The U.S. FDA has granted regenerative medicine advanced therapy (RMAT) designation to enGene Holdings’ non-viral gene-based immunotherapy, detalimogene voraplasmid, the company’s lead investigational therapy for the treatment of high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ.
The designation was based on previously disclosed preliminary results from the ongoing pivotal LEGEND study, which demonstrated compelling clinical activity and a generally favorable tolerability profile in patients with BCG-unresponsive NMIBC with CIS. The RMAT designation provides enGene with several regulatory advantages, including early and frequent engagement with the FDA and the potential for rolling review and priority review.
enGene’s detalimogene is designed to be instilled in the bladder and elicit a powerful yet localized anti-tumor immune response. It was developed using the company’s Dually Derivatized Oligochitosan platform, a technology designed to overcome the limitations of viral-based gene therapies, simplify safe handling and cold storage complexities, and streamline both manufacturing processes and administration paradigms.
If approved, detalimogene will compete with Ferring’s adenovirus vector-based gene therapy, Adstiladrin, approved in December 2022 for the treatment of BCG-unresponsive NMIBC with CIS with or without papillary tumors.
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