2019-04-23| Trials & Approvals

Erdafitinib Approval,a Bright Spot Amid the Troughs in Bladder Cancer Trials

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By GeneOnline staff

In a week that saw Clovis oncology’s phase 2 trial discontinued and Roche’s phase I trial cast aside, the USFDA green light for Erdafitinib comes as a positive news for patients suffering from metastatic Bladder Cancer.


Urothelial cancer ranks sixth among the most common types of cancer prevalent in the US. The cancer originates in the urothelial cells that line the inside of the bladder and metastasizes in a rapid and aggressive fashion. Apart from chemotherapy and surgery, six FDA approved immunotherapy drugs are currently available for metastatic urothelial cancer (mUC) that either target the PD-1 or PD-L1 proteins. While Atezolizumab (Tecentriq), Durvalumab (Imfinzi), and Avelumab (Bavencio) target PD-L1, Nivolumab (Opdivo) and Pembrolizumab (Keytruda) target PD-1. However, a targeted therapy for mUC wasn’t approved until now.

Last week witnessed the lapse of two trials evaluating drugs against bladder cancer. One was Clovis Oncology’s phase 2 open-label ATLAS study that tested Rucaparib for patients with recurrent, metastatic bladder cancer. As per the recommendations of an independent data monitoring committee, the study was discontinued due to lack of efficacy. The repercussions were immediate as the company’s stock slid by 13%. Similarly, Roche set aside a phase I study involving Tecentriq. However, both companies have other studies in their pipeline that plans to test these drugs in different combinations.

Erdafitinib, first FDA approved targeted therapy for mUC

On April 12th, the United States Food and Drug Administration announced the accelerated approval of Erdafitinib for the treatment of locally advanced or metastatic urothelial carcinoma. This makes it, the first FDA approved, fibroblast growth factor receptor (FGFR) kinase inhibitor for this indication. It is recommended for adult patients harboring genetic alterations in FGFR2 or FGFR3 susceptibility genes and who have tried at least one line of prior chemotherapy containing platinum and either did not or stopped responding to the treatment. QIAGEN’s therascreen, an FGFR RGQ RT-PCR Kit was approved as a companion diagnostic for this treatment.

History and background

Erdafitinib is marketed under the brand name Balversa and was codeveloped as an oral tablet by Janssen Pharmaceuticals of Johnson & Johnson and Astex Pharmaceuticals under a joint collaboration. Back in March 2018, the USFDA granted it breakthrough therapy designation after it excelled at a Phase 2 clinical trial BLC2001 (NCT02365597). It is a multicenter, open-label, single-arm study conducted in 87 urothelial carcinoma patients in whom, the disease had progressed on or after at least one prior chemotherapy. Secondly, the patients had at least one of the following genetic alterations: FGFR3 gene mutations (R248C, S249C, G370C, Y373C) or FGFR gene fusions (FGFR3-TACC3, FGFR3-BAIAP2L1, FGFR2-BICC1, FGFR2-CASP7).

Clinical trial data

The Balversa treated patients exhibited an overall response rate of 32.2% [95% CI(22.4, 42.0)], of which 2.3% of patients showed complete response while 29.9% showed a partial response. The results also showed a median duration of response (DoR) of 5.4 months [95% CI(4.2, 6.9)]. Many of the respondents were patients who did not show any response to PD-1/PD-L1 immunotherapies. The data from this study were presented at the ASCO 2018 Annual Meeting.

Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC shared his excitement over the approval. “We recognize the significant unmet need that persists in the treatment of men and women diagnosed with this form of urothelial carcinoma, and we have worked expeditiously to develop Balversa for patients in close consultation with the FDA,” he said in a statement. “We look forward to the continued development of Balversa to understand how this important new therapy may further inform the care of patients with metastatic urothelial carcinoma and its investigational use in other cancers where FGFR alterations may be present in the future.”



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