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Regenerative Medicine Advanced Therapy (RMAT) designation provides access to accelerated development tools
Designation recognizes the potential of detalimogene to address significant unmet need
enGene Holdings Inc. (NASDAQ:ENGN, or "enGene" or the "Company"))), a clinical-stage, non-viral gene-based immunotherapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to detalimogene voraplasmid (also known as detalimogene, and previously EG-70), 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 (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.
"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 Officer 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."
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.
Detalimogene is designed for streamlined administration in urology clinics — including community practices, where approximately 70% of urologists provide care. The therapy is being evaluated for its ability to treat NMIBC though the non-viral stimulation of a local immune response in the bladder, presenting a potentially transformative option for patients who otherwise face limited choices beyond radical cystectomy.
About Non-Muscle Invasive Bladder Cancer (NMIBC)
Non-muscle invasive bladder cancer (NMIBC) is a disease that poses a significant burden on both patients and clinics and has a massive economic impact on our healthcare system. NMIBC occurs when cancer cells grow in the tissues that line the interior of the bladder, but the cancer has not yet penetrated the muscle of the bladder wall. NMIBC can take the form of papillary outgrowths from the bladder wall, which are typically resected, or carcinoma in situ (CIS), flat, multifocal lesions that are unable to be resected, and the two can co-occur. About 75-80% of new bladder cancer diagnoses are NMIBC. Patients suffering from high-risk NMIBC who are unresponsive to the standard of care, Bacillus Calmette-Guérin (BCG), face high rates of disease recurrence (50-70%) and are subject to full removal of the bladder (cystectomy) as a curative but life-altering next step.
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