Theriva Biologics’ VCN-01 for retinoblastoma has been granted rare paediatric drug designation (RPDD) by the US Food and Drug Administration (FDA).

The FDA grants RPDD for rare diseases (fewer than 200,000 affected persons in the United States) that are serious and life-threatening and primarily affect children ages 18 years or younger.

VCN-01 is a systemic, selective, stroma-degrading oncolytic adenovirus, which previously received orphan drug designation for the same indication.

“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,” said Steven Shallcross, Chief Executive Officer of Theriva Biologics. “We are encouraged by this important step forward and, in parallel, continue to work closely with leading physicians and regulatory agencies to refine our clinical strategy for VCN-01 as an adjunct to chemotherapy in paediatric patients with advanced retinoblastoma.

“Most recently, results from the investigator sponsored Phase I trial evaluating the safety and activity of intravitreal VCN-01 in paediatric patients with refractory retinoblastoma were determined to be positive by the study Monitoring Committee.”

VCN-01 exerts multiple anti-tumour effects

VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumour cells and degrade the tumour stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment.

This mode-of-action means VCN-01 exerts multiple anti-tumour effects by selectively infecting and lysing tumour cells, enhancing the access and perfusion of co-administered chemotherapy products, and increasing tumour immunogenicity and exposing the tumour to the patient’s immune system and co-administered immunotherapy products.

The therapy has been administered to over 80 patients in Phase I and investigator-sponsored clinical trials of different cancers, including pancreatic ductal adenocarcinoma (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection).

Diana Spencer, Senior Digital Content Editor, DDW