Nadofaragene firadenovec

Nadofaragene firadenovec (nadofaragene firadenovec-vncg) is a recombinant non-replicating adenovirus serotype 5 vector containing a transgene encoding human interferon alfa-2b (IFNα2b). It was approved by the FDA on December 2022 for the treatment of high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is the first gene therapy approved by the FDA for the treatment of bladder cancer.

BCG-unresponsive NMIBC has a high recurrence and is notably difficult to treat. Most patients with this condition undergo radical cystectomy since other non-surgical treatments are far less effective. The use of nadofaragene firadenovec provides a therapeutic alternative to patients seeking non-surgical alternatives for the treatment of BCG-unresponsive NMIBC. Nadofaragene firadenovec is formulated with an excipient (Syn-3) that facilitates gene transfer across the urothelium and promotes the transduction of IFNα2b. The localized expression of this gene induces anti-tumor effects. Nadofaragene firadenovec also has a manageable adverse event profile. Compared to pembrolizumab, a smaller proportion of patients experienced grade 3-4 adverse events (4% vs 12.7%).

Nadofaragene firadenovec is indicated for the treatment of adult patients with high-risk Bacillus CalmetteGuérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.