Theriva Biologics' lead clinical candidate, VCN-01, a systemically administered oncolytic adenovirus, has received orphan medicinal product designation from the European Commission for the treatment of retinoblastoma. This decision underscores the critical need for innovative therapies for this rare and challenging pediatric cancer. The United States Food and Drug Administration (FDA) had previously granted VCN-01 orphan drug and rare pediatric disease designations for the same indication.
Significance of Orphan Drug Designation
The European Medicines Agency (EMA) recommends orphan designation for therapies addressing life-threatening or chronically debilitating conditions with a prevalence of no more than 5 in 10,000 individuals in the EU. The designation also requires that either no satisfactory method of diagnosis, prevention, or treatment exists, or that the new medicine offers significant benefit over existing options. Orphan designation provides developers with incentives, including 10 years of market exclusivity upon marketing authorization in the EU, protocol assistance, and reduced regulatory fees.
VCN-01: A Novel Oncolytic Adenovirus
VCN-01 is designed to selectively replicate within tumor cells and degrade the tumor stroma, which acts as a physical and immunosuppressive barrier. This mechanism of action allows VCN-01 to exert multiple antitumor effects, including lysing tumor cells, enhancing the access of co-administered chemotherapy, and increasing tumor immunogenicity. Its systemic administration allows it to target both the primary tumor and metastases.
Clinical Development and Previous Findings
Steven A. Shallcross, Chief Executive Officer of Theriva Biologics, stated, "We are very pleased with the European Commission’s grant of orphan medicinal product designation to VCN-01, emphasizing the urgent need for new treatment options for retinoblastoma." He also noted encouraging results from an investigator-sponsored Phase 1 trial evaluating the safety and activity of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma. Theriva is collaborating with physicians and regulatory agencies to refine the clinical strategy for VCN-01 as an adjunct to chemotherapy.
Retinoblastoma: An Unmet Need
Retinoblastoma is the most common type of eye cancer in children, with an incidence of approximately 1 in 14,000 to 1 in 18,000 live newborns. It accounts for 15% of tumors in children under one year old, with an average diagnosis age of two years. In Europe, the estimated incidence rate is 1 per 13,844 live births, with approximately 300 children diagnosed annually. Despite advancements, preserving life, preventing eye loss and blindness, and mitigating treatment-related side effects remain significant challenges, especially in low-resource countries where children face higher risks of eye loss and metastatic disease.
