enGene's Drug in the FDA Approval Process
This section highlights FDA-related milestones and regulatory updates for drugs developed by enGene (ENGN).
Over the past two years, enGene has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
EG-70. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
EG-70 FDA Regulatory Timeline and Events
EG-70 is a drug developed by enGene for the following indication: for Expedited Review in High-Risk, Non-Muscle Invasive Bladder Cancer.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- EG-70
- Announced Date:
- December 2, 2025
- Indication:
- for Expedited Review in High-Risk, Non-Muscle Invasive Bladder Cancer
Announcement
enGene Holdings Inc. 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).
AI Summary
enGene Holdings announced that the U.S. Food and Drug Administration selected its investigational non‑viral gene therapy, detalimogene voraplasmid (detalimogene), to join the CMC Development and Readiness Pilot (CDRP) Program. Detalimogene is being developed as an intravesical treatment for patients with high‑risk non‑muscle invasive bladder cancer (NMIBC).
The CDRP Program supports chemistry, manufacturing, and controls (CMC) development for products with compressed clinical timelines and aims to help patients gain earlier access to promising therapies. enGene is one of nine companies chosen for the program each year. Because detalimogene has already been manufactured at commercial scale, participation in CDRP will provide additional, structured FDA CMC interactions to help ensure manufacturing readiness ahead of a potential Biologics License Application (BLA) submission in the second half of 2026.
enGene says the CDRP engagement should strengthen CMC planning and align manufacturing with regulatory expectations as clinical development proceeds toward possible approval.
Read Announcement- Drug:
- EG-70
- Announced Date:
- November 11, 2025
- Indication:
- for Expedited Review in High-Risk, Non-Muscle Invasive Bladder Cancer
Announcement
enGene Holdings Inc. today reported additional preliminary data from the pivotal cohort of its ongoing, Phase 2 LEGEND trial of detalimogene voraplasmid (also known as detalimogene and previously EG-70) in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) patients with carcinoma in situ (CIS) with or without concomitant papillary disease.
AI Summary
enGene reported additional preliminary data from the pivotal cohort of its Phase 2 LEGEND trial of detalimogene voraplasmid in high‑risk, BCG‑unresponsive non‑muscle invasive bladder cancer with carcinoma in situ. The pivotal cohort completed enrollment at 125 patients, exceeding the target. An analysis of patients treated under an amended protocol (62 patients with 3‑month data; 37 with 6‑month data) showed a 63% complete response (CR) rate at any time, 56% CR at 3 months, and 62% CR at 6 months (including four patients who converted to CR after reinduction). All five patients assessed at 9 months had a CR.
Safety among 125 patients was favorable: 42% experienced a treatment‑related adverse event, mostly Grade 1–2; three patients had Grade 3 events. Dose interruptions occurred in 1.6% and discontinuations in 0.8%. Common side effects included fatigue, dysuria, bladder spasm, urgency and pollakiuria. Other LEGEND cohorts continue to enroll, and enGene plans to use these results to support a planned Biologics License Application in the second half of 2026.
Read Announcement- Drug:
- EG-70
- Announced Date:
- September 3, 2025
- Indication:
- for Expedited Review in High-Risk, Non-Muscle Invasive Bladder Cancer
Announcement
enGene Holdings Inc. announced it has achieved its target enrollment milestone of 100 patients for the pivotal cohort of its ongoing, open-label, multi-cohort Phase 2 LEGEND trial of detalimogene voraplasmid ("detalimogene" and previously EG-70) in patients with high-risk, non-muscle invasive bladder cancer (NMIBC).
AI Summary
enGene Holdings Inc. announced it has enrolled 100 patients in the pivotal cohort of its Phase 2 LEGEND trial studying detalimogene voraplasmid in high-risk, non-muscle invasive bladder cancer (NMIBC) patients with carcinoma in situ (CIS), with or without papillary disease. The open-label, multi-cohort study remains open to screening and may overenroll beyond the initial target.
The company expects to share data from LEGEND’s pivotal cohort in the fourth quarter of 2025 and plans to submit a Biologics License Application in the second half of 2026. Overenrollment could lead to a slight shift in that timeline, offering more robust safety and efficacy insights.
“Reaching our enrollment goal for detalimogene in LEGEND’s pivotal cohort marks a key milestone,” said Ron Cooper, CEO of enGene. “It brings us closer to delivering the first non-viral gene therapy option that balances strong anti-tumor activity with safety and ease of use.”
Read Announcement- Drug:
- EG-70
- Announced Date:
- June 25, 2025
- Indication:
- for Expedited Review in High-Risk, Non-Muscle Invasive Bladder Cancer
Announcement
enGene Holdings Inc. 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).
AI Summary
enGene Holdings Inc. announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead investigational therapy, detalimogene voraplasmid. This designation is a key milestone for the company as it highlights the potential of the therapy to address a serious unmet need in patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS).
The RMAT status is part of an FDA program designed to speed up the development and review process for treatments that can impact life-threatening conditions. With this designation, enGene will have early and frequent interactions with the FDA, along with potential benefits like a rolling review and priority review. This progress brings hope for a first-in-class, gene-based immunotherapy that could provide a new treatment option for bladder cancer patients facing limited alternatives.
Read Announcement
enGene FDA Events - Frequently Asked Questions
As of now, enGene (ENGN) has not received any FDA approvals for its therapy in the last two years.
In the past two years, enGene (ENGN) has reported FDA regulatory activity for EG-70.
The most recent FDA-related event for enGene occurred on December 2, 2025, involving EG-70. The update was categorized as "Provided Update," with the company reporting: "enGene Holdings Inc. 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)."
Currently, enGene has one therapy (EG-70) targeting the following condition: for Expedited Review in High-Risk, Non-Muscle Invasive Bladder Cancer.
More FDA Event Resources from MarketBeat
Companies With Recent FDA Events
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
FDA progress for NASDAQ:ENGN last updated on 12/2/2025 by MarketBeat.com Staff. We continuously monitor for new FDA events and market data.
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