ViGeneron Snags FDA Rare Pediatric Disease Designation for CNGA1-Associated Retinitis Pigmentosa Gene Therapy VG901
In addition to the new designation, ViGeneron also announced that the data safety monitoring board for the trial has given the green light for dose escalation.
Bart P. Leroy, MD, PhD
ViGeneron’s VG901, an investigational adeno-associated virus (AAV) vector-based gene therapy being evaluated for the treatment of retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene in a phase 1b clinical trial (NCT06291935), has been granted rare pediatric disease designation (RPDD) by the FDA.1
In addition to the new designation, ViGeneron also announced that the data safety monitoring board (DSMB) for the trial has given the green light for dose escalation. VG90 is intended to deliver a copy of CNGA1 to retinal photoreceptor cells via a proprietary AAV capsid referred to as vgAAV. It is administered by intravitreal injection.
“This RPDD recognition from the FDA highlights the significant unmet medical need in retinitis pigmentosa and underscores VG901’s therapeutic potential as the first-in-class and only clinical-stage therapy targeting retinitis pigmentosa associated with mutations in the CNGA1 gene,” Caroline Man Xu, PhD, ViGeneron’s cofounder and chief executive officer, said in a statement.1 “In addition to the previously granted FDA orphan drug designation for VG901, the RPDD designation further supports our efforts to accelerate the development of VG901.”
VG901 received orphan drug designation from the FDA in April 2024, around the same time that the first patient in the trial was dosed.2 According to the clinicaltrials.gov page for the study, which was most recently updated on March 4, 2024, the trial is recruiting patients at the Center for Ophthalmology, University of Tuebingen, in Tuebingen, Germany.
“The DSMB has unanimously recommended proceeding with dose escalation in the ongoing VG901 Phase 1b clinical trial,” DSMB chair Bart P. Leroy, MD, PhD, the head of the Department of Ophthalmology at the Center for Medical Genetics at Ghent University Hospital, added to the statement.1 “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.”
ViGeneron notes that the ability of VG901 to deliver CNGA1 was previously confirmed in mouse model research and that safety, durable expression, and sustained tolerability has been demonstrated in the 6-month observation period of a good laboratory practice safety study. According to the company, autosomal recessive RP can be attributed to CNGA1 mutations in about 2% to 8% of cases.
Notably, ViGeneron is not the only company to have announced an update on an RP gene therapy product in the past week.3 On January 13, Ocugen reported that OCU400, an investigational gene therapy that uses a gene-agnostic mechanism to target RP, had demonstrated the ability to sustain or improve visual function in comparison to untreated eyes in patients with RP treated in a phase 1/2 clinical trial (NCT05203939), according to newly announced 2-year follow-up data. At 2 years posttreatment, the improvements recorded in visual function compared to untreated fellow eyes were determined to be statistically significant (P = .01) without regard to the underlying mutation causing the patient’s RP.
"It is truly remarkable to see the significant improvements in visual acuity in patients treated with OCU400 sustained at 2 years,” Syed M. Shah, MD, FACS, vice chair for research and digital medicine, and the director of retina service at Gundersen Health System, La Crosse, Wisconsin, said in a January 13 statement.3 “The broad spectrum of genes and mutations causing RP presents a unique challenge in developing treatments for this unmet need. This is where the promise of mutation-agnostic therapies becomes particularly compelling. OCU400’s demonstrated effectiveness across multiple mutations not only offers hope to RP patients but also opens new possibilities for treating other retinal diseases."
