FDA grants RMAT designation for enGene’s detalimogene

The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to detalimogene voraplasmid (previously known as EG-70), Canadian company enGene’s (Nasdaq: ENGN) lead investigational therapy for the treatment of high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS).

The RMAT program is intended to expedite the development and review of regenerative medicine therapies for serious or life-threatening conditions, where preliminary clinical evidence suggests potential to address unmet medical needs. This designation provides enGene with several regulatory advantages, including early and frequent engagement with the FDA, potential for rolling review and priority review, and other benefits like Fast Track and Breakthrough Therapy designations. The news pushed enGene’s shares up 9% to $3.26.

“Receiving the RMAT designation highlights the promising profile of detalimogene and its potential to address the high unmet need in NMIBC,” stated Ron Cooper, chief executive of enGene. “Bladder cancer patients with limited options cannot wait, and we are enthusiastic about potentially expediting the regulatory process to bring a first-in-class treatment to patients,” he added.