enGene Holdings Inc. , a clinical-stage, non-viral genetic medicines company, announced that the U.S. Food and Drug Administration (FDA) has selected detalimogene voraplasmid (also known as detalimogene and previously EG-70) to participate in the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program.Detalimogene is a novel, investigational, non-viral gene therapy for patients with high-risk, non-muscle invasive bladder cancer (NMIBC).
“Manufacturing readiness in drug development is often underappreciated. We have already scaled detalimogene manufacturing to commercial-level, and CDRP is expected to help ensure CMC readiness for filing and commercialization,” said Ron Cooper, President and Chief Executive Officer. “Our progress on the manufacturing front is in sync with detalimogene clinical development, where we recently announced improved six-month complete response rate in the LEGEND trial pivotal cohort.”
The FDA created the CDRP Program to facilitate CMC development for therapies with compressed clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy. The initiative is designed to promote earlier and more structured engagement between sponsors and FDA on CMC development strategies, and since its inception, has led to increased collaboration with the FDA so sponsors can confidently scale up manufacturing capacity while clinical development is ongoing. Acceptance into the program further supports enGene’s confidence in detalimogene’s potential and aligns with enGene’s commitment to strong manufacturing practices as clinical development advances.
Last month, enGene reported positive preliminary results from the LEGEND trial’s pivotal cohort, studying detalimogene in patients with high-risk, BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without concomitant papillary disease. Results showed an improved complete response (CR) rate of 62% at six months. Safety data from 125 patients demonstrated a favorable tolerability profile, with low rates of treatment-related adverse events (42%) and dose interruptions (1.6%).
Detalimogene has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA. The Company plans to submit a Biologics License Application (BLA) for detalimogene in the second half of 2026.
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 the 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 present as papillary outgrowths from the bladder wall, which are typically resected, or as carcinoma in situ (CIS), which consists of flat, multifocal lesions that cannot be resected. The two forms can also 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 potentially subject to full removal of the bladder (cystectomy) as a curative but life-altering next step.
About Detalimogene Voraplasmid
Detalimogene is a novel, investigational, non-viral gene therapy for patients with high-risk, non-muscle invasive bladder cancer (NMIBC), including Bacillus Calmette-Guérin (BCG)-unresponsive disease. It is designed to be instilled in the bladder and elicit a powerful yet localized anti-tumor immune response.
Detalimogene was developed using the Company’s Dually Derivatized Oligochitosan (DDX) platform, a technology designed to transform how gene therapies are accessed by patients and utilized by clinicians. Medicines developed with the DDX platform can potentially overcome the limitations of viral-based gene therapies, reduce complexities related to safe handling and cold storage, and streamline both manufacturing processes and administration paradigms.
Detalimogene has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration (FDA) based on its potential to address the high unmet medical need for patients with BCG-unresponsive, carcinoma in situ (CIS) NMIBC with or without resected papillary tumors who are unable to undergo cystectomy. 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 the potential to address unmet medical needs. Similarly, Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
Health Technology Insights: BOOST and American Diabetes Association Partner to Promote Diabetes Awareness
To participate in our interviews, please write to our HealthTech Media Room at info@intentamplify.com
Source- businesswire
