ViGeneron Announces FDA Rare Pediatric Disease Designation for VG901; DSMB Approval to Advance Dose Escalation in Phase 1b RP Trial

ViGeneron announced two important milestones for its novel gene therapy candidate VG901 to treat patients with retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. The FDA granted Rare Pediatric Disease Designation (RPDD) to VG901, and the independent Data Safety Monitoring Board (DSMB) has unanimously approved dose escalation in the ongoing phase 1b clinical trial. 

The FDA’s RPDD acknowledges the need for treatments addressing rare pediatric conditions like retinitis pigmentosa caused by CNGA1 mutations. This designation qualifies ViGeneron to potentially secure a priority review voucher (PRV) upon VG901’s marketing approval. A PRV enables accelerated FDA review of any drug candidate in the company’s pipeline, reducing the review period to 6 months, and is transferable for use or sale. 

“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,” Dr. Caroline Man Xu, ViGeneron’s Co-founder and CEO, said in a company news release. “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.” 

The ongoing phase 1b trial is designed to evaluate the safety and preliminary efficacy of VG901, which leverages ViGeneron’s novel next-generation vgAAV capsid to deliver the functional CNGA1 gene to retinal photoreceptor cells via intravitreal injection. The DSMB, composed of independent experts, conducts a rigorous evaluation of trial data after each cohort of patients. 

“The DSMB has unanimously recommended proceeding with dose escalation in the ongoing VG901 phase 1b clinical trial,” said Dr. Bart P Leroy, ophthalmologist & clinical geneticist, Head of Department of Ophthalmology, Ghent University Hospital, Belgium, and the DSMB Chair for this trial. "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.”