Theriva Biologics (NYSE American:TOVX) has announced that the European Commission (EC) has adopted the European Medicines Agency’s (EMA) recommendation to grant orphan medicinal product designation to its lead candidate, VCN-01, for the treatment of retinoblastoma.
The company highlights that retinoblastoma, a tumor that originates in the retina, is the most common type of pediatric eye cancer accounting for 15% of tumors in children under one year of age. VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma, which serves as a significant physical and immunosuppressive barrier to cancer treatment.
“We are very pleased with the EC’s grant of orphan medicinal product designation to VCN-01, emphasizing the urgent need for new treatment options for retinoblastoma,” said Steven A. Shallcross, chief executive officer of Theriva Biologics.
“We have previously reported encouraging results from an investigator sponsored Phase 1 trial evaluating the safety and activity of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma, and we are working closely with leading physicians and regulatory agencies worldwide to refine our clinical strategy for VCN-01 as an adjunct to chemotherapy in children with this challenging disease,” he adds.
