FDA OK’s Ferring’s Bladder Cancer Gene Therapy

FDA has approved a Ferring Pharmaceuticals BLA for Adstiladrin (nadofaragene firadenovec-vncg), a non-replicating adenoviral vector‑based gene therapy indicated for treating adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

Approval is based on safety and effectiveness data from a multicenter clinical study that included 157 patients with high-risk BCG-unresponsive NMIBC, 98 of whom had BCG-unresponsive CIS with or without papillary tumors, the agency says in a release. “Overall, 51% of enrolled patients using Adstiladrin therapy achieved a complete response (the disappearance of all signs of cancer as seen on cystoscopy, biopsied tissue, and urine),” it says. The median duration of response was 9.7 months, and 46% of responding patients remained in complete response for at least one year.