Theriva Biologics, a clinical-stage biotechnology company focused on developing therapies for cancer and related diseases in underserved areas, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead clinical candidate, VCN-01, for the treatment of pancreatic cancer.
Pancreatic ductal adenocarcinoma (PDAC) has one of the lowest survival rates among all cancers, and there is an urgent need for new treatment options. VCN-01 is an oncolytic adenovirus designed to selectively replicate and aggressively target tumor cells, degrading the tumor stroma barrier that serves as a physical and immunosuppressive barrier to cancer treatment.
The orphan drug designation by the FDA provides various benefits to support the development of novel drugs, including potential market exclusivity for seven years following FDA approval, eligibility for tax credits for qualified clinical trials, waiver of application fees, reduction of annual product fees, assistance in clinical protocols, and potential qualification for accelerated development programs.
Steven A. Shallcross, CEO of Theriva Biologics, stated, «The FDA’s decision to grant orphan drug designation to VCN-01 underscores the urgent need for new treatment options for patients with PDAC. Standard treatment efforts have largely reached a plateau, despite the increasing incidence of PDAC, and the need for novel therapies in this indication is pressing.»
VCN-01 is currently being evaluated in the VIRAGE phase 2b multinational clinical trial in combination with standard chemotherapy (gemcitabine/nab-paclitaxel) as first-line treatment for patients with PDAC. The primary endpoints of the trial include overall survival and safety/tolerability of VCN-01.
Theriva Biologics is also developing other clinical candidates, including SYN-004, designed to degrade certain commonly used intravenous antibiotics within the gastrointestinal tract to protect the microbiome, and SYN-020, an oral recombinant formulation of intestinal alkaline phosphatase for the treatment of GI and systemic diseases.
The orphan drug designation for VCN-01 highlights the promising potential of this therapy to address the unmet medical need for PDAC treatment. Theriva Biologics is committed to advancing the development of innovative therapies for cancer and related diseases, with the goal of improving patient outcomes and quality of life.
