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

Funding The preparation of this review was not supported by any external funding.

Authorship and conflict of interest T. Nie is a salaried employee of Adis International Ltd/Springer Nature and declares no relevant conflicts of interest. All authors contributed to this article and are responsible for its content.

Ethics approval, Consent to participate, Consent for publication, Availability of data and material, Code availability Not applicable.

Additional information about this Adis Drug Review can be found here.

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.