerable, and as a result, the FDA granted this therapy accelerated approval in April of 2019.”

Providing a new, efficacious option, erdafitinib will fill an unmet need for many bladder cancer patients. Arlene concluded our interview saying:

“The response rate does diminish in patients with additional metastases, and especially with liver metastases, the response rate of immunotherapy is extraordinarily poor. However, we saw a response to erdafitinib in patients with large volume, rapidly progressive disease. So, I do think this therapy meets an unmet need for our patient population.”

Further study into the use of erdafitinib and its combination with immunotherapy is currently underway and is hoped to offer even greater options to patients and physicians alike.

For more information on this trial, please click here


Ibrahim T, Gizzi M, Ratislav B, et al. Clinical Development of FGFR3 Inhibitors for the Treatment of Urothelial Cancer. Bladder Cancer. 2019 April; (Pre-print):1-5.
Loriot Y, Necchi A, Hoon Park, S, et al. Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma. The New England Journal of Medicine. 2019 July; 381:338-348.
Janssen Research & Development, LLC. (2019). An Efficacy and Safety Study of Erdafitinib (JNJ-42756493) in Participants With Urothelial Cancer. Available: Last accessed 25/07/2019.

Written by Thomas Southgate

Edited by Cally Cameron Smith