EMA Accepts Application for Erdafitinib in EGFR+ Urothelial Carcinoma

The FDA granted accelerated approval to erdafitinib for treating adult patients with advanced or metastatic urothelial carcinoma harboring FGFR2/3 alterations in April 2019 based on data from a phase 2 study (NCT02365597).

The European Medicines Agency has accepted a marketing authorization application for erdafitinib (Balversa) as a treatment for adult patients with FGFR3-altered, locally advanced unresectable or metastatic urothelial carcinoma that has progressed following therapy with a PD-L1 inhibitor, according to a press release from The Janssen Pharmaceutical Companies of Johnson & Johnson.¹

Developers supported their application with findings from the phase 3 THOR study (NCT03390504). According to findings presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting, the median overall survival (OS) in cohort 1, which included those previously treated with an anti–PD-L1 agent, was 12.1 months in patients treated with erdafitinib vs 7.8 months in those who received chemotherapy; the experimental agent reduced the risk of death by 36% compared with chemotherapy (HR, 0.64; 95% CI, 0.47-0.88; P = .005).²

As a result, treatment with erdafitinib met the trial’s primary end point of OS and predefined criteria for superiority compared with chemotherapy. Additionally, the safety profile of erdafitinib in the THOR study was comparable with prior reports of the agent in metastatic urothelial carcinoma.

“For patients with advanced [urothelial carcinoma], including FGFR-driven tumors, outcomes remain poor and treatment options are limited; therefore, there is a need for novel, targeted therapies,” Martin Vogel, EMEA Therapeutic Area Lead Oncology at Janssen-Cilag GmbH, said in the press release.¹ “We are excited by the prospect of bringing innovative, personalized approaches to market for patients as we work towards our wider goal of making this complex disease a more manageable and ultimately curable condition.”

In the open-label, randomized THOR study, patients in cohort 1 received 8 mg of erdafitinib daily with pharmacodynamically guided uptitration to 9 mg or investigator’s choice of docetaxel or vinflunine. The study’s primary end point was OS, and secondary end points included progression-free survival, objective response rate, and duration of response.

The FDA granted accelerated approval to erdafitinib for treating adult patients with advanced or metastatic urothelial carcinoma harboring FGFR2/3 alterations in April 2019 based on data from a phase 2 study (NCT02365597).³ Additionally, the FDA accepted a supplemental new drug application (sNDA) for erdafitinib’s full approval in the same patient population in August 2023, which was supported by findings from cohort 1 of the THOR study.⁴

“Through the ongoing development of [erdafitinib], we are committed to transforming bladder cancer treatment to positively impact the lives of patients,” Peter Lebowitz, MD, PhD, global therapeutic head of Oncology at Janssen Research & Development, said in a press release at the time the FDA accepted the sNDA.⁴

References

1. Janssen submits Marketing Authorisation Application to the European Medicines Agency seeking approval of erdafitinib for the treatment of patients with locally advanced or metastatic urothelial cancer with susceptible FGFR alterations. News release. Janssen. September 8, 2023. Accessed September 11, 2023. bit.ly/3Ze9xXx
2. Loriot Y, Matsubara N, Park SH, et al. Phase 3 THOR study: results of erdafitinib (erda) versus chemotherapy (chemo) in patients (pts) with advanced or metastatic urothelial cancer (mUC) with select fibroblast growth factor receptor alterations (FGFRalt). J Clin Oncol. 2023;41(suppl 17):LBA4619. doi:10.1200/JCO.2023.41.17_suppl.LBA4619
3. BALVERSA™ (erdafitinib) receives U.S. FDA approval for the treatment of patients with locally advanced or metastatic urothelial carcinoma with certain FGFR genetic alterations. News release. Janssen. April 12, 2019. Accessed September 11, 2023. https://shorturl.at/dkK02
4. Janssen submits supplemental new drug application to the U.S. Food and Drug Administration seeking full approval of BALVERSA® (erdafitinib) for the treatment of patients with locally advanced or metastatic urothelial carcinoma and selected fibroblast grow. News release. Janssen. August 28, 2023. Accessed September 11, 2023. https://shorturl.at/oqru3