Theriva’s VCN-01 Granted EU Orphan Status for Retinoblastoma Treatment

Theriva Biologics (NYSE American: TOVX), a clinical-stage company developing cancer therapeutics, announced that the European Commission has granted orphan medicinal product designation to its lead candidate, VCN-01, for the treatment of retinoblastoma, following a recommendation from the European Medicines Agency (EMA). VCN-01 is a systemic, stroma-degrading oncolytic adenovirus. The FDA has previously granted orphan drug and rare pediatric disease designations to VCN-01 for retinoblastoma.

CEO Steven A. Shallcross highlighted the significance of the designation, underscoring the need for new treatment options for retinoblastoma. He also noted the positive results from an investigator-sponsored Phase 1 trial assessing VCN-01’s safety and activity in pediatric patients with refractory retinoblastoma. Theriva is collaborating with top physicians and regulatory agencies to advance VCN-01 as a potential adjunct therapy to chemotherapy for children with this difficult-to-treat disease.

Orphan designation in the EU is granted to products aimed at treating life-threatening or chronically debilitating diseases with a prevalence of fewer than 5 in 10,000 individuals. It offers benefits such as 10 years of market exclusivity, regulatory support, and reduced fees to promote the development of novel therapies.