FDA Grants Rare Pediatric Drug Designation to Theriva Biologics' VCN-01

The FDA has awarded VCN-01, a rare paediatric drug designation (RPDD), to TherivaTM Biologics, a diversified clinical-stage company that develops therapeutics to treat cancer and related diseases in high-unmet need areas. The FDA made this announcement today. VCN-01 is being used to treat retinoblastoma. Theriva's primary product candidate, VCN-01, is an oncolytic adenovirus that selectively degrades stroma and is systemic. VCN-01 was formerly designated as an orphan medication by the FDA to treat retinoblastoma.

Theriva Biologics CEO Steven A. Shallcross stated, "The FDA's decision to grant rare paediatric drug designation to VCN-01 highlights the urgent need for new treatment options for paediatric patients with retinoblastoma." "This significant advancement gives us hope, and we will keep collaborating closely with top medical professionals and government organisations to improve our clinical approach for VCN-01 as a chemotherapy adjuvant in paediatric patients with advanced retinoblastoma. The study Monitoring Committee most recently declared that the findings of the investigator-sponsored Phase 1 trial, which assessed the efficacy and safety of intravitreal VCN-01 in paediatric patients with refractory retinoblastoma, were favourable. The information obtained from this study will help to improve our clinical development strategy in this highly unmet need sector.

Less than 200,000 people in the US have rare diseases that are considered severe and potentially fatal, especially affecting children under the age of 18. The FDA authorises RPDD for certain conditions. Theriva may be eligible to obtain a Priority Review Voucher if the FDA approves a Biologics License Application for VCN-01 for the treatment of retinoblastoma. This voucher can be sold or transferred, or used to get a priority review for any later marketing application.