Nadofaragene firadenovec in high-risk Bacillus Calmette Guérin unresponsive non-muscle invasive bladder cancer: a profile of its use

Nadofaragene firadenovec (Adstiladrin®) is an important bladder-sparing option in the treatment of patients with high-risk Bacillus Calmette Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC). Radical cystectomy is the recommended treatment in these patients; however, many are ineligible or refuse to undergo this major procedure and other options are limited. Intravesical nadofaragene firadenovec, a replication-deficient adenovirus-based gene therapy that causes localized expression of interferon (IFN) α2b in the bladder, is approved in the USA for the treatment of adults with high-risk BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors. In a phase 3 clinical trial including patients with BCG-unresponsive NMIBC with CIS, nadofaragene firadenovec was efficacious in producing complete responses. Nadofaragene firadenovec had an acceptable safety profile and was generally well tolerated, with a small number of patients experiencing a grade 3 treatment-related adverse event and none experiencing a grade 4 or 5 drug-related adverse event. Cystectomy should be considered in patients who do not have a complete response to nadofaragene firadenovec or who have recurrence of CIS.