FDA OK for first gene therapy for high-risk, non-muscle-invasive bladder cancer

The US Food and Drug Administration has approved Adstiladrin (nadofaragene firadenovec-vncg), a non-replicating (cannot multiply in human cells) adenoviral vector-based gene therapy indicated for the treatment of 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.

The FDA granted approval of Adstiladrin to privately-held Swiss drugmaker Ferring Pharmaceuticals, reversing an earlier decision in May 2020, when it rejected the drug, citing outstanding questions for the company’s manufacturing partner.

Just last month, Ferring also gained FDA approval for its first fecal microbiota product in the shape of Rebyota (fecal microbiota, live-jslm).