FDA grants rare pediatric disease designation for retinitis pigmentosa gene therapy candidate VG901 from ViGeneron

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ViGeneron GmbH, a clinical-stage gene therapy company based in Munich, Germany, shared details of two new milestones in an ongoing clinical trial of its therapeutic candidate VG901 (NCT06291935). In a press release, ViGeneron stated that the US Food and Drug Administration (FDA) granted VG901 a Rare Pediatric Disease Designation (RPDD); in addition, the independent Data Safety Monitoring Board (DSMB) unanimously approved dose escalation in the phase 1b trial.¹

VG901 uses a novel, next-generation adeno-associated virus (AAV) vector to treat CNGA1-associated retinitis pigmentosa (RP). The proprietary AAV, called vgAAV, delivers the CNGA1 gene via intravitreal injection rather than subretinal administration to avoid retinal damage, the company stated. RP is the most common type of inherited retinal disease, estimated to affect 1 in 4,000 people in Europe and 1 in 3,500 in the US.¹

The RPDD may qualify ViGeneron for a priority review voucher upon marketing approval for VG901, which would enable accelerated FDA review (reducing the review period to 6 months). The voucher can be used for review of any drug candidate in ViGeneron’s pipeline, and is transferable for use or sale, according to the press release. The FDA previously issued Orphan Drug Designation for VG901.

Meanwhile, the dose escalation approval reflects a unanimous decision from the DSMB, a group of independent experts who conduct rigorous review of trial data following each patient cohort. The ongoing Phase 1b trial is designed to evaluate VG901 for safety and preliminary efficacy.

Bart P Leroy, MD, PhD, is head of the Department of Ophthalmology at Ghent University Hospital in Belgium, and the DSMB Chair for the clinical trial. “The DSMB has unanimously recommended proceeding with dose escalation in the ongoing VG901 Phase 1b clinical trial,” Prof Leroy said in the press release. “No dose-limiting adverse events related to VG901 have been reported in the first-dose cohort to date. This marks a critical step toward advancing to the higher dose and represents an important milestone in its clinical development.”

In addition to VG901, ViGeneron's portfolio includes two additional next-generation gene therapy platforms. The REconstitution Via mRNA Trans-splicing technology platform (REVeRT) is engineered for efficient recostitution of large genes (>5kb), targed with a given capsid. The AAV Transactivation platform is a CRISPR-Cas-based AAV gene therapy, which allows regulation of one or more genes in vivo.

The European Medicines Agency issued approval for the Clinical Trial Application for VG901 in August of 2023.² The first patient was dosed in the phase 1b trial in April 2024.

References

1. ViGeneron Announces FDA Rare Pediatric Disease Designation for VG901 and DSMB Approval to Advance Dose Escalation in Phase 1b Retinitis Pigmentosa Trial. ViGeneron GmbH. Press release. Published January 8, 2025. Accessed January 8, 2025. https://vigeneron.com/press/vigeneron-announces-fda-rare-pediatric-disease-designation-for-vg901-and-dsmb-approval-to-advance-dose-escalation-in-phase-1b-retinitis-pigmentosa-trial/

2. Hayes H. EMA approves Clinical Trial Application for retinitis pigmentosa therapy from ViGeneron. Ophthalmology Times Europe. Published August 22, 2023. Accessed January 8, 2025. https://europe.ophthalmologytimes.com/view/ema-approves-clinical-trial-application-retinitis-pigmentosa-therapy-from-vigeneron