Study assesses benefit of erdafitinib in patients with advanced urothelial carcinoma and FGFR mutation

Study results BLC2001 (757P – An observational study of outcomes of patients (pts) with advanced urothelial carcinoma (UC) after anti-programmed death- (Ligand) 1 (PD- [L] 1) therapy by fibroblast growth factor receptor gene alteration (FGFRa) status), presented at the ESMO Virtual Congress 2020. This is a retrospective analysis of clinical outcomes in response to immunotherapy (anti PD-L1) in patients with locally advanced or metastatic urothelial carcinoma FGFR + versus FGFR-.

The BLC2001 trial showed an objective response rate of around 40% with the use of erdafitinib in patients with locally advanced or metastatic urothelial carcinoma, which led to the approval of the drug in Brazil and other countries.

In addition, another interesting point observed by the BLC2001 researchers is that 22 of the 99 recruited patients had previously received immunotherapy and, of these 22, only one responded to treatment. These data thus reinforce the idea that the FGFR gene mutation could be a factor of negative predictive value for response to immunotherapy.

About the poster presented at ESMO, a database of 92 patients treated with immunotherapy was evaluated, of which 38 had FGFR mutation or fusion. What was observed was that the chance of response with previous immunotherapy in those who have changes in FGFR is less than in those who do not have such changes.

However, there is still a chance of a response to immunotherapy, since among the 38 patients with mutated FGFR, ORR was 15.8% in immunotherapy against 25.9% in patients without this mutation. With the use of immunotherapy in the first line, these numbers become 28.6% against 30%.

Another important result of the study was the evaluation of the median overall survival of the population with a FGFR mutation compared to that of the population without the mutation: 7.5 months versus 11.4 months, respectively.

For some experts, the data presented does not yet exclude patients without a FGFR mutation from receiving immunotherapy previously.

References:

757P – An observational study of outcomes of patients (pts) with advanced urothelial carcinoma (UC) after anti-programmed death-(Ligand) 1 (PD-[L]1) therapy by fibroblast growth factor receptor gene alteration (FGFRa) status. ESMO Virtual Congress, 2020.

Loriot Y, Necchi A, et al. Erdafitinib in Locally Advanced or Metastatic Urothelial Carcinoma. N Engl J Med 2019