15 April 2019 - - US-based healthcare company Johnson and Johnson's (NYSE: JNJ) The Janssen Pharmaceutical Companies business has received accelerated approval from the US Food and Drug Administration 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, the company said.

Balversa is the first FGFR kinase inhibitor approved by the FDA.

Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.1 Today's 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 2 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)].1 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 (RECIST v1.1) 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.1 Data from the BLC2001 study were presented at the American Society of Clinical Oncology 2018 annual meeting (Abstract #4503) and were recognized as a "Best of ASCO" selection.

The 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.

Janssen is offering Balversa and associated patient services through a single source specialty pharmacy provider, US Bioservices.

This model is part of Janssen's ongoing commitment to provide high-quality products, services, access, and support to healthcare professionals and patients.

Urothelial carcinoma, also known as transitional cell carcinoma, starts in the innermost lining of the bladder.3 It is the most common and frequent form of bladder cancer, representing more than 90% of all bladder cancers.

About one in five patients with mUC have a FGFR genetic alteration. FGFRs are a family of receptor tyrosine kinases which can be activated by genetic alterations in a range of tumor types, and these alterations may lead to increased tumor cell growth and survival.

Balversa is approved specifically for the treatment of patients with mUC harboring FGFR3 or FGFR2 genetic alterations. In the US, it is estimated that up to 3,000 people with urothelial carcinoma will test FGFR positive on an annual basis.

FGFR genetic alterations can be detected through an FDA-approved companion diagnostic.

The five-year survival rate for patients with Stage IV metastatic bladder cancer that has spread to distant parts of the body is currently 5%.

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.

This indication is approved under accelerated approval based on tumor response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

The pivotal multicenter, open-label Phase 2 BLC2001 (NCT02365597) clinical trial evaluated the efficacy and safety of Balversa for the treatment of adults with mUC whose tumors have certain FGFR alterations.

In 2008, Janssen entered into an exclusive worldwide license and collaboration agreement with Astex Pharmaceuticals to develop and commercialise Balversa.

Balversa will be commercially available through the single-source specialty pharmacy provider US Bioservices.

Balversa is a kinase inhibitor indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma which has susceptible FGFR3 or FGFR2 genetic alterations and progressed during or following at least one line of prior platinum-containing chemotherapy including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.