In VIRAGE, the ongoing multinational Phase 2b clinical study, intravenous VCN-01 is being evaluated in combination with standard-of-care (SoC) chemotherapy (gemcitabine/nab-paclitaxel) as a first line therapy for patients with pancreatic ductal adenocarcinoma (PDAC). Previously, the FDA granted orphan drug designation to VCN-01 for treatment of retinoblastoma.

“The FDA’s decision to grant orphan drug designation to VCN-01 highlights the urgent need for new treatment options for patients with PDAC, which has one of the lowest survival rates among all cancers,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “Efforts to improve upon the standard of care treatment have largely stalled, despite the growing incidence of PDAC, and the need for novel therapies in this indication is acute. The growing clinical data that underscore VCN-01’s multiple modes of action and the compelling clinical outcomes observed in Phase 1 studies of VCN-01 in combination with chemotherapy or immunotherapy in patients with PDAC and other solid tumors, give us confidence that VCN-01 has the potential to address this unmet medical need.”

The FDA’s Office of Orphan Products Development grants orphan status to drugs being developed to treat, diagnose, or prevent a rare disease or condition affecting fewer than 200,000 people in the United States. Orphan Drug Designation is designed to provide drug developers with various benefits to support the development of novel drugs, including the potential for market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, waiver of application fees, reduced annual product fees, clinical protocol assistance and potential qualification for expedited development programs.