FDA approves Balversa for urothelial carcinoma with FGFR genetic alterations

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Janssen Pharmaceuticals said Balversa (erdafitinib) received accelerated approval from FDA for the treatment of adults with locally advanced or metastatic urothelial carcinoma which has susceptible fibroblast growth factor receptor 3 or FGFR2 genetic alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.

Balversa is the first FGFR kinase inhibitor approved by FDA. The accelerated approval follows FDA Breakthrough Therapy Designation in March 2018 and Priority Review Designation of the New Drug Application submitted in September 2018.

Balversa, a once-daily oral FGFR kinase inhibitor, received accelerated approval based on results from a phase II clinical trial (BLC2001, NCT02365597), a multicenter, open-label, single-arm study, of 87 patients with disease that had progressed on or after at least one prior chemotherapy and that 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), as determined by a clinical trial assay performed at a central laboratory.

The results demonstrated a 32.2% objective response rate as assessed by Blinded Independent Review Committee [95% CI (22.4, 42.0)]. Responders included patients who had previously not responded to anti PD-L1/PD-1 therapy.

In the trial, ORR was defined as the percentage of patients with measurable lesions achieving a complete response [2.3%] or partial response [29.9%] to treatment using the Response Evaluation Criteria in Solid Tumors Version 1.1 criteria, a standard way to measure how well a patient responds to treatment based on whether tumors shrink, stay the same, or get bigger as assessed per investigator.

Results also showed a median duration of response of 5.4 months [95% CI (4.2, 6.9)] in patients treated with Balversa. There were no confirmed responses to Balversa in the FGFR2 fusion patient population (n=6). Data from the BLC2001 study were presented at the ASCO 2018 Annual Meeting (Abstract #4503) and were recognized as a “Best of ASCO” selection.

FDA simultaneously approved a companion diagnostic for use with Balversa, the QIAGEN therascreen FGFR RGQ Reverse-transcription-polymerase chain reaction Kit, which is the first PCR-based companion diagnostic approved to detect FGFR alterations.

The therascreen FGFR test detects the presence of FGFR alterations in the tumor tissue of patients with mUC. If one or more of the genetic alterations or fusions are detected, the patient may be a candidate for treatment with Balversa.

Balversa (erdafitinib) is a once-daily, oral fibroblast growth factor receptor kinase inhibitor indicated for the treatment of adults with locally advanced or metastatic urothelial carcinoma which has susceptible FGFR3 or FGFR2 genetic alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.

In 2008, Janssen entered into an exclusive worldwide license and collaboration agreement with Astex Pharmaceuticals to develop and commercialize Balversa. Balversa will be commercially available through the single-source specialty pharmacy provider US Bioservices.

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