On February 8, 2022 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company developing therapeutics designed to treat diseases in areas of high unmet need, reported that VCN Biosciences, S.L.’s (VCN) VCN-01 received Orphan Drug Designation for retinoblastoma from the U.S. Food & Drug Administration (FDA) (Press release, Synthetic Biologics, FEB 8, 2022, View Source [SID1234607854]). This announcement follows Synthetic Biologics’ recent announcement that it had entered a definitive agreement to acquire VCN, which is subject to conditions that must be met prior to closing. VCN is developing a new oncolytic adenovirus (OV) platform designed to trigger tumor cell death and promote immune cell infiltration into tumors.
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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.
Steven Shallcross, Chief Executive Officer and Chief Financial Officer of Synthetic Biologics, commented, "We are pleased to report that VCN’s VCN-01 was granted Orphan Drug Designation for retinoblastoma by the FDA. We believe VCN-01 may represent a novel rescue therapy for patients who fail standard therapy, or may be used as an adjunct to chemotherapy, to provide improved outcomes for these patients. We are highly encouraged by the preliminary clinical data thus far and look forward to conducting a pivotal Phase 2/3 trial in these patients following our expected completion of the acquisition."